New Enterprise Associates

Abcuro, Inc. is a biotechnology company focused on developing immunotherapies for the treatment of autoimmunity and cancer. Based in Newton, Massachusetts, Abcuro creates therapeutic antibodies targeting novel mechanisms related to these diseases. The company utilizes advanced bioinformatics to analyze transcription data from human diseases, which aids in the identification of new therapeutic targets. Through ex-vivo validation in diseased tissues, Abcuro seeks to uncover innovative methods to modulate key components of the immune system. Established in 2015, Abcuro aims to enhance treatment strategies in the healthcare industry by offering effective solutions for complex immunological conditions.

Tune Therapeutics is focused on developing innovative cell and gene therapies that utilize epigenetic programming to address serious diseases. By harnessing the epigenome, the company aims to create new pathways for treatment without making changes to the DNA sequence itself. This approach allows for the potential development of therapies for complex and widespread medical conditions, positioning Tune Therapeutics at the forefront of advancements in the field of epigenetics.

Ouro Medicines is a biotechnology company dedicated to immunology, focusing on treating chronic, immune-mediated illnesses. It specializes in integrating scientific knowledge and clinical trial experience to develop innovative, cell-depleting therapies. The company's core technology involves T cell engager antibodies that target and eliminate specific pathogenic cells, aiming to provide extended remissions for patients without the need for continuous immunosuppression.

Orbis Medicines is a company focused on drug discovery, utilizing a unique macrocyclic chemistry and computational platform to develop new therapeutics. By employing high-throughput techniques, Orbis aims to accelerate the drug discovery process, particularly for challenging targets that address significant unmet medical needs. Their innovative approach is designed to enhance the efficiency and effectiveness of developing treatments, ultimately improving patient outcomes.

Senti Bio, established in 2016 and headquartered in South San Francisco, specializes in synthetic biotechnology for therapeutic development. The company's core focus is engineering gene circuits, a platform technology that enables precise control over cellular behavior. Senti Bio's innovative approach allows for the creation of therapies with enhanced specificity and control, particularly in oncology. Their product pipeline includes SENTI-202, an off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A, a treatment for hepatocellular carcinoma.

Avenzo Therapeutics is a clinical-stage company focused on developing innovative oncology therapies aimed at treating cancer. The company’s platform is dedicated to creating novel treatments specifically for underserved cancers, including rare and genomically defined types. By advancing a pipeline of targeted drug candidates, Avenzo Therapeutics seeks to enhance treatment options for both researchers and patients, addressing significant gaps in current cancer care.

Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies for patients with significant unmet medical needs, particularly in fibrosis, lysosomal storage disorders, and oncology. The company specializes in drugs that target nuclear receptors, transcription factors, and epigenetic modulation. Its primary product candidate, lanifibranor, is being developed for the treatment of non-alcoholic steatohepatitis (NASH), a condition currently lacking approved therapies. Inventiva has established a robust pipeline supported by a proprietary discovery engine, a dedicated research and development facility, and a skilled team with extensive experience in drug development. In addition to advancing lanifibranor, the company is also working on several earlier-stage programs aimed at addressing various medical conditions. All operations are conducted in France.

858 Therapeutics is a biotechnology company dedicated to the discovery of small molecule therapeutics aimed at treating cancers and other diseases, particularly those resistant to existing therapies. The company employs innovative strategies in areas such as synthetic lethality, innate immunity, and RNA modulation to develop its therapeutic candidates. Its lead program focuses on a PARG inhibitor, which is currently in early clinical development. By leveraging its platform to investigate the role of RNA modifying proteins in disease biology, 858 Therapeutics is generating a pipeline of targeted small molecules that address critical unmet medical needs in oncology and immunology.

Sakana AI is a research-focused company dedicated to developing innovative foundation models inspired by nature. It specializes in creating an AI model capable of generating text, images, video, code, and multimedia, facilitating collaborative research and development in generative AI. The company's AI development process emphasizes evolution and collective intelligence.

MBX Biosciences, Inc. is a biotechnology company based in Carmel, Indiana, specializing in the development of therapeutics for rare endocrine disorders. Established in 2018, the company focuses on creating peptides aimed at treating genetic diseases that affect glandular hormones, which play a crucial role in the body’s chemical messaging system. By targeting conditions with inadequate treatment options, MBX Biosciences is dedicated to discovering, developing, and commercializing innovative endocrine therapeutics.

Cardurion Pharmaceuticals, LLC is a biotechnology company based in Boston, Massachusetts, established in 2017. The company specializes in developing innovative therapeutics aimed at treating heart failure and other cardiovascular diseases. Cardurion is advancing clinical programs that focus on inhibiting PDE9 and CaMKII, utilizing its knowledge of cardiovascular signaling pathways to meet significant patient needs in this area.

Zenas BioPharma is a clinical-stage biopharmaceutical company based in Florida, dedicated to developing immune-based therapies for patients. The company's lead product candidate, obexelimab, is a bifunctional monoclonal antibody that targets both CD19 and FcγRIIb, which are found on B cells. This innovative approach aims to inhibit the activity of B cells involved in various autoimmune diseases while preserving their overall population, thus offering a potential therapeutic advantage over traditional treatments that deplete these cells.

Xaira Therapeutics is an integrated biotechnology company focused on revolutionizing drug discovery and development through the application of artificial intelligence. Headquartered in the San Francisco Bay Area, Xaira combines expertise in machine learning, extensive biological and clinical data generation, and robust therapeutic product development. By harnessing advanced AI techniques, the company aims to enhance the understanding of biological processes and improve the effectiveness of therapies. Xaira's approach encompasses all stages of drug discovery, striving to transform how diseases are treated and enabling clients to deliver innovative therapeutic solutions.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

Avenzo Therapeutics is a clinical-stage company focused on developing innovative oncology therapies aimed at treating cancer. The company’s platform is dedicated to creating novel treatments specifically for underserved cancers, including rare and genomically defined types. By advancing a pipeline of targeted drug candidates, Avenzo Therapeutics seeks to enhance treatment options for both researchers and patients, addressing significant gaps in current cancer care.

Regulus Therapeutics Inc. is a clinical stage biopharmaceutical company based in San Diego, California, specializing in the discovery and development of therapies that target microRNAs for various diseases. The company is advancing its lead product candidates, RG-012 and RGLS4326, which are in Phase II and Phase Ib clinical trials, respectively. RG-012 targets miR-21 for the treatment of Alport syndrome, a severe kidney disease, while RGLS4326 targets miR-17 for autosomal dominant polycystic kidney disease. In addition to these candidates, Regulus is developing a pipeline of preclinical drug products, including RGLS5579, aimed at inhibiting miR-10b, as well as programs targeting hepatitis B virus and non-alcoholic steatohepatitis. Founded in 2007, Regulus Therapeutics is at the forefront of leveraging the therapeutic potential of microRNA, a significant scientific breakthrough in recent years.

Comanche Biopharma is a biopharmaceutical company focused on developing an investigational small interfering RNA (siRNA) medicine aimed at treating preterm preeclampsia. The company is committed to ensuring that all women and their babies have access to safe and effective therapies for life-threatening complications during pregnancy. Comanche Biopharma's mission emphasizes the importance of evidence-based and affordable solutions, enabling women to receive cost-effective treatments that address the root causes of preeclampsia.

Nexo Therapeutics is a biotechnology company focused on developing small-molecule therapies for cancer patients who have few effective treatment options. The company utilizes a specialized platform that integrates covalent ligand discovery with chemical biology to create innovative oncology drugs aimed at challenging targets that have been difficult to address in the past. By advancing its pipeline of novel therapies, Nexo Therapeutics seeks to provide new avenues for treatment, ultimately enhancing the care available to individuals battling cancer and other related diseases.

Gossamer Bio is a clinical-stage biopharmaceutical company based in San Diego that specializes in the discovery, development, and commercialization of innovative therapeutic products targeting significant unmet medical needs. Founded by former executives from Receptos, the company focuses on areas such as immunology, inflammation, fibrosis, and oncology. Its pipeline includes several promising candidates: Seralutinib, aimed at addressing pulmonary arterial hypertension; GB004, for inflammatory bowel disease; GB1275, targeting various oncology indications; and GB001, designed for moderate-to-severe eosinophilic asthma. Gossamer Bio's strategy involves leveraging a rich in-licensing environment to enhance its therapeutic offerings, supported by a team recognized for their successful track record in drug development.

Tagworks Pharmaceuticals is a privately held biotech company based in the Netherlands, established in 2011 as a spin-out from Philips Healthcare. The company focuses on developing innovative in vivo chemistry to enhance the effectiveness of cancer therapies and to facilitate novel companion diagnostics. Operating from the Radboud University Medical Center in Nijmegen, Tagworks specializes in antibody-based imaging and therapy technologies. Its proprietary approach allows for the selective chemical manipulation of tagged antibodies in vivo, which improves the efficacy of established methods like radioimmuno-imaging and antibody-drug conjugates. This technology enables medical professionals to conduct imaging with significantly reduced radiation exposure for patients.

Regulus Therapeutics Inc. is a clinical stage biopharmaceutical company based in San Diego, California, specializing in the discovery and development of therapies that target microRNAs for various diseases. The company is advancing its lead product candidates, RG-012 and RGLS4326, which are in Phase II and Phase Ib clinical trials, respectively. RG-012 targets miR-21 for the treatment of Alport syndrome, a severe kidney disease, while RGLS4326 targets miR-17 for autosomal dominant polycystic kidney disease. In addition to these candidates, Regulus is developing a pipeline of preclinical drug products, including RGLS5579, aimed at inhibiting miR-10b, as well as programs targeting hepatitis B virus and non-alcoholic steatohepatitis. Founded in 2007, Regulus Therapeutics is at the forefront of leveraging the therapeutic potential of microRNA, a significant scientific breakthrough in recent years.

Launchpad Therapeutics is an antibody-focused precision oncology company that specializes in developing early mutant-selective antibodies to enhance cancer therapies. Utilizing artificial intelligence-driven programs, the company aims to create novel monoclonal antibodies, facilitating drug design, development, and testing for research purposes. This innovative approach provides medical professionals with new alternatives for cancer treatment, addressing the complexities of the disease through targeted therapeutic strategies.

Escient Pharmaceuticals is a biotechnology company based in San Diego, California, that develops and manufactures drugs targeting G protein-coupled receptors (GPCRs) for the treatment of neuro-immuno-inflammatory and autoreactive diseases. Founded in 2017, the company specializes in harnessing the therapeutic potential of specific orphan GPCRs, particularly the Mas-Related G-Protein Receptors (Mrgprs), to address various neurosensory-inflammatory disorders. Escient's innovative approach aims to provide first-in-class therapies for conditions associated with mast cell-mediated disorders and cholestatic pruritus, thereby expanding treatment options for healthcare providers and patients facing these challenging diseases.

MBX Biosciences, Inc. is a biotechnology company based in Carmel, Indiana, specializing in the development of therapeutics for rare endocrine disorders. Established in 2018, the company focuses on creating peptides aimed at treating genetic diseases that affect glandular hormones, which play a crucial role in the body’s chemical messaging system. By targeting conditions with inadequate treatment options, MBX Biosciences is dedicated to discovering, developing, and commercializing innovative endocrine therapeutics.

Inversago Pharma Inc. is a preclinical-stage biotechnology company based in Montreal, Canada, focused on developing innovative therapies targeting metabolic and fibrotic disorders. The company specializes in peripherally-restricted CB1 receptor inverse agonists, which aim to offer new treatment options for conditions such as Prader-Willi syndrome, diabetes, and related metabolic disorders. Inversago Pharma's research includes addressing complications associated with diabetic kidney disease, non-alcoholic steatohepatitis, and various fibrotic diseases, including interstitial lung disease. Founded in 2015, the company is dedicated to enhancing patient outcomes through its pioneering approach to CB1 blockade.

Gossamer Bio is a clinical-stage biopharmaceutical company based in San Diego that specializes in the discovery, development, and commercialization of innovative therapeutic products targeting significant unmet medical needs. Founded by former executives from Receptos, the company focuses on areas such as immunology, inflammation, fibrosis, and oncology. Its pipeline includes several promising candidates: Seralutinib, aimed at addressing pulmonary arterial hypertension; GB004, for inflammatory bowel disease; GB1275, targeting various oncology indications; and GB001, designed for moderate-to-severe eosinophilic asthma. Gossamer Bio's strategy involves leveraging a rich in-licensing environment to enhance its therapeutic offerings, supported by a team recognized for their successful track record in drug development.

X4 Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of innovative therapeutics for primary immune deficiencies and cancer. The company's lead drug candidate, mavorixafor, is an oral small molecule antagonist of the chemokine receptor CXCR4, currently undergoing multiple clinical trials. These include a Phase III trial for patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome, as well as Phase Ib and Phase IIa trials focused on severe congenital neutropenia and clear cell renal cell carcinoma, respectively. In addition, X4 Pharmaceuticals is advancing X4P-002, targeting glioblastoma multiforme, and X4P-003 aimed at primary immune deficiencies. The company has also established a license agreement with Abbisko Therapeutics to develop mavorixafor in combination with checkpoint inhibitors for oncology applications. Founded in 2014, X4 Pharmaceuticals was previously known as Arsanis, Inc. before rebranding in March 2019.

Senti Bio, established in 2016 and headquartered in South San Francisco, specializes in synthetic biotechnology for therapeutic development. The company's core focus is engineering gene circuits, a platform technology that enables precise control over cellular behavior. Senti Bio's innovative approach allows for the creation of therapies with enhanced specificity and control, particularly in oncology. Their product pipeline includes SENTI-202, an off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A, a treatment for hepatocellular carcinoma.

Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies to treat serious and life-threatening genetic diseases. Code Bio leverages its novel multivalent synthetic DNA delivery platform, 3DNA, which has been engineered to overcome many of the challenges inherent with viral-based gene therapies such as immunogenicity, size and delivery limitations, re-dosability, and manufacturing complexity. The company is advancing an internal pipeline focused on select rare disease programs as well as establishing partnerships to take forward programs in both rare and prevalent diseases.

Celcuity Inc. is a clinical-stage biotechnology company based in Minneapolis, Minnesota, focused on advancing cancer treatment through innovative diagnostics. The company's CELsignia diagnostic platform utilizes living tumor cells to uncover specific cellular processes that drive cancer in individual patients. This approach enables the identification of new cancer sub-types that traditional molecular diagnostics may overlook, ultimately expanding therapeutic options. Celcuity is developing the CELsignia HSF test to diagnose two new sub-types of HER2-negative breast cancer and the CELsignia MP test for diagnosing 11 sub-types across various cancers, including breast, lung, colon, ovarian, kidney, and bladder. The company is also advancing its lead therapeutic candidate, gedatolisib, a pan-PI3K and mTOR inhibitor, currently being evaluated in a Phase 3 clinical trial for patients with advanced HR+/HER2- breast cancer.

Splice Bio is a biotechnology company based in Barcelona, Spain, focused on developing innovative gene therapies using its proprietary intein platform technology. Founded in 2012 and originally named Proteodesign, S.L., the company aims to address the needs of patients suffering from incurable genetic diseases. The intein platform, developed from research in the Muir Lab at Princeton University, seeks to overcome two significant limitations of traditional adeno-associated virus (AAV) therapies: it enhances the capacity for larger gene delivery and broadens the range of tissues that can be targeted for treatment.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

Sorriso Pharmaceuticals is a biotechnology company based in San Diego, California, founded in 2020. The company focuses on developing disease-modifying antibodies aimed at treating inflammatory diseases such as Crohn's disease and ulcerative colitis. Sorriso's innovative platform produces potent antibodies that can be delivered orally, ensuring they remain effective throughout the intestinal system. This approach aims to provide clinical benefits comparable to traditional injectable antibody therapeutics, thereby alleviating the treatment burden for patients who typically rely on injections for their care. By advancing its pipeline of oral therapies, Sorriso Pharmaceuticals seeks to improve patient outcomes and enhance the management of inflammatory diseases.

AviadoBio is focused on developing innovative gene therapies aimed at transforming the lives of individuals affected by neurodegenerative disorders, specifically frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The company leverages advanced research from King's College London and the UK Dementia Research Institute to create its therapeutic platform. By integrating a comprehensive understanding of brain biology with proprietary gene therapy techniques, AviadoBio seeks to address the challenges of effectively delivering treatments to targeted areas in the brain. This approach aims to enhance the therapeutic potential of gene therapies, offering hope for halting or potentially reversing the progression of these debilitating diseases.

Tune Therapeutics is focused on developing innovative cell and gene therapies that utilize epigenetic programming to address serious diseases. By harnessing the epigenome, the company aims to create new pathways for treatment without making changes to the DNA sequence itself. This approach allows for the potential development of therapies for complex and widespread medical conditions, positioning Tune Therapeutics at the forefront of advancements in the field of epigenetics.

Regulus Therapeutics Inc. is a clinical stage biopharmaceutical company based in San Diego, California, specializing in the discovery and development of therapies that target microRNAs for various diseases. The company is advancing its lead product candidates, RG-012 and RGLS4326, which are in Phase II and Phase Ib clinical trials, respectively. RG-012 targets miR-21 for the treatment of Alport syndrome, a severe kidney disease, while RGLS4326 targets miR-17 for autosomal dominant polycystic kidney disease. In addition to these candidates, Regulus is developing a pipeline of preclinical drug products, including RGLS5579, aimed at inhibiting miR-10b, as well as programs targeting hepatitis B virus and non-alcoholic steatohepatitis. Founded in 2007, Regulus Therapeutics is at the forefront of leveraging the therapeutic potential of microRNA, a significant scientific breakthrough in recent years.

Recludix Pharma is a biotechnology company focused on drug discovery, specifically targeting the development of potent and selective inhibitors for challenging protein targets associated with cancer and autoimmune diseases. The company utilizes a unique platform that combines custom-generated DNA-encoded libraries with massively parallel structure-activity relationship determination and proprietary screening tools. This integrated approach aims to enhance the identification and development of novel small molecule medicines, enabling healthcare professionals to create precision therapies that effectively address specific protein targets in these complex diseases.

858 Therapeutics is a biotechnology company dedicated to the discovery of small molecule therapeutics aimed at treating cancers and other diseases, particularly those resistant to existing therapies. The company employs innovative strategies in areas such as synthetic lethality, innate immunity, and RNA modulation to develop its therapeutic candidates. Its lead program focuses on a PARG inhibitor, which is currently in early clinical development. By leveraging its platform to investigate the role of RNA modifying proteins in disease biology, 858 Therapeutics is generating a pipeline of targeted small molecules that address critical unmet medical needs in oncology and immunology.

Shape Therapeutics, Inc. is a biotechnology company founded in 2018 and based in Seattle, Washington. The company specializes in RNA-editing gene therapy targeting neurodegenerative disorders, oncology, metabolic conditions, and rare genetic diseases. Its proprietary RNAfix technology enables precise editing of RNA using the body's natural cellular mechanisms, which minimizes risks associated with immunogenicity, cellular toxicity, and off-target DNA editing. Shape Therapeutics employs a comprehensive approach to RNA gene therapy, integrating innovative payload, delivery, and manufacturing platform technologies. This strategy aims to facilitate the development of effective treatments and potential cures for a wide range of diseases, with the goal of enhancing patient outcomes across the healthcare landscape.

Stablix Therapeutics is a biotechnology company specializing in Targeted Protein Stabilization (TPS). The company has developed a platform that produces heterobifunctional small molecules, known as RESTORACS, which selectively recruit deubiquitinase enzymes to remove ubiquitin from targeted proteins. This process enhances the stability and activity of these proteins. Stablix focuses on utilizing its innovative technology to create therapeutic programs aimed at treating rare diseases, cancer, and immunological disorders. By restoring protein function, Stablix aims to provide more effective treatment options for complex medical conditions.

Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.

Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies to treat serious and life-threatening genetic diseases. Code Bio leverages its novel multivalent synthetic DNA delivery platform, 3DNA, which has been engineered to overcome many of the challenges inherent with viral-based gene therapies such as immunogenicity, size and delivery limitations, re-dosability, and manufacturing complexity. The company is advancing an internal pipeline focused on select rare disease programs as well as establishing partnerships to take forward programs in both rare and prevalent diseases.

Arcellx is a clinical-stage biotechnology company focused on developing innovative cell therapies for patients with cancer and other incurable diseases. The company aims to enhance the safety, effectiveness, and accessibility of these therapies. Its lead product candidate, CART-ddBCMA, targets relapsed or refractory multiple myeloma and is currently undergoing a Phase 1 clinical study. This therapy has received multiple designations from the U.S. Food and Drug Administration, including Fast Track and Orphan Drug status. Additionally, Arcellx is advancing its controllable CAR-T therapy, ARC-SparX, through two clinical programs aimed at treating relapsed or refractory multiple myeloma and acute myeloid leukemia.

Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

Sagimet Biosciences is a clinical-stage biopharmaceutical company based in San Mateo, California, that specializes in developing novel therapeutics for oncology and infectious diseases. The company focuses on targeting dysfunctional metabolic pathways, particularly through its expertise in fatty acid synthase (FASN) biology. Its lead drug candidate, denifanstat, is a selective FASN inhibitor designed for the treatment of nonalcoholic steatohepatitis (NASH) and is administered as an oral, once-daily pill. Additionally, Sagimet is working on antiviral therapeutics aimed at addressing respiratory syncytial virus, cytomegalovirus, and other human pathogens. Originally known as 3-V Biosciences, Inc., the company rebranded to Sagimet Biosciences in August 2019 and has been active since its incorporation in 2006.

Personal Genome Diagnostics, Inc. specializes in patient-specific cancer genome analysis using advanced digital characterization and monitoring technologies. Founded in 2010 and based in Baltimore, Maryland, the company offers a range of products and services aimed at improving cancer diagnosis and treatment. Their METDetect Assay detects MET gene amplifications in cancer patients' circulation, providing detailed reports on tumor-specific alterations. The LungSelect product identifies actionable genetic changes in plasma from non-small cell lung cancer patients, while PGDx elio plasma resolve is a diagnostic test that detects various genetic alterations in circulating cell-free DNA. Additionally, the company offers RNAcomplete, which allows for the extraction of total RNA and genomic DNA from single tissue samples, and CancerXOME, which analyzes the coding regions of numerous genes. Personal Genome Diagnostics collaborates with institutions like Mayo Clinic and has partnerships with KingMed Diagnostics to further enhance cancer genome research and diagnostics.

Senti Bio, established in 2016 and headquartered in South San Francisco, specializes in synthetic biotechnology for therapeutic development. The company's core focus is engineering gene circuits, a platform technology that enables precise control over cellular behavior. Senti Bio's innovative approach allows for the creation of therapies with enhanced specificity and control, particularly in oncology. Their product pipeline includes SENTI-202, an off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A, a treatment for hepatocellular carcinoma.

Resilience, established in 2020 and headquartered in La Jolla, California, is a biopharmaceutical manufacturing and technology company. It specializes in providing end-to-end manufacturing and development solutions for complex medicines, including biologics, vaccines, nucleic acids, and cell and gene therapies. Resilience invests in developing new manufacturing technologies to ensure rapid, safe, and scalable production, thereby enhancing patient access to these treatments. The company partners with biopharmaceutical companies to focus on breakthrough discoveries while Resilience manages the manufacturing process, safeguarding supply chains against future disruptions.

Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

Polyneuron Pharmaceuticals AG is a biotechnology company based in Basel, Switzerland, founded in 2014. The company focuses on developing innovative therapeutics for antibody-mediated autoimmune disorders, particularly those affecting the peripheral nervous system. Its lead candidates include PN-1007 for anti-MAG neuropathy, PN-1018 for multifocal motor neuropathy, and PN-032 and PN-056 for ABO-incompatible transplantation. Polyneuron employs a unique platform technology called Antibody-Catch, which utilizes biodegradable glycopolymers to selectively target and eliminate pathogenic autoantibodies while preserving the overall integrity of the immune system. This approach aims to provide safe and effective treatment options for patients suffering from these complex immune disorders.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

MBX Biosciences, Inc. is a biotechnology company based in Carmel, Indiana, specializing in the development of therapeutics for rare endocrine disorders. Established in 2018, the company focuses on creating peptides aimed at treating genetic diseases that affect glandular hormones, which play a crucial role in the body’s chemical messaging system. By targeting conditions with inadequate treatment options, MBX Biosciences is dedicated to discovering, developing, and commercializing innovative endocrine therapeutics.

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing innovative therapies for patients suffering from classical complement-mediated disorders affecting the body, brain, and eye. The company's research is centered around its platform technology that targets classical complement-mediated autoimmune and neurodegenerative diseases associated with the abnormal activation of C1q, a key molecule in the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody intended for intravenous use in treating autoimmune and neurodegenerative disorders. Another candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration aimed at neurodegenerative ophthalmic conditions. The company employs a disciplined, biomarker-driven development strategy to ensure its therapies effectively engage the target at tolerable doses within the intended patient tissues, while also exploring additional indications within both orphan and larger market segments.

Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

Amplyx Pharmaceuticals is a small molecule drug development company focused on creating innovative therapies for life-threatening infections, particularly in patients with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in developing oral and injectable anti-infective drugs, including antifungal agents aimed at treating invasive fungal infections caused by pathogens such as Candida and Aspergillus. Amplyx employs a unique platform approach that enhances the efficacy and reduces the toxicity of existing drugs by combining them with additional small molecules. This strategy addresses critical treatment needs in hospital settings and provides effective therapy options for patients after discharge, particularly those undergoing chemotherapy and other immunosuppressive treatments.

Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.

Tempus, established in 2015 and based in Chicago, Illinois, is a technology company specializing in precision medicine. It develops a comprehensive library of molecular and clinical data, along with an operating system that makes this information accessible and useful for patients, physicians, and researchers. Tempus offers services such as genomic sequencing, clinical data restructuring, and biological modeling, enabling real-time data-driven decisions for personalized patient care and facilitating the discovery of optimized therapeutic options.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies for patients with significant unmet medical needs, particularly in fibrosis, lysosomal storage disorders, and oncology. The company specializes in drugs that target nuclear receptors, transcription factors, and epigenetic modulation. Its primary product candidate, lanifibranor, is being developed for the treatment of non-alcoholic steatohepatitis (NASH), a condition currently lacking approved therapies. Inventiva has established a robust pipeline supported by a proprietary discovery engine, a dedicated research and development facility, and a skilled team with extensive experience in drug development. In addition to advancing lanifibranor, the company is also working on several earlier-stage programs aimed at addressing various medical conditions. All operations are conducted in France.

Geron Corporation is a late-stage clinical biopharmaceutical company headquartered in Menlo Park, California, that specializes in developing therapies for hematologic myeloid malignancies. The company's lead product, imetelstat, is a first-in-class telomerase inhibitor currently undergoing Phase 2/3 clinical trials. This investigational drug aims to inhibit the uncontrolled proliferation of malignant progenitor cells, which can lead to dysfunctional blood cell production, thereby facilitating the recovery of normal blood cell development. Founded in 1990, Geron focuses on advancing treatments for conditions such as myelodysplastic syndromes and myelofibrosis, a rare blood cancer affecting bone marrow. The company also generates revenue through collaboration agreements, milestones, royalties, and licensing arrangements.

ERYtech Pharma specializes in the encapsulation of therapeutic molecules and enzymes in red blood cells. It has achieved a major breakthrough in the fields of cancer and enzyme therapy by significantly increasing the therapeutic index of the encapsulated molecule or enzyme.

ChromaCode, Inc. is a molecular diagnostics company based in Carlsbad, California, founded in 2012. The company specializes in developing innovative testing solutions for infectious diseases, cancer, and non-invasive prenatal testing (NIPT) by utilizing patented mathematical methods and algorithmic enhancements. ChromaCode's products significantly enhance the multiplexing capabilities of quantitative polymerase chain reaction (qPCR) instruments, allowing laboratories to perform cost-effective testing of multiple targets from a single sample. Their offerings include software, reagents, and assays that improve signal processing and overall assay performance, making diagnostics more accessible and efficient. Among their notable products is the HDPCR Tick-Borne Pathogen Panel, a multiplex test that detects various tick-borne pathogens in one assay.

Black Diamond Therapeutics, Inc., established in 2014 and headquartered in Cambridge, Massachusetts, specializes in precision oncology medicine. The company discovers and develops small molecule therapies that are tumor-agnostic, meaning they target specific genetic mutations across various cancer types rather than being limited to a single cancer indication. Its lead product candidate, BDTX-189, inhibits non-canonical and oncogenic mutations of ErbB kinases epidermal growth factor receptor (EGFR). The company's Mutation-Allostery-Pharmacology platform enables it to target undrugged mutations in patients with genetically defined cancers, offering new treatment options where few exist. Black Diamond Therapeutics is also advancing early-stage pipeline programs targeting allosteric mutations in other cancer-related kinases.

Shape Therapeutics, Inc. is a biotechnology company founded in 2018 and based in Seattle, Washington. The company specializes in RNA-editing gene therapy targeting neurodegenerative disorders, oncology, metabolic conditions, and rare genetic diseases. Its proprietary RNAfix technology enables precise editing of RNA using the body's natural cellular mechanisms, which minimizes risks associated with immunogenicity, cellular toxicity, and off-target DNA editing. Shape Therapeutics employs a comprehensive approach to RNA gene therapy, integrating innovative payload, delivery, and manufacturing platform technologies. This strategy aims to facilitate the development of effective treatments and potential cures for a wide range of diseases, with the goal of enhancing patient outcomes across the healthcare landscape.

Arcellx is a clinical-stage biotechnology company focused on developing innovative cell therapies for patients with cancer and other incurable diseases. The company aims to enhance the safety, effectiveness, and accessibility of these therapies. Its lead product candidate, CART-ddBCMA, targets relapsed or refractory multiple myeloma and is currently undergoing a Phase 1 clinical study. This therapy has received multiple designations from the U.S. Food and Drug Administration, including Fast Track and Orphan Drug status. Additionally, Arcellx is advancing its controllable CAR-T therapy, ARC-SparX, through two clinical programs aimed at treating relapsed or refractory multiple myeloma and acute myeloid leukemia.

Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies for patients with significant unmet medical needs, particularly in fibrosis, lysosomal storage disorders, and oncology. The company specializes in drugs that target nuclear receptors, transcription factors, and epigenetic modulation. Its primary product candidate, lanifibranor, is being developed for the treatment of non-alcoholic steatohepatitis (NASH), a condition currently lacking approved therapies. Inventiva has established a robust pipeline supported by a proprietary discovery engine, a dedicated research and development facility, and a skilled team with extensive experience in drug development. In addition to advancing lanifibranor, the company is also working on several earlier-stage programs aimed at addressing various medical conditions. All operations are conducted in France.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cells for the treatment of cancer and autoimmune diseases. The company aims to enhance the efficacy of cell therapy by leveraging the inherent ability of NK cells to identify and eliminate abnormal cells without the need for genetic alteration. This approach allows for a broader application across various hematologic and solid tumor malignancies while potentially minimizing side effects associated with traditional T-cell therapies. Nkarta combines its NK expansion platform technology with proprietary cell engineering methods to produce a substantial supply of NK cells, enhancing their ability to recognize therapeutic targets and improving their persistence in the body for sustained activity. Through this innovative strategy, Nkarta seeks to provide more potent, well-tolerated, and rapidly available therapies for patients facing diverse cancer types.

Sagimet Biosciences is a clinical-stage biopharmaceutical company based in San Mateo, California, that specializes in developing novel therapeutics for oncology and infectious diseases. The company focuses on targeting dysfunctional metabolic pathways, particularly through its expertise in fatty acid synthase (FASN) biology. Its lead drug candidate, denifanstat, is a selective FASN inhibitor designed for the treatment of nonalcoholic steatohepatitis (NASH) and is administered as an oral, once-daily pill. Additionally, Sagimet is working on antiviral therapeutics aimed at addressing respiratory syncytial virus, cytomegalovirus, and other human pathogens. Originally known as 3-V Biosciences, Inc., the company rebranded to Sagimet Biosciences in August 2019 and has been active since its incorporation in 2006.

Tempus, established in 2015 and based in Chicago, Illinois, is a technology company specializing in precision medicine. It develops a comprehensive library of molecular and clinical data, along with an operating system that makes this information accessible and useful for patients, physicians, and researchers. Tempus offers services such as genomic sequencing, clinical data restructuring, and biological modeling, enabling real-time data-driven decisions for personalized patient care and facilitating the discovery of optimized therapeutic options.

Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.

Polyneuron Pharmaceuticals AG is a biotechnology company based in Basel, Switzerland, founded in 2014. The company focuses on developing innovative therapeutics for antibody-mediated autoimmune disorders, particularly those affecting the peripheral nervous system. Its lead candidates include PN-1007 for anti-MAG neuropathy, PN-1018 for multifocal motor neuropathy, and PN-032 and PN-056 for ABO-incompatible transplantation. Polyneuron employs a unique platform technology called Antibody-Catch, which utilizes biodegradable glycopolymers to selectively target and eliminate pathogenic autoantibodies while preserving the overall integrity of the immune system. This approach aims to provide safe and effective treatment options for patients suffering from these complex immune disorders.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing and commercializing therapeutics for rare genetic disorders of hemoglobin, including sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing its lead product candidate, IMR-687, which is a highly selective small molecule inhibitor of PDE9. IMR-687 is designed as an oral, once-a-day treatment that aims to modify disease through a multimodal mechanism of action affecting various cell types, including red and white blood cells. Imara is dedicated to addressing the unmet medical needs of patients suffering from these serious hemoglobinopathies.

Oyster Point Pharma, Inc., established in 2015 and headquartered in Princeton, New Jersey, is a clinical-stage biopharmaceutical company dedicated to developing and commercializing innovative therapies for ocular surface diseases. Its lead product candidate, OC-01, is a nasal spray formulated as a highly selective nicotinic acetylcholine receptor (nAChR) agonist, currently in Phase III trials for treating the signs and symptoms of dry eye disease, a chronic condition affecting over 30 million Americans. OC-01's novel mechanism of action stimulates tear production by activating the trigeminal parasympathetic pathway, re-establishing tear film homeostasis.

Black Diamond Therapeutics, Inc., established in 2014 and headquartered in Cambridge, Massachusetts, specializes in precision oncology medicine. The company discovers and develops small molecule therapies that are tumor-agnostic, meaning they target specific genetic mutations across various cancer types rather than being limited to a single cancer indication. Its lead product candidate, BDTX-189, inhibits non-canonical and oncogenic mutations of ErbB kinases epidermal growth factor receptor (EGFR). The company's Mutation-Allostery-Pharmacology platform enables it to target undrugged mutations in patients with genetically defined cancers, offering new treatment options where few exist. Black Diamond Therapeutics is also advancing early-stage pipeline programs targeting allosteric mutations in other cancer-related kinases.

Tiburio Therapeutics, Inc. is a biotechnology company focused on developing and manufacturing medicines for diseases related to the endocrine system. Founded in 2018 and based in Cambridge, Massachusetts, the company is advancing clinical-stage compounds aimed at treating rare neuroendocrine tumors and endocrine diseases. Notably, Tiburio is developing TBR-760, which targets non-functioning pituitary adenoma using a dopamine-somatostatin chimeric molecule designed to shrink or halt tumor growth. Additionally, Tiburio is working on TBR-065 for other rare endocrine diseases, addressing significant patient needs in these underserved areas.

Curzion Pharmaceuticals is a privately held development-stage biopharma company, and its development-stage oral selective lysophosphatidic acid 1 receptor (LPAR1) antagonist, CZN001 (renamed HZN-825).

Xenon Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company based in Burnaby, Canada, focused on developing therapeutics for neurological disorders. The company's clinical pipeline includes several product candidates aimed at treating epilepsy, such as XEN496 and XEN1101, both Kv7 potassium channel modulators currently in Phase II trials, and XEN901, a selective Nav1.6 sodium channel inhibitor in Phase I trials. Additionally, Xenon is developing XEN007, a central nervous system-acting calcium channel modulator, also in Phase I trials. The company leverages insights from genetic research, particularly from families with severe phenotypes, to identify drug targets, which may extend beyond rare genetic disorders. Furthermore, Xenon has established a collaboration with Neurocrine Biosciences to advance the development of innovative epilepsy treatments.

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing innovative therapies for patients suffering from classical complement-mediated disorders affecting the body, brain, and eye. The company's research is centered around its platform technology that targets classical complement-mediated autoimmune and neurodegenerative diseases associated with the abnormal activation of C1q, a key molecule in the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody intended for intravenous use in treating autoimmune and neurodegenerative disorders. Another candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration aimed at neurodegenerative ophthalmic conditions. The company employs a disciplined, biomarker-driven development strategy to ensure its therapies effectively engage the target at tolerable doses within the intended patient tissues, while also exploring additional indications within both orphan and larger market segments.

Mirum Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing therapies for serious liver diseases. Founded in 2018 and based in Foster City, California, the company focuses on addressing rare and orphan diseases. Its lead product candidate, maralixibat, is an investigational oral drug currently in Phase 3 clinical trials for treating progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome (ALGS), and biliary atresia. Additionally, Mirum is developing volixibat, aimed at treating intrahepatic cholestasis of pregnancy and primary sclerosing cholangitis. The company’s commitment to innovative therapies positions it as a key player in the biopharmaceutical landscape dedicated to improving the lives of patients with debilitating liver conditions.

Qpex Biopharma is a San Diego-based biopharmaceutical company founded in 2018, focused on developing innovative therapies to address the growing challenge of antimicrobial resistance. The company specializes in creating a pipeline of antibiotic drugs aimed at treating infectious diseases in both inpatient and outpatient settings. With a strong emphasis on the discovery, development, and regulatory approval of anti-infective medicines, Qpex Biopharma leverages its expertise and experience in collaboration with public and private partnerships to meet critical medical needs. The company's mission is to provide effective solutions for healthcare professionals facing the complexities of resistant infections, thereby enhancing treatment options for patients.

Galera Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in Malvern, Pennsylvania, dedicated to developing innovative therapeutics aimed at enhancing the effectiveness of radiotherapy for cancer patients. The company's lead product candidate, avasopasem manganese (GC4419), is a small molecule dismutase mimetic currently undergoing Phase III clinical trials for the treatment of radiation-induced severe oral mucositis in patients with head and neck cancer. Additionally, GC4419 is being evaluated in Phase IIa trials for the treatment of radiotherapy-induced esophagitis in lung cancer patients. Galera is also advancing another candidate, GC4711, which is in Phase I/II trials for pancreatic and lung cancers. The company's research is focused on targeting oxygen metabolic pathways to improve patient outcomes in radiation therapy, and GC4419 has received Fast Track and Breakthrough Therapy designations from the U.S. Food and Drug Administration.

PhaseBio Pharmaceuticals, Inc., established in 2002 and based in Malvern, Pennsylvania, is a clinical-stage biopharmaceutical company dedicated to developing novel therapies for cardiopulmonary diseases. Its primary focus is on PB2452, a reversal agent for the antiplatelet drug ticagrelor, currently in Phase III trials for managing uncontrolled bleeding events or urgent surgeries. Additionally, PhaseBio is developing PB1046, a fusion protein in Phase IIb trials for treating pulmonary arterial hypertension, and PB6440, an oral agent for resistant hypertension. The company's proprietary technology, elastin-like polypeptides (ELPs), enhances the stability, bioavailability, and efficacy of proteins and peptides, aiming to improve patient outcomes and reduce side effects.

Tempus, established in 2015 and based in Chicago, Illinois, is a technology company specializing in precision medicine. It develops a comprehensive library of molecular and clinical data, along with an operating system that makes this information accessible and useful for patients, physicians, and researchers. Tempus offers services such as genomic sequencing, clinical data restructuring, and biological modeling, enabling real-time data-driven decisions for personalized patient care and facilitating the discovery of optimized therapeutic options.

Millendo Therapeutics is a biopharmaceutical company dedicated to developing innovative treatments for endocrine diseases resulting from hormone dysregulation. The company focuses on creating distinct and transformative therapies that address significant unmet medical needs in this area. By leveraging scientific advancements, Millendo aims to develop novel compounds that can effectively treat complex conditions, ultimately enhancing clinical care and improving the quality of life for patients, families, and caregivers.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

Catalog Technologies Inc. specializes in a DNA-based platform that revolutionizes digital data storage and computation. Founded in 2016 and headquartered in Boston, Massachusetts, the company provides a method for encoding digital information into DNA, making it a viable and cost-effective solution for long-term data archiving. By leveraging advanced synthetic biology technologies, Catalog aims to transform how information is stored, allowing customers to utilize DNA molecules as a primary medium for digital records. This innovative approach positions Catalog at the forefront of next-generation data storage solutions.

Metacrine, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing innovative therapies for liver and gastrointestinal diseases. The company is advancing its lead programs, which target the farnesoid X receptor (FXR), a key drug target in these conditions. Currently, MET409 is undergoing a Phase Ib proof-of-concept clinical trial for patients with non-alcoholic steatohepatitis, while MET642 is in a Phase 1 clinical trial for the same patient population. In addition to its FXR-focused programs, Metacrine is exploring a pipeline of novel drug candidates through its internal research and development capabilities. The company also collaborates with Novo Nordisk A/S to further research related to fibroblast growth factor 1. Metacrine was founded in 2014 and is committed to addressing unmet medical needs in liver and gastrointestinal health.

Tempus, established in 2015 and based in Chicago, Illinois, is a technology company specializing in precision medicine. It develops a comprehensive library of molecular and clinical data, along with an operating system that makes this information accessible and useful for patients, physicians, and researchers. Tempus offers services such as genomic sequencing, clinical data restructuring, and biological modeling, enabling real-time data-driven decisions for personalized patient care and facilitating the discovery of optimized therapeutic options.

Senti Bio, established in 2016 and headquartered in South San Francisco, specializes in synthetic biotechnology for therapeutic development. The company's core focus is engineering gene circuits, a platform technology that enables precise control over cellular behavior. Senti Bio's innovative approach allows for the creation of therapies with enhanced specificity and control, particularly in oncology. Their product pipeline includes SENTI-202, an off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A, a treatment for hepatocellular carcinoma.

Liquidia Technologies is a late-stage clinical biopharmaceutical company based in the United States, dedicated to developing and commercializing innovative therapeutics. Utilizing its proprietary PRINT technology, a particle engineering platform, Liquidia produces uniform drug particles aimed at enhancing the safety and efficacy of various therapies. The company is advancing two key product candidates: LIQ861, intended for the treatment of pulmonary arterial hypertension, and LIQ865, designed for managing local post-operative pain. Additionally, Liquidia collaborates with leading pharmaceutical firms to leverage its PRINT technology across multiple therapeutic areas, molecule types, and administration routes, addressing unmet patient needs, particularly in the realm of pulmonary hypertension.

Xoc Pharmaceuticals, Inc. is a biotechnology company based in Los Gatos, California, founded in 2013. The company focuses on developing novel oral therapies aimed at migraine prevention and the management of Parkinson's disease through dopamine agonism. Utilizing advanced receptor characterization technology and innovative medicinal chemistry, Xoc Pharmaceuticals identifies product candidates that enhance safety and efficacy for patients. The company's development strategy involves targeted molecular modifications and optimized delivery methods, which aim to improve the therapeutic outcomes for individuals with neurological disorders. Additionally, Xoc Pharmaceuticals effectively manages clinical and regulatory risks by leveraging the pharmacological history of compounds similar to its product candidates.

CTI BioPharma Corp. is a biopharmaceutical company headquartered in Seattle, Washington, that specializes in the acquisition, development, and commercialization of targeted therapies for blood-related cancers. Founded in 1991 and formerly known as Cell Therapeutics, Inc., the company is primarily focused on developing novel treatments to improve patient outcomes in oncology. Its key investigational product, pacritinib, is an oral kinase inhibitor currently in Phase III clinical trials for the treatment of myelofibrosis in adults. In addition to pacritinib, CTI BioPharma's portfolio includes other candidates like Pixantrone, which is undergoing Phase III trials for non-Hodgkin's lymphoma, and Brostallicin, currently in a Phase II study for sarcoma. The company has established collaboration and licensing agreements with several pharmaceutical partners.

Personal Genome Diagnostics, Inc. specializes in patient-specific cancer genome analysis using advanced digital characterization and monitoring technologies. Founded in 2010 and based in Baltimore, Maryland, the company offers a range of products and services aimed at improving cancer diagnosis and treatment. Their METDetect Assay detects MET gene amplifications in cancer patients' circulation, providing detailed reports on tumor-specific alterations. The LungSelect product identifies actionable genetic changes in plasma from non-small cell lung cancer patients, while PGDx elio plasma resolve is a diagnostic test that detects various genetic alterations in circulating cell-free DNA. Additionally, the company offers RNAcomplete, which allows for the extraction of total RNA and genomic DNA from single tissue samples, and CancerXOME, which analyzes the coding regions of numerous genes. Personal Genome Diagnostics collaborates with institutions like Mayo Clinic and has partnerships with KingMed Diagnostics to further enhance cancer genome research and diagnostics.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

Pionyr Immunotherapeutics Inc. is a biotechnology company based in San Francisco, California, that focuses on developing innovative cancer immunotherapies. Founded in 2015, the company aims to enhance the body's antitumor immunity by targeting the tumor microenvironment. Utilizing advanced target discovery and antibody generation technologies, Pionyr has created a unique approach called Myeloid Tuning™, which modifies the cellular infiltrate within tumors to improve the immune system's response against cancer. This strategy is intended to be particularly effective when used in conjunction with checkpoint inhibitors, offering new avenues for cancer treatment.

Metacrine, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing innovative therapies for liver and gastrointestinal diseases. The company is advancing its lead programs, which target the farnesoid X receptor (FXR), a key drug target in these conditions. Currently, MET409 is undergoing a Phase Ib proof-of-concept clinical trial for patients with non-alcoholic steatohepatitis, while MET642 is in a Phase 1 clinical trial for the same patient population. In addition to its FXR-focused programs, Metacrine is exploring a pipeline of novel drug candidates through its internal research and development capabilities. The company also collaborates with Novo Nordisk A/S to further research related to fibroblast growth factor 1. Metacrine was founded in 2014 and is committed to addressing unmet medical needs in liver and gastrointestinal health.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.

Oyster Point Pharma, Inc., established in 2015 and headquartered in Princeton, New Jersey, is a clinical-stage biopharmaceutical company dedicated to developing and commercializing innovative therapies for ocular surface diseases. Its lead product candidate, OC-01, is a nasal spray formulated as a highly selective nicotinic acetylcholine receptor (nAChR) agonist, currently in Phase III trials for treating the signs and symptoms of dry eye disease, a chronic condition affecting over 30 million Americans. OC-01's novel mechanism of action stimulates tear production by activating the trigeminal parasympathetic pathway, re-establishing tear film homeostasis.

Cydan is an orphan drug accelerator focused on developing therapies for patients with rare genetic diseases. The organization identifies and mitigates the risks associated with compounds that have therapeutic and commercial potential. Through a rigorous process, Cydan generates data to support the development of new therapies and the formation of new companies. Their experienced team leverages strong relationships with academia and patient advocacy groups, along with deep expertise in drug development and a proven track record of successful product commercialization. By guiding therapies through clinical, regulatory, and commercial development, Cydan aims to improve the lives of patients and families affected by rare diseases.