New Enterprise Associates

Zenas BioPharma is a biopharmaceutical company that focuses on developing immune-based therapies for patients. The company is headquartered in Florida, USA.

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.

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 focuses on the discovery and development of innovative therapeutic products for unmet medical needs. Founded by former executives from Receptos, the company aims to leverage a strong in-licensing strategy and a team with extensive experience in immunology, inflammation, fibrosis, and oncology. Gossamer Bio's pipeline includes several promising candidates: Seralutinib, which targets pulmonary arterial hypertension; GB004, aimed at treating inflammatory bowel diseases such as ulcerative colitis and Crohn's disease; GB1275, designed for various oncology indications; and GB001, intended for moderate-to-severe eosinophilic asthma. Through these efforts, Gossamer Bio seeks to improve patient outcomes in critical therapeutic areas.

Tagworks Pharmaceuticals is a privately held biotech company based in the Netherlands. Tagworks develops In vivo chemistry to improve the efficacy of cancer therapies and to enable novel approaches in companion diagnostics. The company’s facilities are located at the Radboud University Medical Center in Nijmegen. Founded in 2011, Tagworks is a spin-out of Philips Healthcare, and originates from Philips’ molecular imaging expertise.

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 operates as an antibody-focused precision oncology company.

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 focuses on the discovery and development of innovative therapeutic products for unmet medical needs. Founded by former executives from Receptos, the company aims to leverage a strong in-licensing strategy and a team with extensive experience in immunology, inflammation, fibrosis, and oncology. Gossamer Bio's pipeline includes several promising candidates: Seralutinib, which targets pulmonary arterial hypertension; GB004, aimed at treating inflammatory bowel diseases such as ulcerative colitis and Crohn's disease; GB1275, designed for various oncology indications; and GB001, intended for moderate-to-severe eosinophilic asthma. Through these efforts, Gossamer Bio seeks to improve patient outcomes in critical therapeutic areas.

Senti Biosciences is a clinical-stage biotechnology company specializing in synthetic biology and gene circuit engineering for therapeutic purposes. The company employs its Gene Circuits platform to develop innovative therapies that enhance precision in targeting cancer cells while avoiding damage to healthy cells. Senti Bio's approach involves the use of allogeneic chimeric antigen receptor natural killer (CAR-NK) cells that incorporate its gene circuit technologies, aimed at addressing various oncology indications. Among its product candidates are SENTI-202, a Logic Gated off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells without harming healthy bone marrow, and SENTI-301A, which targets hepatocellular carcinoma. Senti Biosciences aims to provide effective treatment options for difficult-to-treat diseases through advanced cell and gene therapies.

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.

Sorriso Pharmaceuticals is a biotechnology company advancing a pipeline of disease-modifying antibodies for the treatment of inflammatory disease, including Crohn’s disease and ulcerative colitis. The Sorriso platform generates potent antibodies that can be delivered orally and are designed to maintain activity throughout the intestinal system. The company was founded in 2020 and is based in San Diego, California.

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.

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.

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.

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.

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.

Resilience is a technology-driven biomanufacturing company focused on enhancing access to complex medicines, including biologics, vaccines, nucleic acids, and cell and gene therapies. The firm aims to transform the production of medicine by providing a comprehensive range of customized manufacturing capabilities that cater to the diverse needs of biopharmaceuticals throughout all stages of drug development, from pre-clinical to commercial supply. Resilience is committed to creating a sustainable network of advanced manufacturing solutions that ensure rapid, safe, and scalable production of treatments, thereby enabling partners to concentrate on breakthrough discoveries that improve patient outcomes. The company's innovative approach also strengthens biopharmaceutical supply chains, preparing them for future challenges.

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. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing nucleic acid-based therapeutics aimed at editing RNA to treat rare and prevalent diseases. Founded in 2018, the company has created a proprietary platform that allows for efficient and selective RNA editing, leveraging natural processes found in multicellular organisms. This innovative approach enables precise modifications to messenger RNA, allowing for targeted changes in protein structure and function across various tissues. By focusing on RNA instead of DNA, Korro Bio aims to enhance the precision and adaptability of genetic medicines, expanding their potential applications in treating a range of medical conditions.

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 pre-clinical stage company focusing on the development of small molecule drugs with enhanced efficacy and lower toxicity. Amplyx employs a platform approach to improve small molecule drugs by the addition of a second small molecule to an existing drug. The company develops innovative therapies for debilitating and life-threatening diseases that affect people with compromised immune systems. Amplyx Pharmaceuticals was founded in 2006 and is headquartered in San Diego, California.

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 is a technology company improving precision medicine through the practical application of artificial intelligence in healthcare. Their platform is designed to analyze and interpret clinical and molecular data to help healthcare providers deliver more personalized and effective treatments.

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.

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.

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.

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 is a precision oncology medicine company focused on discovering and developing small molecule therapies that are tumor-agnostic. The company’s lead product candidate, BDTX-189, targets non-canonical and oncogenic mutations in ErbB kinases, specifically the epidermal growth factor receptor and tyrosine-protein kinase. In addition to BDTX-189, Black Diamond is developing allosteric-EGFR mutation inhibitors and other early-stage pipeline programs that address allosteric mutations in kinases associated with cancer and rare genetic diseases. The company's approach is centered on its Mutation-Allostery-Pharmacology platform, which aims to create targeted treatments for genetically defined cancers, especially for patients with limited existing treatment options. Founded in 2014 and headquartered in Cambridge, Massachusetts, Black Diamond Therapeutics was previously known as ASET Therapeutics until its name change in January 2018.

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.

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 is a technology company improving precision medicine through the practical application of artificial intelligence in healthcare. Their platform is designed to analyze and interpret clinical and molecular data to help healthcare providers deliver more personalized and effective treatments.

Inozyme is a provider of therapies for rare diseases of calcification affecting soft tissues and bone created to offer potentially disease-modifying therapies to help children who are affected with rare, but severe and debilitating disorders of metabolism. The company's enzyme replacement therapy provides the treatment of Generalized Arterial Calcification of Infancy (GACI) and Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2), enabling patients to treat diseases characterized by mineral imbalances in the body.

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 is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for ocular surface diseases. The company's primary focus is on its lead product candidate, OC-01, a nasal spray that acts as a selective nicotinic acetylcholine receptor agonist. OC-01 aims to address the signs and symptoms of dry eye disease, a chronic condition that affects over 30 million Americans. By activating the trigeminal parasympathetic pathway, OC-01 seeks to restore tear film homeostasis and stimulate the natural production of tears through the Lacrimal Functional Unit.

Black Diamond Therapeutics is a precision oncology medicine company focused on discovering and developing small molecule therapies that are tumor-agnostic. The company’s lead product candidate, BDTX-189, targets non-canonical and oncogenic mutations in ErbB kinases, specifically the epidermal growth factor receptor and tyrosine-protein kinase. In addition to BDTX-189, Black Diamond is developing allosteric-EGFR mutation inhibitors and other early-stage pipeline programs that address allosteric mutations in kinases associated with cancer and rare genetic diseases. The company's approach is centered on its Mutation-Allostery-Pharmacology platform, which aims to create targeted treatments for genetically defined cancers, especially for patients with limited existing treatment options. Founded in 2014 and headquartered in Cambridge, Massachusetts, Black Diamond Therapeutics was previously known as ASET Therapeutics until its name change in January 2018.

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.

Qpex Biopharma develops a pipeline of agents addressing critical needs for the treatment of infectious diseases. It develops multiple antibiotic drugs intended to combat antimicrobial resistance. The company's technology is focused on targeting the treatment of infectious diseases for both inpatient and outpatient cases, deep expertise in the discovery, development and regulatory approval of anti-infective medicines, including an extensive record of working with public/private partnerships focused on new antibiotics to combat antimicrobial resistance, enabling medical businesses optimal use of products and critical needs for the treatment of infectious diseases in the inpatient and outpatient settings. It was founded in 2018 and is headquartered in San Diego, California.

PhaseBio is a clinical-stage biopharmaceutical company focused on developing innovative therapies for endocrine and metabolic disorders, as well as cardiovascular diseases. The company leverages its proprietary elastin-like polypeptides (ELPs) technology to enhance the stability, bioavailability, and efficacy of proteins and peptides. Its lead candidate, PB1023, is in a phase 2b clinical trial for managing hyperglycemia in type 2 diabetes, with potential applications in obesity. Additionally, PhaseBio is advancing Vasomera, a candidate in phase 1 trials aimed at treating pulmonary arterial hypertension and heart failure. The company is also exploring PB2452, a novel reversal agent for the antiplatelet drug ticagrelor, and PB1046 for pulmonary arterial hypertension. With a commitment to addressing unmet medical needs, PhaseBio is headquartered in Malvern, Pennsylvania.

Tempus is a technology company improving precision medicine through the practical application of artificial intelligence in healthcare. Their platform is designed to analyze and interpret clinical and molecular data to help healthcare providers deliver more personalized and effective treatments.

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

Catalog is a developer of a data conversion and storage platform used to offer the next generation of digital data archives and computation. Its platform facilitates the encoding of data and information into DNA format and makes it economically attractive to use DNA as the major medium for long-term archival of data that enables customers to store digital information in DNA molecules. It brings cutting-edge synthetic biology technologies to the world of information storage and computation. The company was founded in 2016 and is headquartered in Boston, Massachusetts.

Metacrine is a clinical-stage biopharmaceutical company focused on building an innovative pipeline of best-in-class drugs to treat liver and gastrointestinal (GI) diseases. The most advanced program is focused on the farnesoid X receptor (FXR) an important drug target in multiple liver and GI diseases. Beyond the FXR program, a pipeline of novel drug candidates against other drug targets is being explored by taking advantage of internal drug discovery and development capabilities.

Tempus is a technology company improving precision medicine through the practical application of artificial intelligence in healthcare. Their platform is designed to analyze and interpret clinical and molecular data to help healthcare providers deliver more personalized and effective treatments.

Senti Biosciences is a clinical-stage biotechnology company specializing in synthetic biology and gene circuit engineering for therapeutic purposes. The company employs its Gene Circuits platform to develop innovative therapies that enhance precision in targeting cancer cells while avoiding damage to healthy cells. Senti Bio's approach involves the use of allogeneic chimeric antigen receptor natural killer (CAR-NK) cells that incorporate its gene circuit technologies, aimed at addressing various oncology indications. Among its product candidates are SENTI-202, a Logic Gated off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells without harming healthy bone marrow, and SENTI-301A, which targets hepatocellular carcinoma. Senti Biosciences aims to provide effective treatment options for difficult-to-treat diseases through advanced cell and gene therapies.

Liquidia Technologies is a clinical biopharmaceutical company dedicated to developing and commercializing therapeutics that address unmet patient needs, primarily through its proprietary PRINT technology. This particle engineering platform allows for the precise production of uniform drug particles, enhancing the safety and efficacy of various treatments. The company is currently advancing two key product candidates: LIQ861, aimed at treating pulmonary arterial hypertension, and LIQ865, designed for managing local post-operative pain. In addition to its internal pipeline, Liquidia collaborates with leading pharmaceutical companies to leverage its PRINT technology across diverse therapeutic areas and drug types. One of its marketed products is YUTREPIA, an inhalation powder for treating pulmonary arterial hypertension.

CTI BioPharma Corp. is a biopharmaceutical company headquartered in Seattle, Washington, that specializes in the development and commercialization of targeted therapies for blood-related cancers. The company is advancing its lead product, pacritinib, an investigational oral kinase inhibitor currently in Phase III clinical trials for myelofibrosis, a type of bone marrow cancer. In addition to pacritinib, CTI BioPharma's development portfolio includes Pixantrone, which is in a Phase III trial for non-Hodgkin's lymphoma, and Brostallicin, currently undergoing a Phase II study for sarcoma. The company has established collaboration and licensing agreements with various pharmaceutical firms, including Teva Pharmaceutical Industries Ltd. and Novartis International Pharmaceutical, Ltd. Founded in 1991, CTI BioPharma aims to deliver innovative and less toxic cancer therapies to patients.

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. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing nucleic acid-based therapeutics aimed at editing RNA to treat rare and prevalent diseases. Founded in 2018, the company has created a proprietary platform that allows for efficient and selective RNA editing, leveraging natural processes found in multicellular organisms. This innovative approach enables precise modifications to messenger RNA, allowing for targeted changes in protein structure and function across various tissues. By focusing on RNA instead of DNA, Korro Bio aims to enhance the precision and adaptability of genetic medicines, expanding their potential applications in treating a range of medical conditions.

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 is a clinical-stage biopharmaceutical company focused on building an innovative pipeline of best-in-class drugs to treat liver and gastrointestinal (GI) diseases. The most advanced program is focused on the farnesoid X receptor (FXR) an important drug target in multiple liver and GI diseases. Beyond the FXR program, a pipeline of novel drug candidates against other drug targets is being explored by taking advantage of internal drug discovery and development capabilities.

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 is a provider of therapies for rare diseases of calcification affecting soft tissues and bone created to offer potentially disease-modifying therapies to help children who are affected with rare, but severe and debilitating disorders of metabolism. The company's enzyme replacement therapy provides the treatment of Generalized Arterial Calcification of Infancy (GACI) and Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2), enabling patients to treat diseases characterized by mineral imbalances in the body.

Oyster Point Pharma is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for ocular surface diseases. The company's primary focus is on its lead product candidate, OC-01, a nasal spray that acts as a selective nicotinic acetylcholine receptor agonist. OC-01 aims to address the signs and symptoms of dry eye disease, a chronic condition that affects over 30 million Americans. By activating the trigeminal parasympathetic pathway, OC-01 seeks to restore tear film homeostasis and stimulate the natural production of tears through the Lacrimal Functional Unit.

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.

Tempus is a technology company improving precision medicine through the practical application of artificial intelligence in healthcare. Their platform is designed to analyze and interpret clinical and molecular data to help healthcare providers deliver more personalized and effective treatments.

Amplyx Pharmaceuticals is a pre-clinical stage company focusing on the development of small molecule drugs with enhanced efficacy and lower toxicity. Amplyx employs a platform approach to improve small molecule drugs by the addition of a second small molecule to an existing drug. The company develops innovative therapies for debilitating and life-threatening diseases that affect people with compromised immune systems. Amplyx Pharmaceuticals was founded in 2006 and is headquartered in San Diego, California.

Complexa, Inc. is a biopharmaceutical company based in Berwyn, Pennsylvania, focused on developing treatments for severe and life-threatening diseases related to fibrosis and inflammation. Established in 2008, the company is advancing its lead compound, CXA-10, an oral nitrated fatty acid aimed at treating focal segmental glomerulosclerosis (FSGS) and pulmonary arterial hypertension (PAH). Complexa utilizes proprietary technologies that involve the synthesis of endogenous nitro-fatty acids, which serve as cell-signaling agents regulating key inflammatory pathways. This approach aims to enhance the body's natural mechanisms for resolving and repairing acute and chronic tissue injuries, with a focus on addressing various inflammatory, fibrotic, and central nervous system diseases.

Catalog is a developer of a data conversion and storage platform used to offer the next generation of digital data archives and computation. Its platform facilitates the encoding of data and information into DNA format and makes it economically attractive to use DNA as the major medium for long-term archival of data that enables customers to store digital information in DNA molecules. It brings cutting-edge synthetic biology technologies to the world of information storage and computation. The company was founded in 2016 and is headquartered in Boston, Massachusetts.

Senseonics Holdings, Inc. is a medical technology company based in Germantown, Maryland, specializing in continuous glucose monitoring (CGM) systems for individuals with diabetes. Founded in 1996, the company focuses on developing innovative implantable CGM products, including the Eversense and Eversense XL systems, which measure glucose levels for up to 90 and 180 days, respectively. These systems utilize advanced fluorescence sensing technology to provide accurate and stable glucose monitoring, allowing users to manage their diabetes effectively. Senseonics primarily generates revenue from markets outside the United States, highlighting its commitment to enhancing the lives of people with diabetes through long-term, differentiated glucose management solutions.

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.

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.

Addex Therapeutics Ltd is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, that specializes in the discovery, development, and commercialization of small-molecule drugs targeting central nervous system (CNS) disorders. The company primarily focuses on allosteric modulators, a novel class of orally available drugs that interact with G-protein coupled receptors, potentially offering advantages over traditional therapeutic approaches. Its lead product candidates include Dipraglurant for treating Parkinson's disease-related dyskinesia and dystonia, ADX71149 for epilepsy and other CNS disorders, and GABAB PAM for addiction. Addex has established a collaboration with Janssen Pharmaceuticals Inc. to develop mGluR2PAM compounds aimed at addressing various health issues. Founded in 2002, Addex Therapeutics continues to advance its innovative drug discovery platform to improve therapeutic outcomes for patients with neurological conditions.

Rhythm is a biopharmaceutical company focused on developing and commercializing peptide therapeutics for the treatment of rare genetic deficiencies that result in life-threatening metabolic disorders. Their lead peptide product candidate, setmelanotide, is a potent, first-in-class melanocortin-4 receptor (MC4R) agonist for the treatment of rare genetic disorders of obesity. They believe that setmelanotide, for which they have exclusive worldwide rights, has the potential to serve as replacement therapy for the treatment of melanocortin 4, or MC4, pathway deficiencies. The MC4 pathway is a compelling target for treating these genetic disorders because of its critical role in regulating appetite and weight, and peptide therapeutics are uniquely suited to activating this target.

Tarveda Therapeutics is a developer of miniature drug conjugates designed for the treatment of patients with a wide range of solid tumors. The company's HSP90 targeted drug conjugate platform with lead drug candidate PEN-866 selectively binds in tumors to the activated form of Heat Shock Protein 90 (HSP90) and releases its potent topoisomerase 1 inhibitor payload, SN38 and it also develops a miniature drug conjugate in clinical evaluation for the treatment of patients with somatostatin receptor 2 (SSTR2) expressing neuroendocrine, small cell lung and other solid tumors, enabling companies to extend the lives of patients with hard to treat cancers while minimizing potential toxicities.

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.

Immunomedics is a biopharmaceutical company that specializes in developing monoclonal antibody-based products for the treatment of cancer and other serious diseases. Its corporate objective is to become a fully-integrated biopharmaceutical company. The company was founded in 1982 and headquartered in Morris Plains, New Jersey.

Trillium Therapeutics Inc. is a clinical-stage immuno-oncology company focused on developing innovative therapies for cancer treatment. The company's lead candidate, TTI-621, is a SIRPaFc fusion protein currently undergoing Phase I clinical trials for advanced relapsed or refractory hematologic malignancies, as well as solid tumors and mycosis fungoides. Another product, TTI-622, is also in Phase I clinical trials and is based on an immunoglobulin G4 SIRPaFc protein. Additionally, Trillium is exploring TTI-10001, a small molecule stimulator of interferon genes agonist in the discovery phase. Founded in 2004 and headquartered in Mississauga, Canada, Trillium Therapeutics has evolved from its previous name, Stem Cell Therapeutics Corp., to focus primarily on harnessing the immune system to combat cancer. The company operates across the United States and Canada, emphasizing the development of targeted protein therapeutics.

Cleave Therapeutics develops oncology pharmaceuticals designed to target neurodengeneration and pathways used by cancer cells to grow. The company is engaged in the discovery of novel, small-molecule drugs that affect protein-degradation pathways, upon which cancer cells depend for survival, enabling patients to get cured from cancer.

Vtesse, Inc. is a biopharmaceutical company based in Gaithersburg, Maryland, focused on developing treatments for patients with rare diseases. The company is conducting clinical studies for VTS-270, aimed at treating Niemann-Pick disease type C (NPC), and is also engaged in pre-clinical discovery and development of additional therapies for NPC and other lysosomal storage diseases. Vtesse originated as the first spin-out from Cydan Development, an orphan-drug accelerator that identifies promising therapeutic programs. The company collaborates with the National Institutes of Health (NIH) to advance its clinical studies and is supported by a consortium of experienced investors. Vtesse's management team has a strong background, having participated in the development of over 20 approved drugs and vaccines, which underscores its commitment to addressing the needs of underserved patient populations.

Ardelyx, Inc. is a biopharmaceutical company specializing in the development and commercialization of innovative oral therapeutics for cardiorenal diseases. Founded in 2007 and headquartered in Fremont, California, the company focuses on addressing significant unmet medical needs through first-in-class medicines. Its lead product candidate, tenapanor, has completed Phase 3 clinical trials for treating irritable bowel syndrome with constipation and hyperphosphatemia in end-stage renal disease patients on dialysis. Additionally, Ardelyx is advancing RDX013, a small molecule potassium secretagogue targeting hyperkalemia, and has other candidates in its pipeline, including RDX002 and RDX009, which are in preclinical development for chronic kidney disease and type 2 diabetes, respectively. Ardelyx employs a proprietary drug discovery platform aimed at creating non-systemic products that target specific transporters and receptors to minimize side effects associated with systemic exposure.

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.

Verona Pharma is a clinical-stage biopharmaceutical company based in London, established in 2005. The company specializes in developing and commercializing therapies for respiratory diseases that have significant unmet medical needs, including chronic obstructive pulmonary disease (COPD), cystic fibrosis, and asthma. Its lead product candidate, ensifentrine, is an inhaled dual inhibitor of the enzymes phosphodiesterase 3 and 4, functioning as both a bronchodilator and an anti-inflammatory agent. Ensifentrine is being developed in various formulations, including nebulized, dry powder inhaler, and pressurized metered-dose inhaler, with the nebulized form currently in Phase 2b clinical development for the maintenance treatment of COPD. Verona Pharma's commitment is to enhance the health and quality of life for individuals suffering from chronic respiratory conditions through innovative therapeutic solutions.

Mersana Therapeutics is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of antibody-drug conjugates (ADCs) for cancer treatment. The company utilizes its proprietary biodegradable polymer platform, Dolaflexin, to create a pipeline of ADC product candidates aimed at addressing oncology indications with significant unmet needs. Its lead candidate, XMT-1536, is currently in Phase I clinical trials targeting NaPi2b for the treatment of ovarian cancer, non-small cell lung cancer, and other rare conditions. Mersana is also advancing additional candidates, including XMT-1660, targeting B7-H4, and XMT-2056, a STING agonist ADC, both designed to improve tolerability and therapeutic outcomes. The company has established strategic partnerships with Merck KGaA and Asana BioSciences to further develop its ADC candidates. Founded in 2005, Mersana Therapeutics aims to deliver innovative treatments to patients through its advanced research and development efforts.

AVEO Oncology is a commercial-stage biopharmaceutical company headquartered in Boston, Massachusetts, focused on developing and marketing targeted therapies for cancer. Its lead product, tivozanib, marketed as FOTIVDA, is an oral, once-daily vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor used for treating renal cell carcinoma (RCC). The company has completed a Phase III trial of tivozanib and is investigating its use in combination with the immune checkpoint inhibitor Opdivo. Additionally, AVEO is developing Ficlatuzumab, an antibody targeting hepatocyte growth factor, currently in Phase II trials for several cancers, and AV-203, an anti-ErbB3 monoclonal antibody that has completed Phase I trials for esophageal cancer. The company also has preclinical products aimed at cachexia and pulmonary arterial hypertension. AVEO collaborates with various pharmaceutical companies to enhance its research and development efforts. Established in 2001, AVEO Oncology aims to address unmet medical needs in oncology through its innovative therapies.

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.

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.

Galera Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Malvern, Pennsylvania, dedicated to developing innovative therapies that aim to enhance the effectiveness of radiotherapy in cancer treatment. 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 severe oral mucositis induced by radiation in patients with head and neck cancer. Additionally, it is being tested in Phase IIa trials for radiotherapy-induced esophagitis in lung cancer patients. Galera is also advancing a second candidate, GC4711, which is in Phase I/II trials targeting pancreatic and lung cancers. The company was founded in 2012 and is committed to addressing severe side effects associated with cancer treatments to improve patient outcomes.

Tarveda Therapeutics is a developer of miniature drug conjugates designed for the treatment of patients with a wide range of solid tumors. The company's HSP90 targeted drug conjugate platform with lead drug candidate PEN-866 selectively binds in tumors to the activated form of Heat Shock Protein 90 (HSP90) and releases its potent topoisomerase 1 inhibitor payload, SN38 and it also develops a miniature drug conjugate in clinical evaluation for the treatment of patients with somatostatin receptor 2 (SSTR2) expressing neuroendocrine, small cell lung and other solid tumors, enabling companies to extend the lives of patients with hard to treat cancers while minimizing potential toxicities.

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

AVEO Oncology is a commercial-stage biopharmaceutical company headquartered in Boston, Massachusetts, focused on developing and marketing targeted therapies for cancer. Its lead product, tivozanib, marketed as FOTIVDA, is an oral, once-daily vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor used for treating renal cell carcinoma (RCC). The company has completed a Phase III trial of tivozanib and is investigating its use in combination with the immune checkpoint inhibitor Opdivo. Additionally, AVEO is developing Ficlatuzumab, an antibody targeting hepatocyte growth factor, currently in Phase II trials for several cancers, and AV-203, an anti-ErbB3 monoclonal antibody that has completed Phase I trials for esophageal cancer. The company also has preclinical products aimed at cachexia and pulmonary arterial hypertension. AVEO collaborates with various pharmaceutical companies to enhance its research and development efforts. Established in 2001, AVEO Oncology aims to address unmet medical needs in oncology through its innovative therapies.