OrbiMed

Aadi Bioscience is a clinical-stage biopharmaceutical company developing precision therapies for cancers driven by mTOR pathway alterations. It focuses on targeted mTOR inhibitors to treat genetically defined cancers, including alterations in TSC1 or TSC2 genes, where conventional inhibitors have struggled with pharmacology, delivery, safety, or tumor targeting. The company is advancing ABI-009, a clinical-phase mTOR inhibitor intended for oncology indications as well as cardiovascular and metabolic contexts. Aadi aims to bring transformative therapies to patients with ultra-rare cancers such as PEComa, and it traces its origins to an earlier name, with its operations based in Pacific Palisades, California, and a founding year of 2011.

Aadi Bioscience is a clinical-stage biopharmaceutical company developing precision therapies for cancers driven by mTOR pathway alterations. It focuses on targeted mTOR inhibitors to treat genetically defined cancers, including alterations in TSC1 or TSC2 genes, where conventional inhibitors have struggled with pharmacology, delivery, safety, or tumor targeting. The company is advancing ABI-009, a clinical-phase mTOR inhibitor intended for oncology indications as well as cardiovascular and metabolic contexts. Aadi aims to bring transformative therapies to patients with ultra-rare cancers such as PEComa, and it traces its origins to an earlier name, with its operations based in Pacific Palisades, California, and a founding year of 2011.

Avenzo Therapeutics focuses on developing innovative cancer treatments. It operates a clinical-stage platform dedicated to creating novel therapies for underserved cancers, with a pipeline targeting rare and genomically defined cancers.

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.

Founded in 2020 and headquartered in Boston, Massachusetts, Scorpion Therapeutics is a clinical-stage biotechnology company specializing in precision oncology. It develops targeted small-molecule drugs to treat cancer by focusing on three areas: therapies against known oncogenes, agents for currently undruggable targets, and drugs for new targets.

Alterome Therapeutics is a precision oncology biotechnology company focused on developing alteration-specific therapeutics to target high-value, validated oncogenic drivers. It employs structure-guided drug discovery and advanced computational chemistry to create small-molecule therapies that selectively address oncogenic alterations, with the aim of improving patient outcomes in cancer.

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.

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 develops gene medicines to provide long-lasting treatments for a wide range of diseases. It employs proprietary development and delivery platforms to create optimized therapeutics in a revocable, non-viral format, advancing gene-based therapies with improved safety and controllability. The company integrates capabilities from gene delivery, protein sciences, and clinical translation to address genetically driven diseases and translate scientific advances into clinical options.

Adela specializes in developing innovative technologies for early detection of high-morbidity conditions like cancer through routine blood tests. Its proprietary platform efficiently captures and analyzes the methylome, focusing on highly informative regions to enhance diagnostic accuracy.

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 precision oncology company focused on developing targeted cancer therapies guided by functional genomics. It aims to create a pipeline of treatments for patients with currently unaddressed mutations across established oncology targets.

ADARx Pharmaceuticals specializes in the development of RNA editing therapeutics. Founded in 2019, the company focuses on discovering and developing innovative treatments that utilize endogenous enzymes to precisely target and correct single point mutations in mRNA transcripts, restoring the production of functional proteins.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative cell therapies for cancer treatment. Based in Shanghai, China, Gracell's lead product candidates include GC012F, a dual-targeting autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia. Both candidates are currently undergoing Phase I clinical trials. Additionally, the company is advancing GC007F for B cell non-Hodgkin's lymphoma and GC027 for adult T cell acute lymphoblastic leukemia, among others. Gracell also has a range of earlier-stage product candidates addressing various cancer types, such as ovarian cancer and breast cancer. Founded in 2017, the company aims to provide effective cellular therapeutics to improve outcomes for patients with hematological malignancies and solid tumors.

ADARx Pharmaceuticals specializes in the development of RNA editing therapeutics. Founded in 2019, the company focuses on discovering and developing innovative treatments that utilize endogenous enzymes to precisely target and correct single point mutations in mRNA transcripts, restoring the production of functional proteins.

Caris Life Sciences specializes in advanced molecular profiling technologies for cancer diagnostics and drug development. Established in 1987, it offers prognostic testing services, genomic profiling, and clinico-genomic data platforms to improve patient outcomes. The company operates globally with offices in the US and Switzerland.

iECURE is a gene editing company developing mutation-agnostic in vivo gene insertion therapies to treat rare, life-threatening liver disorders in children. Its approach seeks long-term, potentially curative treatment for metabolic liver diseases caused by loss-of-function mutations, with initial programs targeting ornithine transcarbamylase deficiency, citrullinemia type 1, and phenylketonuria.

Alterome Therapeutics is a precision oncology biotechnology company focused on developing alteration-specific therapeutics to target high-value, validated oncogenic drivers. It employs structure-guided drug discovery and advanced computational chemistry to create small-molecule therapies that selectively address oncogenic alterations, with the aim of improving patient outcomes in cancer.

PIC Therapeutics, Inc. is a biotechnology company dedicated to advancing cancer treatment through the selective modulation of oncogene translation. Founded in 2016 and headquartered in Boston, Massachusetts, the company develops precision-based therapeutics that target the master switch of cancer signaling pathways. By modulating the pre-initiation complex responsible for messenger RNA translation, PIC Therapeutics aims to selectively block the production of oncogene proteins. This innovative approach has the potential to simultaneously address multiple oncogenic drivers, paving the way for a new generation of cancer therapies that could significantly enhance patient outcomes.

Treeline Biosciences is a biotechnology company focused on developing precision medicines for cancer and other serious diseases. Founded in 2021 and based in Stamford, Connecticut, the company uses a platform that integrates pathophysiology, genomics, and pattern recognition to identify therapeutic approaches that address unmet patient needs.

Delfi Diagnostics, Inc. is a biotechnology company based in Baltimore, Maryland, focused on the early detection of cancer through innovative blood tests. Founded in 2018, the company employs artificial intelligence and genome sequencing to identify unique patterns of DNA fragmentation in the blood, enabling the detection of previously unrecognized cancer-associated cell-free DNA fragments. By utilizing machine learning, Delfi Diagnostics aims to develop high-precision, non-invasive blood tests that allow healthcare professionals to detect cancer at its most curable stage, thereby facilitating timely and effective treatment options for patients.

Frontera Therapeutics is a biotechnology company dedicated to developing and commercializing recombinant virus-based gene therapies. Its mission is to establish an affordable and scalable platform for producing high-quality rAAV therapies, targeting genetic disorders such as those affecting the eye, liver, metabolism, and neuromuscular systems.

Aspen Neuroscience develops autologous neuron replacement therapies based on induced pluripotent stem cells to treat neurological conditions, starting with Parkinson's disease. The company uses patient-specific restorative cell therapies by combining pluripotent stem cell biology with genomic approaches and artificial intelligence to address both sporadic and familial forms of Parkinson's disease, focusing on autologous neuron replacement to modify disease progression. Based in San Diego, California, the company is a development-stage biotechnology company pursuing expansion of its iPSC-based platform to other brain disorders.

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.

ImmPACT Bio USA Inc. is a cell therapy company focused on developing engineered T-cells for the treatment of solid tumors. The company specializes in creating chimeric antigen receptor (CAR) T-cell therapies that target specific gene loss features in tumor cells, allowing for the selective destruction of cancerous tissues while preserving healthy ones. This technology aims to enhance tumor specificity, enabling the CAR-T cells to induce cytotoxicity only when engaging with tumor cells, thereby minimizing the safety risks commonly associated with existing CAR-T therapies. ImmPACT Bio's innovative approach seeks to improve treatment outcomes for cancer patients by offering a more precise and safer therapeutic option.

Alterome Therapeutics is a precision oncology biotechnology company focused on developing alteration-specific therapeutics to target high-value, validated oncogenic drivers. It employs structure-guided drug discovery and advanced computational chemistry to create small-molecule therapies that selectively address oncogenic alterations, with the aim of improving patient outcomes in cancer.

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.

Scout Bio, Inc. is a biotechnology company focused on advancing veterinary medicine through innovative therapies for chronic pet health conditions. Founded in 2016 and headquartered in Philadelphia, Pennsylvania, the company specializes in developing one-time treatments utilizing veterinary biotechnology and gene therapy. Scout Bio's offerings include therapies for conditions such as anemia associated with chronic kidney disease in cats, as well as a range of small molecular drugs, protein therapies, and gene therapies. Employing adeno-associated viral vector technology, Scout Bio aims to transform veterinary care by providing effective protein and monoclonal antibody therapeutics delivered through single intra-muscular injections, thereby enhancing the quality of life for pets and supporting veterinary professionals.

Vanqua Bio is a biotechnology company dedicated to discovering and developing innovative medicines for patients with neurodegenerative diseases. The company's proprietary platform employs human genetics and patient-derived neuronal cells to identify and validate novel disease pathways related to lysosomal dysfunction or aberrant immune system activation, aiming to translate these findings into clinically effective treatments.

iECURE is a gene editing company developing mutation-agnostic in vivo gene insertion therapies to treat rare, life-threatening liver disorders in children. Its approach seeks long-term, potentially curative treatment for metabolic liver diseases caused by loss-of-function mutations, with initial programs targeting ornithine transcarbamylase deficiency, citrullinemia type 1, and phenylketonuria.

ADARx Pharmaceuticals specializes in the development of RNA editing therapeutics. Founded in 2019, the company focuses on discovering and developing innovative treatments that utilize endogenous enzymes to precisely target and correct single point mutations in mRNA transcripts, restoring the production of functional proteins.

GentiBio, Inc. is a biotherapeutics company that specializes in developing engineered regulatory T cells, known as 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, Washington, GentiBio employs a proprietary platform that combines autologous and allogeneic technologies. This platform enhances the ability to restore immune tolerance and addresses significant limitations found in existing regulatory T-cell therapeutics. By focusing on the underlying mechanisms of immune system disorders, GentiBio aims to provide innovative therapeutic solutions that tackle the root causes of diseases caused by dysregulated immune responses. The company is co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of advanced immunotherapy development.

Adela specializes in developing innovative technologies for early detection of high-morbidity conditions like cancer through routine blood tests. Its proprietary platform efficiently captures and analyzes the methylome, focusing on highly informative regions to enhance diagnostic accuracy.

Biocytogen specializes in developing gene-targeted animal models for life science research, offering customized mouse models, phenotype analysis, and extreme genome editing platforms. It also provides integrated solutions for antibody drug development, including animal model generation, therapeutic antibody discovery, and preclinical validation studies.

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.

Magenta Therapeutics, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on advancing stem cell transplantation and related therapies. Founded in 2015, the company aims to enhance the patient experience in transplant medicine by developing innovative treatments that make stem cell transplants safer and more accessible. Its pipeline includes several key candidates, such as C100, C200, and C300 targeted antibody-drug conjugates designed for transplant conditioning; MGTA-145, a stem cell mobilization product; and MGTA-456, an allogeneic stem cell therapy. Additionally, Magenta is exploring E478, a small molecule aimed at expanding gene-modified stem cells, and G100, an antibody-drug conjugate to mitigate acute graft-versus-host disease. The company has established research collaborations with other biotechnology firms to further investigate the potential of its therapies in treating blood cancers, genetic disorders, and autoimmune diseases, ultimately striving to transform stem cell transplantation from a last-resort option into a more viable and effective treatment for patients.

Caris Life Sciences specializes in advanced molecular profiling technologies for cancer diagnostics and drug development. Established in 1987, it offers prognostic testing services, genomic profiling, and clinico-genomic data platforms to improve patient outcomes. The company operates globally with offices in the US and Switzerland.

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.

Treeline Biosciences is a biotechnology company focused on developing precision medicines for cancer and other serious diseases. Founded in 2021 and based in Stamford, Connecticut, the company uses a platform that integrates pathophysiology, genomics, and pattern recognition to identify therapeutic approaches that address unmet patient needs.

Theseus Pharmaceuticals is a biopharmaceutical company dedicated to enhancing the lives of cancer patients through the development of targeted therapies. The company focuses on discovering and commercializing innovative treatments that specifically address drug resistance mutations in critical driver oncogenes, which are genes that, when mutated, contribute to cancer progression. By leveraging advanced technology, Theseus Pharmaceuticals aims to transform cancer care and improve patient outcomes.

Founded in 2018, Scribe Therapeutics specializes in engineering and developing in vivo therapies based on CRISPR technology to rewrite and repair genetic disorders. Its proprietary platform, X-Editing (XE), enables greater efficacy, specificity, and deliverability compared to existing CRISPR technologies.

Graphite Bio is a clinical-stage gene editing company focused on targeted DNA integration to treat serious diseases. Its platform aims to precisely insert, repair, or replace genetic sequences by integrating therapeutic cargoes at predefined genomic locations, enabling correction of mutations or replacement of disease-causing genes while preserving normal regulatory control. Based in South San Francisco, the company pursues high-efficiency precision gene editing to address underlying causes of disease.

Century Therapeutics is a biotechnology company developing allogeneic cell therapies derived from induced pluripotent stem cells to treat cancer and related diseases. Its platform enables engineering of iPSCs into NK and T cells with master cell banks of modified cells that can be expanded and differentiated into standardized, off-the-shelf therapies. The company combines CRISPR-mediated gene editing, proprietary chimeric antigen receptors, Allo-Evasion technology, and manufacturing capabilities to enable scalable production of homogeneous cell products for hematologic and solid tumors. While focused on oncology, its platform also targets autoimmune and inflammatory diseases with the goal of improving cell therapy performance and reducing host immune rejection.

AnchorDx is a biotechnology company specializing in next-generation sequencing techniques, particularly through the use of molecular liquid biopsies. This innovative approach allows for non-invasive access to genetic information, enabling patients to gain insights into their physiological conditions and better prepare for potential health issues. With a core team boasting over 20 years of experience in cancer genomics, genetics, and bioinformatics, AnchorDx aims to lead in precision medicine by developing comprehensive clinical applications and research platforms. The company fosters collaborations with various stakeholders, including hospitals, research institutes, and third-party testing agencies, to create a complete industrial chain that facilitates the transformation of academic research into clinical products. Through its commitment to developing independent intellectual property and competitive clinical testing solutions, AnchorDx provides accurate and comprehensive services to patients, healthcare providers, and medical institutions.

Evox Therapeutics Limited is a biotechnology company based in Oxford, United Kingdom, that specializes in developing exosome-based therapeutics for the treatment of severe diseases with limited treatment options. Founded in 2016, the company focuses on harnessing and engineering extracellular vesicles, known as exosomes, to facilitate targeted delivery of nucleic acids and proteins. Evox's innovative platform allows for the modification of exosomes using advanced molecular engineering techniques, enabling effective drug delivery to specific organs, including the brain and central nervous system. By leveraging these natural delivery capabilities, Evox aims to overcome the limitations associated with conventional protein, antibody, and nucleic acid therapies, thereby creating novel therapeutic solutions that could significantly impact human health. The company is supported by a robust intellectual property portfolio and is positioned as a leader in this emerging therapeutic space.

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.

Delfi Diagnostics, Inc. is a biotechnology company based in Baltimore, Maryland, focused on the early detection of cancer through innovative blood tests. Founded in 2018, the company employs artificial intelligence and genome sequencing to identify unique patterns of DNA fragmentation in the blood, enabling the detection of previously unrecognized cancer-associated cell-free DNA fragments. By utilizing machine learning, Delfi Diagnostics aims to develop high-precision, non-invasive blood tests that allow healthcare professionals to detect cancer at its most curable stage, thereby facilitating timely and effective treatment options for patients.

Founded in 2020 and headquartered in Boston, Massachusetts, Scorpion Therapeutics is a clinical-stage biotechnology company specializing in precision oncology. It develops targeted small-molecule drugs to treat cancer by focusing on three areas: therapies against known oncogenes, agents for currently undruggable targets, and drugs for new targets.

Achilles Therapeutics Ltd. is a biopharmaceutical company based in London, United Kingdom, specializing in the development of patient-specific immunotherapies designed to combat cancer. Founded in 2016, the company focuses on personalized vaccines and T cell therapies that utilize the body's immune system to target and eliminate cancer cells. Achilles Therapeutics aims to identify and target truncal tumor neoantigens and other markers present on cancer cells, allowing for precise attacks on tumors while sparing healthy tissue. The company is currently conducting clinical trials, including the CHIRON trial for non-small-cell lung cancer and the THETIS trial for recurrent or metastatic melanoma, to evaluate the efficacy and safety of its innovative therapies.

Decibel Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing innovative therapies aimed at protecting, repairing, and restoring hearing. The company addresses various hearing disorders, including those related to pediatric cancers, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as conditions characterized by persistent tinnitus. Decibel Therapeutics employs a multifaceted approach that includes drug delivery to the inner ear, advanced imaging techniques, and pharmacokinetic and pharmacodynamic modeling. The company leverages its expertise in inner ear biology, single-cell genomics, and bioinformatics to develop effective treatments for hearing loss and related disorders. Established in 2013 and headquartered in Boston, Massachusetts, Decibel Therapeutics was originally known as Hearing, Inc. before rebranding in 2014.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative cell therapies for cancer treatment. Based in Shanghai, China, Gracell's lead product candidates include GC012F, a dual-targeting autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia. Both candidates are currently undergoing Phase I clinical trials. Additionally, the company is advancing GC007F for B cell non-Hodgkin's lymphoma and GC027 for adult T cell acute lymphoblastic leukemia, among others. Gracell also has a range of earlier-stage product candidates addressing various cancer types, such as ovarian cancer and breast cancer. Founded in 2017, the company aims to provide effective cellular therapeutics to improve outcomes for patients with hematological malignancies and solid tumors.

Caris Life Sciences specializes in advanced molecular profiling technologies for cancer diagnostics and drug development. Established in 1987, it offers prognostic testing services, genomic profiling, and clinico-genomic data platforms to improve patient outcomes. The company operates globally with offices in the US and Switzerland.

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 precision oncology company focused on developing targeted cancer therapies guided by functional genomics. It aims to create a pipeline of treatments for patients with currently unaddressed mutations across established oncology targets.

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.

Kinnate Biopharma Inc. is a biopharmaceutical company dedicated to the discovery and development of small molecule kinase inhibitors aimed at treating genomically defined cancers. Established in 2018 and headquartered in San Diego, California, the company focuses on precision oncology therapeutics, leveraging its expertise in structure-based drug discovery and translational research. Kinnate's lead product candidate, KIN002787, is a rapidly accelerated fibrosarcoma inhibitor currently in preclinical development for lung cancer, melanoma, and other solid tumors. Additionally, the company is advancing other preclinical candidates, including KIN003, which targets cancer-associated alterations in FGFR2 and FGFR3 genes, and a Cyclin-Dependent Kinase 12 inhibitor in the KIN004 program. Kinnate Biopharma is committed to developing targeted therapeutics for underserved patient populations, aiming to expand the reach of precision medicine in cancer treatment.

ImmPACT Bio USA Inc. is a cell therapy company focused on developing engineered T-cells for the treatment of solid tumors. The company specializes in creating chimeric antigen receptor (CAR) T-cell therapies that target specific gene loss features in tumor cells, allowing for the selective destruction of cancerous tissues while preserving healthy ones. This technology aims to enhance tumor specificity, enabling the CAR-T cells to induce cytotoxicity only when engaging with tumor cells, thereby minimizing the safety risks commonly associated with existing CAR-T therapies. ImmPACT Bio's innovative approach seeks to improve treatment outcomes for cancer patients by offering a more precise and safer therapeutic option.

GentiBio, Inc. is a biotherapeutics company that specializes in developing engineered regulatory T cells, known as 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, Washington, GentiBio employs a proprietary platform that combines autologous and allogeneic technologies. This platform enhances the ability to restore immune tolerance and addresses significant limitations found in existing regulatory T-cell therapeutics. By focusing on the underlying mechanisms of immune system disorders, GentiBio aims to provide innovative therapeutic solutions that tackle the root causes of diseases caused by dysregulated immune responses. The company is co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of advanced immunotherapy development.

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 develops autologous neuron replacement therapies based on induced pluripotent stem cells to treat neurological conditions, starting with Parkinson's disease. The company uses patient-specific restorative cell therapies by combining pluripotent stem cell biology with genomic approaches and artificial intelligence to address both sporadic and familial forms of Parkinson's disease, focusing on autologous neuron replacement to modify disease progression. Based in San Diego, California, the company is a development-stage biotechnology company pursuing expansion of its iPSC-based platform to other brain disorders.

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.

Emendo Biotherapeutics is a biotechnology company focused on advancing genome editing technologies to develop genetic medicines. Established by a team of experts with backgrounds in protein engineering and DNA repair from prestigious institutions, Emendo aims to tackle the challenges associated with gene therapy. By innovating and transforming existing tools, the company seeks to address diseases and disorders that are presently deemed untreatable. Emendo's approach fuses diverse scientific disciplines with creative problem-solving, enabling the development of advanced gene editing solutions that enhance the efficacy of gene therapy and expand its potential applications. Through this commitment to pushing the boundaries of science, Emendo Biotherapeutics strives to fulfill the promise of gene editing for improved patient outcomes.

Kinnate Biopharma Inc. is a biopharmaceutical company dedicated to the discovery and development of small molecule kinase inhibitors aimed at treating genomically defined cancers. Established in 2018 and headquartered in San Diego, California, the company focuses on precision oncology therapeutics, leveraging its expertise in structure-based drug discovery and translational research. Kinnate's lead product candidate, KIN002787, is a rapidly accelerated fibrosarcoma inhibitor currently in preclinical development for lung cancer, melanoma, and other solid tumors. Additionally, the company is advancing other preclinical candidates, including KIN003, which targets cancer-associated alterations in FGFR2 and FGFR3 genes, and a Cyclin-Dependent Kinase 12 inhibitor in the KIN004 program. Kinnate Biopharma is committed to developing targeted therapeutics for underserved patient populations, aiming to expand the reach of precision medicine in cancer treatment.

Aspen Neuroscience develops autologous neuron replacement therapies based on induced pluripotent stem cells to treat neurological conditions, starting with Parkinson's disease. The company uses patient-specific restorative cell therapies by combining pluripotent stem cell biology with genomic approaches and artificial intelligence to address both sporadic and familial forms of Parkinson's disease, focusing on autologous neuron replacement to modify disease progression. Based in San Diego, California, the company is a development-stage biotechnology company pursuing expansion of its iPSC-based platform to other brain disorders.

Repare Therapeutics is a precision oncology company that discovers and develops novel therapeutics using its synthetic lethality approach. It employs its proprietary SNIPRx platform to systematically identify and target genomic instability, particularly DNA damage repair pathways in cancer cells. The company's lead product candidate, RP-3500, is an oral small molecule inhibitor for treating solid tumors with specific DNA damage repair-related alterations.

Passage Bio is a gene therapy company dedicated to developing transformative treatments for rare, monogenic central nervous system disorders. It has an integrated pipeline of five AAV-delivered therapeutics and collaborates with the University of Pennsylvania's Gene Therapy Program for preclinical work.

SpringWorks Therapeutics is a clinical-stage biopharmaceutical company applying precision medicine to develop medicines for severe rare diseases and cancer. It maintains a differentiated targeted oncology portfolio with lead candidates nirogacestat, a gamma secretase inhibitor, and mirdametinib, a MEK inhibitor, both in late-stage trials; the pipeline also includes BGB-3245, an oral inhibitor of activating BRAF mutations, in Phase I, and several combination programs. The company pursues collaborations with BeiGene, GlaxoSmithKline, and Allogene and has license agreements with Pfizer for nirogacestat and mirdametinib. Founded in 2017 and headquartered in Stamford, Connecticut, SpringWorks aims to advance potentially registrational studies in rare tumor types and genetically defined cancers through partnerships that expand its portfolio and patient access.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, that specializes in developing and commercializing therapeutics for patients with rare genetic disorders of hemoglobin. Founded in 2016, the company is advancing IMR-687, an oral, once-daily therapeutic designed to treat sickle cell disease and beta-thalassemia. IMR-687 is a highly selective small molecule inhibitor of PDE9, engineered to have a multimodal mechanism of action that impacts various cell types, including red blood cells and white blood cells, as well as adhesion mediators. Through its innovative approach, Imara aims to provide disease-modifying treatments for individuals affected by hemoglobinopathies.

Passage Bio is a gene therapy company dedicated to developing transformative treatments for rare, monogenic central nervous system disorders. It has an integrated pipeline of five AAV-delivered therapeutics and collaborates with the University of Pennsylvania's Gene Therapy Program for preclinical work.

Modis Therapeutics, Inc. is a biopharmaceutical company focused on developing disease-modifying therapies for rare genetic diseases characterized by high unmet medical needs. Incorporated in 2016 and based in Oakland, California, the company is known for its lead therapy, MT1621, which aims to restore mitochondrial DNA replication fidelity. This therapy specifically targets thymidine kinase 2 deficiency (TK2d), an inherited mitochondrial disorder. By improving nucleotide balance and increasing mitochondrial DNA copy number, Modis Therapeutics' approach seeks to enhance cellular function and prolong the lives of affected patients. As of September 2019, Modis Therapeutics operates as a subsidiary of Zogenix, Inc.

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.

Theseus Pharmaceuticals is a biopharmaceutical company dedicated to enhancing the lives of cancer patients through the development of targeted therapies. The company focuses on discovering and commercializing innovative treatments that specifically address drug resistance mutations in critical driver oncogenes, which are genes that, when mutated, contribute to cancer progression. By leveraging advanced technology, Theseus Pharmaceuticals aims to transform cancer care and improve patient outcomes.

Synthorx, Inc. is a biopharmaceutical company specializing in the development of innovative protein therapeutics using advanced synthetic biology techniques. Founded in 2014 and headquartered in La Jolla, California, Synthorx focuses on its proprietary Synthorin programs aimed at treating cancer and autoimmune disorders. Its lead product candidate, THOR-707, is a modified form of recombinant human IL-2, currently being evaluated for various solid tumors both as a standalone treatment and in combination with immune checkpoint inhibitors. The company also develops Synthorins targeting IL-2 for autoimmune conditions, IL-10 for immuno-oncology applications, and IL-15 for regulating immune responses. Synthorx leverages a unique platform that enables the incorporation of synthetic DNA base pairs to enhance the specificity and efficacy of its protein therapeutics, thus offering improved treatment options for patients. Previously known as Alinos, Inc., the company acquired exclusive rights to its synthetic biology technology based on groundbreaking research from The Scripps Research Institute. As of January 2020, Synthorx operates as a subsidiary of Sanofi.

Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapeutics for unmet medical needs in cancer treatment. The company specializes in the field of epigenetics, aiming to address cancers associated with abnormal gene expression and drug resistance. Its lead product candidates include CPI-0610, which is undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, currently in Phase Ib/II trials for metastatic castration-resistant prostate cancer in conjunction with androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the Phase I dose escalation stage of a Phase I/II trial aimed at treating solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. before rebranding to its current name in March 2008.

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.

SpringWorks Therapeutics is a clinical-stage biopharmaceutical company applying precision medicine to develop medicines for severe rare diseases and cancer. It maintains a differentiated targeted oncology portfolio with lead candidates nirogacestat, a gamma secretase inhibitor, and mirdametinib, a MEK inhibitor, both in late-stage trials; the pipeline also includes BGB-3245, an oral inhibitor of activating BRAF mutations, in Phase I, and several combination programs. The company pursues collaborations with BeiGene, GlaxoSmithKline, and Allogene and has license agreements with Pfizer for nirogacestat and mirdametinib. Founded in 2017 and headquartered in Stamford, Connecticut, SpringWorks aims to advance potentially registrational studies in rare tumor types and genetically defined cancers through partnerships that expand its portfolio and patient access.

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, Inc. is a genome editing company dedicated to developing innovative treatments for rare diseases, particularly for pediatric patients with significant unmet medical needs. Utilizing its proprietary GeneRide technology platform, LogicBio aims to integrate corrective genes into patients' genomes to achieve therapeutic effects. The company's lead product candidate, LB-001, targets Methylmalonic Acidemia, a serious condition that manifests at birth. Additionally, LogicBio is advancing other product candidates, including LB-101 and LB-301, the latter developed in collaboration with Takeda Pharmaceutical Company to address Crigler-Najjar syndrome. The company also partners with the Children's Medical Research Institute to create new viral vectors, enhancing the potential of its gene therapy approaches. Founded in 2014, LogicBio is headquartered in Lexington, Massachusetts.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

Guardant Health is a precision oncology company specializing in blood tests and analytics. It provides liquid biopsy tests like Guardant360 for advanced cancer detection, GuardantOMNI for broader gene panels, LUNAR-1 for minimal residual disease detection, and LUNAR-2 for early cancer detection. The company also offers development services to biopharmaceutical companies.

Rhythm Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing peptide therapeutics for treating rare genetic disorders that cause life-threatening metabolic issues. Its lead product candidate, setmelanotide, is a potent melanocortin-4 receptor agonist designed for precision medicine to treat hyperphagia and severe obesity resulting from rare MC4R pathway diseases.

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.

Emendo Biotherapeutics is a biotechnology company focused on advancing genome editing technologies to develop genetic medicines. Established by a team of experts with backgrounds in protein engineering and DNA repair from prestigious institutions, Emendo aims to tackle the challenges associated with gene therapy. By innovating and transforming existing tools, the company seeks to address diseases and disorders that are presently deemed untreatable. Emendo's approach fuses diverse scientific disciplines with creative problem-solving, enabling the development of advanced gene editing solutions that enhance the efficacy of gene therapy and expand its potential applications. Through this commitment to pushing the boundaries of science, Emendo Biotherapeutics strives to fulfill the promise of gene editing for improved patient outcomes.

Arrowhead Pharmaceuticals is a biotechnology company focused on creating medicines for diseases with a genetic basis driven by overproduction of proteins. The company develops RNA interference therapeutics designed to silence disease-related genes and reduce pathogenic protein production. Its pipeline includes candidates such as ARO-AAT for liver disease caused by alpha-1 antitrypsin deficiency, ARO-APOC3 for high triglycerides, ARO-ANG3 to lower angiopoietin-like protein 3, ARO-HSD for liver disorders, ARO-ENaC to modulate airway sodium channels, ARO-Lung2 for chronic obstructive pulmonary disease, and ARO-COV for pulmonary infections, with ARO-HIF2 in earlier stage for certain kidney cancer. It is also pursuing programs like JNJ-3989 for chronic hepatitis B, Olpasiran for apolipoprotein A production, and ARO-AMG1 for cardiovascular targets. The company collaborates with Janssen and Takeda on RNAi therapeutics and was formerly Arrowhead Research Corporation, renamed Arrowhead Pharmaceuticals in 2016 after being established in 1989. It is headquartered in Pasadena, California.

Fore Biotherapeutics is a precision oncology company focused on developing targeted cancer therapies guided by functional genomics. It aims to create a pipeline of treatments for patients with currently unaddressed mutations across established oncology targets.

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.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

LogicBio Therapeutics, Inc. is a genome editing company dedicated to developing innovative treatments for rare diseases, particularly for pediatric patients with significant unmet medical needs. Utilizing its proprietary GeneRide technology platform, LogicBio aims to integrate corrective genes into patients' genomes to achieve therapeutic effects. The company's lead product candidate, LB-001, targets Methylmalonic Acidemia, a serious condition that manifests at birth. Additionally, LogicBio is advancing other product candidates, including LB-101 and LB-301, the latter developed in collaboration with Takeda Pharmaceutical Company to address Crigler-Najjar syndrome. The company also partners with the Children's Medical Research Institute to create new viral vectors, enhancing the potential of its gene therapy approaches. Founded in 2014, LogicBio is headquartered in Lexington, Massachusetts.

Guardant Health is a precision oncology company specializing in blood tests and analytics. It provides liquid biopsy tests like Guardant360 for advanced cancer detection, GuardantOMNI for broader gene panels, LUNAR-1 for minimal residual disease detection, and LUNAR-2 for early cancer detection. The company also offers development services to biopharmaceutical companies.

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.

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 based in Cambridge, Massachusetts, specializing in the development of innovative treatments for rare and metabolic diseases associated with the liver. Founded in 2013, the company has a robust pipeline of gene therapy programs, including DTX101 and DTX201 for hemophilia B and A, respectively. Additionally, its portfolio encompasses DTX301 for ornithine transcarbamylase deficiency, DTX401 for glycogen storage disease type Ia, DTX501 for phenylketonuria, DTX701 for Wilson disease, and DTX601 for citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, gaining access to a significant portfolio of patents related to adeno-associated virus (AAV) therapeutics. This collaboration supports the company's aim to advance its gene therapy platform and enhance its product development capabilities in the treatment of rare diseases. Dimension Therapeutics operates as a subsidiary of Ultragenyx Pharmaceutical Inc. since November 2017.

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 is a global genomics service company offering DNA sequencing, gene synthesis, molecular biology, next-generation sequencing, bioinformatics, and GLP regulatory services. Established in 1999, it serves pharmaceutical, biotechnology, academic, agricultural, government, and clinical institutions worldwide.

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 based in Cambridge, Massachusetts, specializing in the development of innovative treatments for rare and metabolic diseases associated with the liver. Founded in 2013, the company has a robust pipeline of gene therapy programs, including DTX101 and DTX201 for hemophilia B and A, respectively. Additionally, its portfolio encompasses DTX301 for ornithine transcarbamylase deficiency, DTX401 for glycogen storage disease type Ia, DTX501 for phenylketonuria, DTX701 for Wilson disease, and DTX601 for citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, gaining access to a significant portfolio of patents related to adeno-associated virus (AAV) therapeutics. This collaboration supports the company's aim to advance its gene therapy platform and enhance its product development capabilities in the treatment of rare diseases. Dimension Therapeutics operates as a subsidiary of Ultragenyx Pharmaceutical Inc. since November 2017.

Seragon Pharmaceuticals, Inc., based in Irvine, California, is a research-focused biopharmaceutical company dedicated to enhancing human and animal health through innovative scientific advancements. The company specializes in the development of orally active selective estrogen receptor degraders (SERDs), particularly targeting metastatic breast cancer. Its lead drug candidate, ARN-810, is currently under evaluation for the treatment of estrogen receptor-positive breast cancer. By leveraging cutting-edge research in metabolism, gene therapy, and bioinformatics, Seragon Pharmaceuticals aims to facilitate access to significant medical breakthroughs, encompassing both clinical applications and consumer products.