OrbiMed

Garuda Therapeutics is a biotech company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening diseases, particularly those affecting blood, bone marrow, immunity, and metabolism. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants by leveraging its innovative platform technology to generate self-renewing blood stem cells. This approach is designed to ensure rapid and broad access to consistent, durable, HLA-compatible, and transgene-free blood stem cell therapies for patients. Garuda Therapeutics employs advanced methodologies, including the use of zebrafish, mice, and human-induced pluripotent stem cells, to clone and analyze novel genes and mechanisms critical for blood formation, ultimately developing mechanism-based therapies that address unmet medical needs.

Ikena Oncology, Inc. is a biotechnology company focused on discovering and developing biomarker-driven therapies for cancer treatment. It specializes in precision oncology by targeting pathways essential for cancer growth and therapeutic resistance, particularly within the Hippo and RAS signaling networks. The company's portfolio includes several preclinical and discovery-stage programs, such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-412, a kynurenine-degrading enzyme. Additionally, Ikena is developing IK-930, an oral small-molecule inhibitor of the TEAD transcription factor involved in the Hippo pathway. Ikena Oncology, originally founded as Kyn Therapeutics in 2016, rebranded in December 2019 and is headquartered in Boston, Massachusetts. The company aims to address significant medical needs in oncology through innovative drug development.

enGene, Inc. is a biotechnology company specializing in mucosal immunotherapy platforms aimed at treating inflammatory bowel disease and diabetes. The company has developed a non-integrating biopolymer-based nucleotide delivery technology that enables localized delivery of immune-modulating proteins to mucosal tissues, including the gastrointestinal tract, lung, and bladder. This innovative approach allows for the treatment of various immune disorders and supports systemic release of proteins from the gut to address conditions such as diabetes, anemia, and hemophilia. Founded in 1999 and based in Vancouver, Canada, enGene has formed a strategic alliance with Takeda Pharmaceutical Company Ltd. The company's platform facilitates the induction or suppression of protein expression levels, which can help regenerate physiologic, meal-regulated insulin secretion for diabetes patients.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

Asceneuron SA is a biotechnology company based in Lausanne, Switzerland, founded in 2012, that focuses on discovering and developing therapeutics for neurodegenerative disorders with significant unmet medical needs. The company targets conditions such as progressive supranuclear palsy, orphan tauopathies, Alzheimer’s disease, and Parkinson’s disease. Asceneuron aims to develop orally bioavailable small molecule drugs that address both symptomatic relief and disease progression for these debilitating conditions. Tauopathies, characterized by the accumulation of tau protein deposits in the brain, are a key area of focus, as they currently lack effective treatments. With the increasing prevalence of Alzheimer's disease posing a major healthcare challenge, Asceneuron aspires to become a leading entity in the biotech sector by providing innovative and more effective therapeutic options for patients suffering from neurodegenerative diseases. The company operates as a subsidiary of Merck KGaA.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

Fate Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing programmed cellular immunotherapies for cancer and immune disorders. The company specializes in NK- and T-cell immuno-oncology programs, with a diverse pipeline that includes candidates such as FT516 and FT596 for treating various hematologic malignancies, FT538 for acute myeloid leukemia and multiple myeloma, and FT500 for advanced solid tumors. Fate Therapeutics employs advanced techniques involving induced pluripotent stem cells (iPSCs) to create engineered product candidates, which are designed to be off-the-shelf solutions. The company has established strategic collaborations with notable partners, including Ono Pharmaceutical Co. Ltd. and Janssen Biotech, to enhance its research and development capabilities. Founded in 2007, Fate Therapeutics aims to leverage cutting-edge science to create innovative therapies that restore health by modulating the properties of stem cells.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

BioAge Labs, Inc. is a biotechnology company focused on developing therapies to address aging and age-related diseases. The company utilizes a systems biology and artificial intelligence platform to identify key drug targets that influence aging and analyze extensive datasets to uncover the molecular drivers of age-related conditions. BioAge Labs is advancing several drug candidates, including BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor designed to activate genes involved in critical biological processes such as tissue regeneration and vascular remodeling. Additionally, BGE-175, an orally administered inhibitor of the prostaglandin D2 DP1 signaling pathway, aims to mitigate risks associated with immune aging and respiratory infections. Founded in 2015 and based in Richmond, California, BioAge Labs is committed to discovering and developing innovative treatments that harness the biology of human aging.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

Rampart Bioscience specializes in the development of gene medicines aimed at providing long-lasting treatments for a broad array of diseases. The company has created proprietary development and delivery platforms that enable the production of optimized therapeutics in a non-viral format. By integrating expertise from various scientific disciplines, such as gene delivery, protein sciences, and clinical translation, Rampart Bioscience focuses on assisting patients with genetically driven conditions, ultimately enhancing the effectiveness and safety of therapeutic interventions.

Minervax is focused on developing a vaccine to prevent infections caused by Group B streptococcus (GBS), which can lead to serious health issues in newborns and adverse pregnancy outcomes. The company is researching a specific fusion protein that has demonstrated the ability to stimulate strong protective immunity against various clinically significant GBS strains. By utilizing this innovative approach, Minervax aims to provide medical professionals with an effective tool to protect newborns from potentially life-threatening bacterial infections associated with GBS.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

Garuda Therapeutics is a biotech company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening diseases, particularly those affecting blood, bone marrow, immunity, and metabolism. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants by leveraging its innovative platform technology to generate self-renewing blood stem cells. This approach is designed to ensure rapid and broad access to consistent, durable, HLA-compatible, and transgene-free blood stem cell therapies for patients. Garuda Therapeutics employs advanced methodologies, including the use of zebrafish, mice, and human-induced pluripotent stem cells, to clone and analyze novel genes and mechanisms critical for blood formation, ultimately developing mechanism-based therapies that address unmet medical needs.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

iECURE is a biotechnology company focused on developing gene editing therapies aimed at treating rare and life-threatening liver disorders, particularly in children. The company employs mutation-agnostic in vivo gene insertion techniques, which allow for targeted therapeutic interventions without being limited by specific genetic mutations. Its initial programs concentrate on addressing metabolic "loss of function" disorders, which often have significant unmet medical needs. Through its innovative approach, iECURE aims to provide effective treatments for devastating diseases that currently lack viable options.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

Frontera Therapeutics is a company specializing in adeno-associated virus (AAV) gene therapy, focused on developing and commercializing cost-effective recombinant virus-based therapies. The company aims to create a scalable platform that produces high-quality, affordable rAAV therapies for a range of genetic disorders, including ophthalmic, liver, metabolic, and neuromuscular diseases. Frontera Therapeutics has a diverse product pipeline that encompasses multiple therapeutic areas, with various candidates at different stages of development. By prioritizing affordability and quality, the company seeks to enhance the lives of patients through innovative genetic therapies.

Aspen Neuroscience, Inc. is a biotechnology company focused on developing induced pluripotent stem cells (iPSCs) to create patient-specific therapies for neurological conditions, particularly Parkinson's disease. Founded in 2018 and based in San Diego, California, the company is advancing its pipeline of products, which includes ANPD001, an autologous neuron replacement therapy for sporadic Parkinson's disease, and ANPD002, a gene-edited autologous neuron replacement therapy targeting familial forms of the disease. Aspen Neuroscience employs innovative genomic approaches in conjunction with stem cell biology to address diseases with significant unmet medical needs, aiming to provide restorative cell therapies that can modify the progression of Parkinson's disease and potentially extend to other affected organs.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Cyrus Biotechnology, Inc. is a Seattle-based company that specializes in developing advanced software for protein structure prediction and design. Founded in 2014, it offers several key products, including Rosetta, a tool designed to create biologically active proteins for various medical applications such as treating brain cancer and addressing Celiac Disease. The company also provides Cyrus Bench, an enterprise version of Rosetta that includes a comprehensive suite of bio-molecular computation tools. Additionally, Cyrus Biotechnology offers specialized services like Cyrus CryoEM, which focuses on structure refinement and model building for complex biomolecular structures. Its innovative software solutions cater to pharmaceutical companies, biotech firms, and startups engaged in discovering therapeutics and developing novel consumer products.

PierianDx, Inc. is a biotechnology company that specializes in developing a cloud-based genomic software solution aimed at managing, analyzing, and interpreting complex genomic tests for clinical laboratories in the United States. Founded in 2014 and headquartered in Saint Louis, Missouri, PierianDx offers its Clinical Genomicist Workstation, which streamlines the workflow for patient cases, encompassing aspects from case accessioning to final report generation and integration with electronic health records. The company's software employs artificial intelligence to enhance the efficiency of genomic sequencing processes, thereby empowering healthcare providers to better diagnose and treat patients with cancer and other complex diseases. PierianDx also provides consulting and technical services, and collaborates strategically with organizations like RTI International. Through its accredited laboratory and extensive knowledgebase, the company supports health systems, children's hospitals, comprehensive cancer centers, and commercial laboratories in delivering integrated genomic care.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Garuda Therapeutics is a biotech company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening diseases, particularly those affecting blood, bone marrow, immunity, and metabolism. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants by leveraging its innovative platform technology to generate self-renewing blood stem cells. This approach is designed to ensure rapid and broad access to consistent, durable, HLA-compatible, and transgene-free blood stem cell therapies for patients. Garuda Therapeutics employs advanced methodologies, including the use of zebrafish, mice, and human-induced pluripotent stem cells, to clone and analyze novel genes and mechanisms critical for blood formation, ultimately developing mechanism-based therapies that address unmet medical needs.

iECURE is a biotechnology company focused on developing gene editing therapies aimed at treating rare and life-threatening liver disorders, particularly in children. The company employs mutation-agnostic in vivo gene insertion techniques, which allow for targeted therapeutic interventions without being limited by specific genetic mutations. Its initial programs concentrate on addressing metabolic "loss of function" disorders, which often have significant unmet medical needs. Through its innovative approach, iECURE aims to provide effective treatments for devastating diseases that currently lack viable options.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs technologies. This innovative approach is designed to restore immune tolerance and address limitations present in current regulatory T-cell therapies. By targeting the underlying causes of diseases related to immune system dysfunction, GentiBio aims to provide effective therapeutic solutions for conditions that arise from immune system overshooting or malfunctioning. The company was co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of this emerging field.

Splisense is develops antisense oligonucleotide based therapies to target genetic diseases caused by splicing mutations. The company has developed a compound to cure the lung disease in cystic fibrosis patients carrying a specific splicing mutation. It is also developing a targeted modulation approach to correct the splicing pattern of CFTR transcripts according to the specific mutation carried.

Gennao Bio is a privately held company focused on developing innovative targeted nucleic acid therapeutics, primarily aimed at treating oncology and rare monogenic skeletal muscle diseases. The company utilizes its proprietary gene monoclonal antibody (GMAB) platform technology, which features a novel, cell-penetrating antibody that facilitates the non-covalent binding and delivery of various nucleic acid payloads to specific cells. This non-viral delivery system stands out from traditional gene therapy methods by allowing for multiple types of nucleic acids, enabling repeat dosing, and employing established manufacturing processes. Gennao Bio's approach aims to address significant unmet medical needs and improve patient outcomes in the relevant therapeutic areas.

Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics based on CRISPR technology to address genetic disorders. Founded in 2017 and headquartered in Berkeley, California, the company utilizes its proprietary X-Editing (XE) platform, which features highly engineered CRISPR enzymes designed to improve the efficacy, specificity, and deliverability of genome editing. Scribe Therapeutics aims to overcome existing limitations in genetic medicine by creating custom-engineered enzymes and advanced delivery systems. Their approach seeks to establish CRISPR-based therapies as a new standard in clinical care, potentially transforming the treatment landscape for various conditions, including immuno-oncology, degenerative disorders, and enzyme replacement therapy. By prioritizing therapeutic precision and allele-specific targeting, Scribe Therapeutics is committed to expanding access to groundbreaking treatments that can significantly impact patient lives.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that specializes in targeted DNA integration to develop innovative therapies for severe diseases. The company utilizes a precision gene editing approach to precisely correct genetic mutations, replace malfunctioning genes, and insert therapeutic genetic sequences at specific locations within the genome. Its technology aims to transform treatment paradigms by enabling the targeted integration of genetic payloads, which could potentially cure a range of serious and life-threatening conditions. Graphite Bio was formerly known as Integral Medicines, Inc. before rebranding in August 2020, and it was incorporated in 2019.

Century Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies derived from induced pluripotent stem cells (iPSCs) to treat hematologic and solid malignancies. Founded in 2018 and headquartered in Philadelphia, Pennsylvania, the company utilizes a specialized platform that integrates gene editing and protein engineering to create allogeneic therapies. This platform allows for the generation of master cell banks of modified immune effector cells, specifically engineered to optimize performance and reduce the risk of rejection by the host immune system. By employing advanced technologies such as CRISPR-mediated gene editing and proprietary chimeric antigen receptors, Century Therapeutics aims to address significant unmet medical needs in cancer and other diseases.

AnchorDx, also known as Guangzhou Baseline Medical, specializes in high-throughput sequencing technologies and molecular liquid biopsies, offering non-invasive access to genetic information. The company focuses on precision medicine and aims to be a leading global provider of medical solutions by leveraging its expertise in cancer genomics, genetics, and bioinformatics. With a core team possessing over 20 years of industry experience, AnchorDx collaborates with various downstream enterprises, hospitals, and research institutions to create an integrated industrial chain that facilitates the transformation of academic research into clinical products. The company is committed to developing independent intellectual property rights and competitive clinical testing products, thereby providing accurate and comprehensive services to patients, doctors, and medical institutions.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

Ikena Oncology, Inc. is a biotechnology company focused on discovering and developing biomarker-driven therapies for cancer treatment. It specializes in precision oncology by targeting pathways essential for cancer growth and therapeutic resistance, particularly within the Hippo and RAS signaling networks. The company's portfolio includes several preclinical and discovery-stage programs, such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-412, a kynurenine-degrading enzyme. Additionally, Ikena is developing IK-930, an oral small-molecule inhibitor of the TEAD transcription factor involved in the Hippo pathway. Ikena Oncology, originally founded as Kyn Therapeutics in 2016, rebranded in December 2019 and is headquartered in Boston, Massachusetts. The company aims to address significant medical needs in oncology through innovative drug development.

Syros Pharmaceuticals, Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead product candidates, including SY-1425, a selective retinoic acid receptor alpha agonist currently in Phase II clinical trials for specific patient populations with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor under investigation in a Phase I clinical trial for advanced solid tumors. Syros is also engaged in collaborative research, including a partnership with Incyte Corporation aimed at identifying therapeutic targets for myeloproliferative neoplasms and a licensing agreement with TMRC Co. Ltd. for the development of tamibarotene. With a focus on mapping gene regulatory circuits, Syros leverages its proprietary platform and expertise to address significant unmet medical needs in oncology. Founded in 2011, the company aims to establish new standards of care for patients with hematologic malignancies.

Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing therapies aimed at protecting, repairing, and restoring hearing. The company focuses on a range of hearing disorders, including those related to cancer treatments, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as tinnitus. It utilizes advanced techniques such as single-cell genomics, bioinformatics, and precision gene therapy technologies to enhance its understanding of inner ear pathology and to develop effective treatments. Decibel Therapeutics also offers expertise in drug delivery to the inner ear, imaging, and pharmacokinetic modeling, contributing to innovative therapeutic approaches for patients experiencing various forms of hearing loss. Founded in 2013 and based in Boston, Massachusetts, Decibel Therapeutics was previously known as Hearing, Inc. before rebranding in 2014.

Novellus is a biotechnology company working to develop engineered cellular medicines. The company uses its non-immunogenic mRNA molecules to reprogram and gene-edit cells safely, and with high efficiency. They engineer precision medicines for patient segments and select from its technology toolbox for in vivo, ex vivo, allogeneic and autologous delivery. Novellus platform enables the high-level expression of gene-editing and cell-reprogramming proteins for high-specificity gene repair and both autologous and allogeneic cell therapies. The company is advancing its lead program, a breakthrough therapy for a rare dermatologic disease, through pre-clinical studies. Novellus was founded in 2015 and is headquartered in Cambridge, Massachusetts, United States.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

ESCAPE Bio is a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs technologies. This innovative approach is designed to restore immune tolerance and address limitations present in current regulatory T-cell therapies. By targeting the underlying causes of diseases related to immune system dysfunction, GentiBio aims to provide effective therapeutic solutions for conditions that arise from immune system overshooting or malfunctioning. The company was co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of this emerging field.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

Aspen Neuroscience, Inc. is a biotechnology company focused on developing induced pluripotent stem cells (iPSCs) to create patient-specific therapies for neurological conditions, particularly Parkinson's disease. Founded in 2018 and based in San Diego, California, the company is advancing its pipeline of products, which includes ANPD001, an autologous neuron replacement therapy for sporadic Parkinson's disease, and ANPD002, a gene-edited autologous neuron replacement therapy targeting familial forms of the disease. Aspen Neuroscience employs innovative genomic approaches in conjunction with stem cell biology to address diseases with significant unmet medical needs, aiming to provide restorative cell therapies that can modify the progression of Parkinson's disease and potentially extend to other affected organs.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

EmendoBio, Inc. is focused on advancing the field of genome editing by developing innovative gene editing tools aimed at overcoming challenges in gene therapy. The company leverages expertise in protein engineering and DNA repair, combining insights from top academic institutions to create genetic medicines that address currently untreatable disorders and diseases. By transforming existing methodologies and employing creative approaches, EmendoBio seeks to resolve significant bottlenecks in gene therapy, enabling healthcare professionals to provide effective treatments where none previously existed. Through a commitment to pushing scientific boundaries, EmendoBio strives to deliver on the potential of gene editing for transformative medical solutions.

Aspen Neuroscience, Inc. is a biotechnology company focused on developing induced pluripotent stem cells (iPSCs) to create patient-specific therapies for neurological conditions, particularly Parkinson's disease. Founded in 2018 and based in San Diego, California, the company is advancing its pipeline of products, which includes ANPD001, an autologous neuron replacement therapy for sporadic Parkinson's disease, and ANPD002, a gene-edited autologous neuron replacement therapy targeting familial forms of the disease. Aspen Neuroscience employs innovative genomic approaches in conjunction with stem cell biology to address diseases with significant unmet medical needs, aiming to provide restorative cell therapies that can modify the progression of Parkinson's disease and potentially extend to other affected organs.

Repare Therapeutics Inc. is a precision oncology company based in Montréal, Canada, specializing in the discovery and development of novel therapeutics through a synthetic lethality approach. Utilizing its proprietary SNIPRx platform, which combines genome-wide and CRISPR-enabled technology, the company systematically identifies targeted cancer therapies addressing genomic instability, particularly in DNA damage repair. Its lead candidate, RP-3500, is an oral small molecule inhibitor designed to treat solid tumors with specific DNA damage repair-related genomic alterations. Additionally, Repare is advancing a CCNE1-SL inhibitor program targeting tumors with CCNE1 amplification, along with a Polymerase Theta program focused on BRCA mutations and related genomic alterations. Founded in 2016, the company aims to improve cancer treatment by leveraging innovative drug discovery methods and addressing the unique vulnerabilities of tumor cells.

Passage Bio is a genetic medicines company based in Philadelphia, Pennsylvania, specializing in the development of therapies for rare monogenic central nervous system (CNS) diseases. Founded in 2017, the company aims to create a portfolio of five AAV-delivered therapeutics, targeting conditions such as GM1 Gangliosidosis, Frontotemporal dementia, and Krabbe Disease. Passage Bio collaborates closely with the University of Pennsylvania, leveraging its Gene Therapy Program for preclinical work while managing clinical development, regulatory affairs, manufacturing, and commercialization of its product candidates. The company also engages with the Orphan Disease Center for natural history studies, key opinion leader involvement, and patient advocacy.

Prevail Therapeutics Inc. is a biotechnology company headquartered in New York, specializing in the development and commercialization of gene therapies targeting neurodegenerative diseases. Founded in 2017, the company focuses on adeno-associated virus (AAV)-based therapies, with its lead candidate, PR001, currently undergoing Phase I/II clinical trials for Parkinson's disease associated with GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is advancing PR006 for frontotemporal dementia linked to GRN mutations and PR004 for various synucleinopathies. The company's mission is to provide disease-modifying treatments for patients suffering from these challenging conditions.

Passage Bio is a genetic medicines company based in Philadelphia, Pennsylvania, specializing in the development of therapies for rare monogenic central nervous system (CNS) diseases. Founded in 2017, the company aims to create a portfolio of five AAV-delivered therapeutics, targeting conditions such as GM1 Gangliosidosis, Frontotemporal dementia, and Krabbe Disease. Passage Bio collaborates closely with the University of Pennsylvania, leveraging its Gene Therapy Program for preclinical work while managing clinical development, regulatory affairs, manufacturing, and commercialization of its product candidates. The company also engages with the Orphan Disease Center for natural history studies, key opinion leader involvement, and patient advocacy.

Modis Therapeutics, Inc. is a biopharmaceutical company focused on developing disease-modifying therapies for rare genetic diseases with significant unmet medical needs. The company, based in Oakland, California, was incorporated in 2016 and is a subsidiary of Zogenix, Inc. Modis Therapeutics is advancing its lead therapy, MT1621, which aims to restore mitochondrial DNA replication fidelity. This therapeutic candidate is specifically designed to address thymidine kinase 2 deficiency (TK2d), an inherited mitochondrial disorder. By improving nucleotide balance, increasing mitochondrial DNA copy number, and enhancing cell function, Modis Therapeutics aims to provide meaningful treatment options that can prolong the lives of affected patients.

Therachon is a biotechnology company specializing in the development of treatments for rare genetic diseases that lack available therapies. Primarily based in Basel, Switzerland, with research labs in Nice, France, the company is advancing its lead candidate, TA-46, a novel protein therapy aimed at treating achondroplasia, the most prevalent form of short-limbed dwarfism. In addition to its focus on achondroplasia, Therachon is also committed to developing therapeutics targeting rare gastrointestinal and musculoskeletal disorders, with the goal of enabling medical professionals to effectively manage conditions such as short bowel syndrome.

Alector, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapies for neurodegenerative diseases, including Alzheimer’s disease and frontotemporal dementia. Founded in 2013 and headquartered in South San Francisco, California, the company utilizes advanced antibody technology and insights from neuroimmunology and human genetics to create innovative treatments. Alector’s pipeline includes several candidates, such as AL001, which is undergoing phase II clinical trials for frontotemporal dementia, and AL101, currently in phase I trials for Alzheimer's and Parkinson's diseases. Additionally, AL002 and AL003 are in phase 1b trials for Alzheimer’s disease. The company is actively engaged in research and development, with a total of ten programs at various stages. Alector has formed a strategic collaboration with Adimab, LLC to enhance its antibody research and development capabilities, aiming to efficiently generate and validate antibody drugs that target key disease-related mechanisms.

Constellation Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for cancers linked to abnormal gene expression and drug resistance. The company specializes in the field of epigenetics, targeting selective regulators of epigenetic function that are vital for gene expression control. Its lead product candidates include CPI-0610, currently in Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is undergoing Phase Ib/II clinical trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is advancing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial aimed at treating solid tumors. Founded in 2008 and based in Cambridge, Massachusetts, the company was previously known as Epigenetix, Inc. before rebranding in March 2008.

Prevail Therapeutics Inc. is a biotechnology company headquartered in New York, specializing in the development and commercialization of gene therapies targeting neurodegenerative diseases. Founded in 2017, the company focuses on adeno-associated virus (AAV)-based therapies, with its lead candidate, PR001, currently undergoing Phase I/II clinical trials for Parkinson's disease associated with GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is advancing PR006 for frontotemporal dementia linked to GRN mutations and PR004 for various synucleinopathies. The company's mission is to provide disease-modifying treatments for patients suffering from these challenging conditions.

Ikena Oncology, Inc. is a biotechnology company focused on discovering and developing biomarker-driven therapies for cancer treatment. It specializes in precision oncology by targeting pathways essential for cancer growth and therapeutic resistance, particularly within the Hippo and RAS signaling networks. The company's portfolio includes several preclinical and discovery-stage programs, such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-412, a kynurenine-degrading enzyme. Additionally, Ikena is developing IK-930, an oral small-molecule inhibitor of the TEAD transcription factor involved in the Hippo pathway. Ikena Oncology, originally founded as Kyn Therapeutics in 2016, rebranded in December 2019 and is headquartered in Boston, Massachusetts. The company aims to address significant medical needs in oncology through innovative drug development.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

Cyrus Biotechnology, Inc. is a Seattle-based company that specializes in developing advanced software for protein structure prediction and design. Founded in 2014, it offers several key products, including Rosetta, a tool designed to create biologically active proteins for various medical applications such as treating brain cancer and addressing Celiac Disease. The company also provides Cyrus Bench, an enterprise version of Rosetta that includes a comprehensive suite of bio-molecular computation tools. Additionally, Cyrus Biotechnology offers specialized services like Cyrus CryoEM, which focuses on structure refinement and model building for complex biomolecular structures. Its innovative software solutions cater to pharmaceutical companies, biotech firms, and startups engaged in discovering therapeutics and developing novel consumer products.

ESCAPE Bio is a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

LogicBio Therapeutics is a genome editing company dedicated to developing innovative treatments for rare diseases, particularly those affecting pediatric patients with significant unmet medical needs. Utilizing its proprietary GeneRide technology, LogicBio aims to integrate corrective genes into patients' genomes, offering potential therapeutic benefits. The company's lead product candidate, LB-001, targets Methylmalonic Acidemia, a severe condition that manifests at birth. Additionally, LogicBio is advancing other candidates, including LB-301, which is being developed in collaboration with Takeda Pharmaceutical Company for Crigler-Najjar syndrome. The company also partners with the Children's Medical Research Institute to create new viral vectors. Founded in 2014 and based in Lexington, Massachusetts, LogicBio is focused on delivering safe and effective genetic medicine to patients suffering from previously untreatable genetic and infectious diseases.

Mitoconix Bio Ltd, founded in 2016 and based in Jerusalem, Israel, is focused on developing innovative therapies aimed at enhancing mitochondrial health to address neurodegenerative diseases. The company seeks to improve mitochondrial functions, which are crucial for energy production, cellular metabolism, and maintaining cellular balance. Mitochondria play a vital role in neuronal health through processes such as fission and fusion, which are essential for their motility and functionality. Disruptions in these processes can lead to decreased energy production, increased oxidative stress, and cell death. Mitoconix's lead drug is a pioneering inhibitor that targets pathological mitochondrial fragmentation and dysfunction, demonstrating efficacy in animal models of Huntington’s and Parkinson’s diseases, as well as beneficial effects in patient-derived cells associated with these and Alzheimer’s disease.

Therachon is a biotechnology company specializing in the development of treatments for rare genetic diseases that lack available therapies. Primarily based in Basel, Switzerland, with research labs in Nice, France, the company is advancing its lead candidate, TA-46, a novel protein therapy aimed at treating achondroplasia, the most prevalent form of short-limbed dwarfism. In addition to its focus on achondroplasia, Therachon is also committed to developing therapeutics targeting rare gastrointestinal and musculoskeletal disorders, with the goal of enabling medical professionals to effectively manage conditions such as short bowel syndrome.

EmendoBio, Inc. is focused on advancing the field of genome editing by developing innovative gene editing tools aimed at overcoming challenges in gene therapy. The company leverages expertise in protein engineering and DNA repair, combining insights from top academic institutions to create genetic medicines that address currently untreatable disorders and diseases. By transforming existing methodologies and employing creative approaches, EmendoBio seeks to resolve significant bottlenecks in gene therapy, enabling healthcare professionals to provide effective treatments where none previously existed. Through a commitment to pushing scientific boundaries, EmendoBio strives to deliver on the potential of gene editing for transformative medical solutions.

Arrowhead Pharmaceuticals, Inc. is a biotechnology company based in Pasadena, California, focused on developing innovative medicines for the treatment of intractable diseases, particularly those with a genetic origin characterized by the overproduction of specific proteins. The company's diverse pipeline includes RNA interference (RNAi) therapeutics such as ARO-AAT for liver diseases associated with alpha-1 antitrypsin deficiency, ARO-APOC3 for hypertriglyceridemia, and ARO-HIF2 for clear cell renal cell carcinoma, among others. Arrowhead is also exploring treatments for various conditions, including chronic hepatitis B virus infection and cardiovascular diseases. The company collaborates with prominent pharmaceutical firms, including Janssen Pharmaceuticals and Takeda Pharmaceuticals, to develop additional RNAi therapeutic candidates. Originally founded as Arrowhead Research Corporation in 1989, it adopted its current name in April 2016.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

LogicBio Therapeutics is a genome editing company dedicated to developing innovative treatments for rare diseases, particularly those affecting pediatric patients with significant unmet medical needs. Utilizing its proprietary GeneRide technology, LogicBio aims to integrate corrective genes into patients' genomes, offering potential therapeutic benefits. The company's lead product candidate, LB-001, targets Methylmalonic Acidemia, a severe condition that manifests at birth. Additionally, LogicBio is advancing other candidates, including LB-301, which is being developed in collaboration with Takeda Pharmaceutical Company for Crigler-Najjar syndrome. The company also partners with the Children's Medical Research Institute to create new viral vectors. Founded in 2014 and based in Lexington, Massachusetts, LogicBio is focused on delivering safe and effective genetic medicine to patients suffering from previously untreatable genetic and infectious diseases.

Alector, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapies for neurodegenerative diseases, including Alzheimer’s disease and frontotemporal dementia. Founded in 2013 and headquartered in South San Francisco, California, the company utilizes advanced antibody technology and insights from neuroimmunology and human genetics to create innovative treatments. Alector’s pipeline includes several candidates, such as AL001, which is undergoing phase II clinical trials for frontotemporal dementia, and AL101, currently in phase I trials for Alzheimer's and Parkinson's diseases. Additionally, AL002 and AL003 are in phase 1b trials for Alzheimer’s disease. The company is actively engaged in research and development, with a total of ten programs at various stages. Alector has formed a strategic collaboration with Adimab, LLC to enhance its antibody research and development capabilities, aiming to efficiently generate and validate antibody drugs that target key disease-related mechanisms.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

Therachon is a biotechnology company specializing in the development of treatments for rare genetic diseases that lack available therapies. Primarily based in Basel, Switzerland, with research labs in Nice, France, the company is advancing its lead candidate, TA-46, a novel protein therapy aimed at treating achondroplasia, the most prevalent form of short-limbed dwarfism. In addition to its focus on achondroplasia, Therachon is also committed to developing therapeutics targeting rare gastrointestinal and musculoskeletal disorders, with the goal of enabling medical professionals to effectively manage conditions such as short bowel syndrome.

Alector, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapies for neurodegenerative diseases, including Alzheimer’s disease and frontotemporal dementia. Founded in 2013 and headquartered in South San Francisco, California, the company utilizes advanced antibody technology and insights from neuroimmunology and human genetics to create innovative treatments. Alector’s pipeline includes several candidates, such as AL001, which is undergoing phase II clinical trials for frontotemporal dementia, and AL101, currently in phase I trials for Alzheimer's and Parkinson's diseases. Additionally, AL002 and AL003 are in phase 1b trials for Alzheimer’s disease. The company is actively engaged in research and development, with a total of ten programs at various stages. Alector has formed a strategic collaboration with Adimab, LLC to enhance its antibody research and development capabilities, aiming to efficiently generate and validate antibody drugs that target key disease-related mechanisms.

Intellia Therapeutics, Inc. is a genome editing company headquartered in Cambridge, Massachusetts, specializing in the development of therapeutics using the CRISPR/Cas9 technology. This innovative platform allows for precise alterations of genomic DNA, enabling the company to pursue treatments for a range of severe and life-threatening diseases. Intellia's pipeline includes both in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as ex vivo programs focused on engineered cell therapies for oncological and autoimmune disorders. The company is also exploring treatments for conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Intellia has established collaborations with several organizations, enhancing its research and development capabilities. Founded in 2014, Intellia holds a significant intellectual property portfolio related to the therapeutic applications of CRISPR/Cas9, aiming to address high unmet medical needs through its advanced gene-editing approaches.

Rhythm Metabolic is the subsidiary of Rhythm Pharmaceuticals that develops setmelanotide (RM-493), a novel peptide MC4 agonist for the treatment of obesity caused by genetic deficiencies in the MC4 pathway. It was established in 2013 and is headquartered in Boston, Massachusetts.

Dimension Therapeutics, Inc. is a gene therapy company dedicated to developing innovative treatments for rare and metabolic diseases linked to liver function. Founded in 2013 and based in Cambridge, Massachusetts, the company has established a robust pipeline of gene therapy candidates. Its lead products include DTX101 for hemophilia B and DTX201 for hemophilia A, alongside other programs targeting conditions such as ornithine transcarbamylase deficiency, glycogen storage disease type Ia, phenylketonuria, Wilson disease, and citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, leveraging its extensive patent portfolio related to NAV vector technology. This partnership enhances Dimension's ability to advance its gene therapy initiatives, particularly in the realm of adeno-associated virus therapeutics. In 2017, Dimension Therapeutics became a subsidiary of Ultragenyx Pharmaceutical Inc.

Natera, Inc. is a diagnostic and research company specializing in genetic testing services, particularly in the areas of preconception and prenatal diagnostics. Founded in 2003 and headquartered in San Carlos, California, Natera offers a range of products including the Panorama non-invasive prenatal test, which screens for chromosomal abnormalities in fetuses, and Vistara, a test for single-gene mutations. Additionally, the company provides Horizon carrier screening to assess carrier status for various genetic diseases, and Spectrum, which analyzes genetic conditions during in vitro fertilization. Natera's Anora product analyzes fetal chromosomes to understand miscarriage causes, while its non-invasive paternity tests determine parentage through fetal DNA analysis. The company's Signatera technology focuses on circulating tumor DNA to monitor cancer recurrence. Natera distributes its products through a direct sales force and a network of approximately 100 laboratory and distribution partners, both domestically and internationally. The company collaborates with BGI Genomics to develop genetic testing assays and has partnered with Foundation Medicine for personalized cancer monitoring assays.

GENEWIZ, Inc. is a global contract research organization specializing in genomics and DNA sequencing services. Founded in 1999 and headquartered in South Plainfield, New Jersey, the company serves a diverse clientele, including pharmaceutical, biotechnology, academic, agricultural, clinical, and government institutions. GENEWIZ offers a comprehensive range of services, including Sanger DNA sequencing, gene synthesis, molecular biology techniques, genomic mutation analysis, and bioinformatics, all designed to enhance research productivity and efficiency. The company is also equipped to provide regulatory-compliant laboratory services for FDA applications, including RNA/DNA identity studies and SNP testing. With a focus on delivering high-quality data and exceptional technical support, GENEWIZ is committed to accelerating scientific discovery and advancing translational medicine. The company operates multiple locations across the United States and internationally in China, the United Kingdom, France, Germany, and Japan, solidifying its position as a preferred partner in the life sciences sector. As of November 2018, GENEWIZ operates as a subsidiary of Brooks Automation, Inc.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

Invitae is a genetic information company focused on integrating genetic insights into everyday medical practice to enhance healthcare quality for a large population. The company specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates multiple genetic tests into a single platform. Invitae's offerings include assessments for genes linked to hereditary cancer, neurological disorders, cardiovascular conditions, pediatric issues, metabolic disorders, and other hereditary ailments. By providing high-quality testing with quicker turnaround times and competitive pricing, Invitae aims to make genetic testing more accessible and effective for patients and healthcare providers alike.

Dimension Therapeutics, Inc. is a gene therapy company dedicated to developing innovative treatments for rare and metabolic diseases linked to liver function. Founded in 2013 and based in Cambridge, Massachusetts, the company has established a robust pipeline of gene therapy candidates. Its lead products include DTX101 for hemophilia B and DTX201 for hemophilia A, alongside other programs targeting conditions such as ornithine transcarbamylase deficiency, glycogen storage disease type Ia, phenylketonuria, Wilson disease, and citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, leveraging its extensive patent portfolio related to NAV vector technology. This partnership enhances Dimension's ability to advance its gene therapy initiatives, particularly in the realm of adeno-associated virus therapeutics. In 2017, Dimension Therapeutics became a subsidiary of Ultragenyx Pharmaceutical Inc.

Loxo Oncology, Inc. is a biopharmaceutical company based in Stamford, Connecticut, focused on developing and commercializing targeted therapies for genetically defined cancers in the United States. Founded in 2013, the company aims to bring innovative cancer treatments to the clinic by identifying actionable "driver mutations" in tumors. Its lead product candidate, larotrectinib, is an oral selective inhibitor of tropomyosin receptor kinase (TRK) and is undergoing various clinical trials for multiple cancer types, including lung, melanoma, and breast cancer. Loxo Oncology is also advancing several preclinical programs, including candidates targeting RET fusion proteins and FGFR isoforms. The company has established collaborations with several organizations to enhance its drug discovery efforts and optimize its clinical development strategies. As a subsidiary of Eli Lilly and Company, Loxo Oncology is dedicated to creating effective therapies that address specific genetic vulnerabilities in cancer patients.

Igenica, Inc. is a biotherapeutic company based in Burlingame, California, dedicated to the discovery and development of antibodies and antibody-drug conjugates (ADCs) for cancer treatment. Founded in 2008, Igenica specializes in immunotherapies and functional antibodies designed to target tumors effectively. The company's notable products include IGN523, a humanized monoclonal antibody aimed at treating various hematologic and solid tumors; IGN786, an ADC; and IGN381, which targets immune checkpoints that complement existing therapies in the tumor microenvironment. Igenica has established a strategic oncology research agreement with MedImmune and is supported by a group of prominent life science investors, including The Column Group and OrbiMed. The company operates as a subsidiary of Pierre Fabre Medicament SA, with a leadership team that has significant experience in antibody drug discovery and commercialization.

Alector, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapies for neurodegenerative diseases, including Alzheimer’s disease and frontotemporal dementia. Founded in 2013 and headquartered in South San Francisco, California, the company utilizes advanced antibody technology and insights from neuroimmunology and human genetics to create innovative treatments. Alector’s pipeline includes several candidates, such as AL001, which is undergoing phase II clinical trials for frontotemporal dementia, and AL101, currently in phase I trials for Alzheimer's and Parkinson's diseases. Additionally, AL002 and AL003 are in phase 1b trials for Alzheimer’s disease. The company is actively engaged in research and development, with a total of ten programs at various stages. Alector has formed a strategic collaboration with Adimab, LLC to enhance its antibody research and development capabilities, aiming to efficiently generate and validate antibody drugs that target key disease-related mechanisms.

Loxo Oncology, Inc. is a biopharmaceutical company based in Stamford, Connecticut, focused on developing and commercializing targeted therapies for genetically defined cancers in the United States. Founded in 2013, the company aims to bring innovative cancer treatments to the clinic by identifying actionable "driver mutations" in tumors. Its lead product candidate, larotrectinib, is an oral selective inhibitor of tropomyosin receptor kinase (TRK) and is undergoing various clinical trials for multiple cancer types, including lung, melanoma, and breast cancer. Loxo Oncology is also advancing several preclinical programs, including candidates targeting RET fusion proteins and FGFR isoforms. The company has established collaborations with several organizations to enhance its drug discovery efforts and optimize its clinical development strategies. As a subsidiary of Eli Lilly and Company, Loxo Oncology is dedicated to creating effective therapies that address specific genetic vulnerabilities in cancer patients.

Amoy Diagnostics is a research and development based diagnostic company focusing on molecular diagnostics for oncology precision medicine. AmoyDx has a portfolio of molecular diagnostic assays. Based on multiple technology platforms including PCR, NGS, FISH and IHC, AmoyDx's core product categories include single gene testing kits, multi-gene testing panels, NGS panels, and NGS analytical systems.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

Natera, Inc. is a diagnostic and research company specializing in genetic testing services, particularly in the areas of preconception and prenatal diagnostics. Founded in 2003 and headquartered in San Carlos, California, Natera offers a range of products including the Panorama non-invasive prenatal test, which screens for chromosomal abnormalities in fetuses, and Vistara, a test for single-gene mutations. Additionally, the company provides Horizon carrier screening to assess carrier status for various genetic diseases, and Spectrum, which analyzes genetic conditions during in vitro fertilization. Natera's Anora product analyzes fetal chromosomes to understand miscarriage causes, while its non-invasive paternity tests determine parentage through fetal DNA analysis. The company's Signatera technology focuses on circulating tumor DNA to monitor cancer recurrence. Natera distributes its products through a direct sales force and a network of approximately 100 laboratory and distribution partners, both domestically and internationally. The company collaborates with BGI Genomics to develop genetic testing assays and has partnered with Foundation Medicine for personalized cancer monitoring assays.

Igenica, Inc. is a biotherapeutic company based in Burlingame, California, dedicated to the discovery and development of antibodies and antibody-drug conjugates (ADCs) for cancer treatment. Founded in 2008, Igenica specializes in immunotherapies and functional antibodies designed to target tumors effectively. The company's notable products include IGN523, a humanized monoclonal antibody aimed at treating various hematologic and solid tumors; IGN786, an ADC; and IGN381, which targets immune checkpoints that complement existing therapies in the tumor microenvironment. Igenica has established a strategic oncology research agreement with MedImmune and is supported by a group of prominent life science investors, including The Column Group and OrbiMed. The company operates as a subsidiary of Pierre Fabre Medicament SA, with a leadership team that has significant experience in antibody drug discovery and commercialization.

Good Start Genetics, Inc. is a molecular diagnostics company based in Cambridge, Massachusetts, focused on genetic screening for inherited diseases. Founded in 2007, the company aims to transform reproductive medicine by providing physicians and patients with actionable insights into inherited genetic disorders. Good Start Genetics offers a range of services, including EmbryVu, a preimplantation genetic screening test designed to enhance in vitro fertilization outcomes by identifying suitable embryos for implantation. The company employs advanced next-generation DNA sequencing technology, along with other established genetic screening methods, to deliver high carrier detection rates. Additionally, through its GoodStart Select program, it provides a comprehensive menu of genetic carrier screening tests for both known and novel mutations, all supported by a commitment to customer care and genetic counseling. As of August 2017, Good Start Genetics operates as a subsidiary of Invitae Corporation.

Crown Bioscience International, through its subsidiary Crown Bioscience Inc., is a global company specializing in drug discovery and development solutions. Founded in 2006 and based in Taipei, Taiwan, the company focuses on providing translational platforms for oncology, cardiovascular, and metabolic diseases, helping biopharmaceutical companies enhance drug efficacy and patient response while minimizing clinical failures. Crown Bioscience offers a comprehensive range of products, including antibodies for in vivo and in vitro studies, recombinant proteins, and tumor tissue microarrays, as well as integrated preclinical testing services. The firm has established strategic partnerships, including collaborations with the Shanghai Model Organisms Center and Hubrecht Organoid Technology, to advance its capabilities in genetically engineered models and organoid-based preclinical drug development. With facilities in the United States, United Kingdom, China, and Taiwan, Crown Bioscience serves the biotech and pharmaceutical industries worldwide, facilitating the acceleration of new drug development programs.

Good Start Genetics, Inc. is a molecular diagnostics company based in Cambridge, Massachusetts, focused on genetic screening for inherited diseases. Founded in 2007, the company aims to transform reproductive medicine by providing physicians and patients with actionable insights into inherited genetic disorders. Good Start Genetics offers a range of services, including EmbryVu, a preimplantation genetic screening test designed to enhance in vitro fertilization outcomes by identifying suitable embryos for implantation. The company employs advanced next-generation DNA sequencing technology, along with other established genetic screening methods, to deliver high carrier detection rates. Additionally, through its GoodStart Select program, it provides a comprehensive menu of genetic carrier screening tests for both known and novel mutations, all supported by a commitment to customer care and genetic counseling. As of August 2017, Good Start Genetics operates as a subsidiary of Invitae Corporation.

Complete Genomics specializes in high-quality, affordable DNA sequencing and analysis, facilitating large-scale research into the genetic mechanisms of drug responses and complex diseases. The company has developed a comprehensive genomics analysis platform that integrates proprietary human sequencing technology with advanced informatics and data management software. This approach ensures that customers receive ready-to-use data for genome-based research, supporting advancements in personalized medicine and broader genetic studies. By combining innovative technology with a unique market strategy, Complete Genomics aims to transform the landscape of DNA sequencing.

Igenica, Inc. is a biotherapeutic company based in Burlingame, California, dedicated to the discovery and development of antibodies and antibody-drug conjugates (ADCs) for cancer treatment. Founded in 2008, Igenica specializes in immunotherapies and functional antibodies designed to target tumors effectively. The company's notable products include IGN523, a humanized monoclonal antibody aimed at treating various hematologic and solid tumors; IGN786, an ADC; and IGN381, which targets immune checkpoints that complement existing therapies in the tumor microenvironment. Igenica has established a strategic oncology research agreement with MedImmune and is supported by a group of prominent life science investors, including The Column Group and OrbiMed. The company operates as a subsidiary of Pierre Fabre Medicament SA, with a leadership team that has significant experience in antibody drug discovery and commercialization.

Complete Genomics specializes in high-quality, affordable DNA sequencing and analysis, facilitating large-scale research into the genetic mechanisms of drug responses and complex diseases. The company has developed a comprehensive genomics analysis platform that integrates proprietary human sequencing technology with advanced informatics and data management software. This approach ensures that customers receive ready-to-use data for genome-based research, supporting advancements in personalized medicine and broader genetic studies. By combining innovative technology with a unique market strategy, Complete Genomics aims to transform the landscape of DNA sequencing.

Enobia Pharma focuses on developing therapies for serious genetic bone disorders that currently lack approved treatments. The company specifically targets conditions such as hypophosphatasia (HPP), a rare metabolic disorder that affects bone mineralization and can lead to severe skeletal deformities and other health complications. Through its innovative research and development efforts, Enobia aims to provide effective solutions for patients suffering from these debilitating conditions.

As of August 20, 2008, SGX Pharmaceuticals, Inc. was acquired by Eli Lilly & Co. SGX Pharmaceuticals, Inc., a biotechnology company, focuses on the discovery, development, and commercialization of therapeutics for addressing unmet medical needs in oncology. The company's product pipeline includes drug candidates from its FAST drug discovery platform, which uses X-ray crystallography and complementary biophysical and biochemical methods, combined with medicinal and computational chemistry. Its drug development programs target the c-MET receptor tyrosine kinase (MET), an enzyme implicated in an array of cancers; and the BCR-ABL tyrosine kinase enzyme for the treatment of chronic myelogenous leukemia (CML), a bone marrow cancer. Under the MET development program, the company develops SGX523, which is in Phase I clinical development studies for solid tumor indications; and SGX126, a preclinical development product for solid tumors indications. Under the BCR-ABL Development Program, SGX Pharmaceuticals develops SGX393, an oral therapy for the second-line treatment of CML, which is in preclinical development studies. Its drug discovery technologies are also applied to a portfolio of oncology targets, including JAK2, a non-receptor tyrosine kinase involved in cytokine-induced signaling and growth regulation, survival, and differentiation of cells; RAS, a protein that regulates cell growth; and three other tyrosine kinases. The company has a license and collaboration agreement with Novartis Institutes for Biomedical Research, Inc., Cystic Fibrosis Foundation Therapeutics, Inc., National Institutes of Health, and Eli Lilly & Company. SGX Pharmaceuticals, formerly known as Structural GenomiX, Inc., was founded in 1998 and is headquartered in San Diego, California.

Enobia Pharma focuses on developing therapies for serious genetic bone disorders that currently lack approved treatments. The company specifically targets conditions such as hypophosphatasia (HPP), a rare metabolic disorder that affects bone mineralization and can lead to severe skeletal deformities and other health complications. Through its innovative research and development efforts, Enobia aims to provide effective solutions for patients suffering from these debilitating conditions.