New Enterprise Associates

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.

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.

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.

Strive Health, LLC, founded in 2018 and based in Denver, Colorado, specializes in solutions for chronic kidney disease. The company operates dialysis centers and employs a technology-enhanced clinical model to improve patient outcomes and experiences. Strive Health works collaboratively with innovative payers and providers to transform kidney care by focusing on the entire healthcare journey of chronic kidney disease patients, from primary care engagement to dialysis treatment. The company utilizes predictive analytics and comparative data to identify at-risk patients and aims to reinvent primary care to address kidney disease proactively, reducing the need for inpatient services and promoting home dialysis options. Backed by notable investors, Strive Health is committed to enhancing the management and care of patients with chronic kidney disease.

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.

SetPoint Medical is a clinical-stage bioelectronic medicine company based in Valencia, California, focused on developing implantable neuromodulation devices aimed at treating chronic autoimmune diseases. The company is creating a platform that stimulates the vagus nerve to activate the body's natural inflammatory reflex, leading to a systemic immune-restorative effect. This innovative approach offers an alternative treatment option for conditions such as rheumatoid arthritis and inflammatory bowel disease, with the potential for reduced risks and costs compared to traditional drug therapies. SetPoint Medical, originally known as Innovative Metabolics Inc. until its name change in 2008, aims to provide patients and healthcare providers with effective solutions for managing debilitating inflammatory diseases.

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.

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.

Allakos Inc. is a clinical-stage biopharmaceutical company based in Redwood City, California, founded in 2012. The company focuses on developing antibody-based therapeutics aimed at treating various conditions linked to dysregulation of the T-helper type 2 immune response, such as allergic diseases and chronic inflammation. Its lead product, antolimab (AK002), targets conditions including eosinophilic gastritis, eosinophilic gastroenteritis, chronic urticaria, indolent systemic mastocytosis, and severe allergic conjunctivitis. Additionally, Allakos is developing AK006, an antibody that targets Siglec-6, an inhibitory receptor found on mast cells. By binding to Siglec-6, AK006 is designed to enhance the receptor's natural inhibitory function, thereby reducing mast cell activation and associated inflammatory responses.

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.

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.

Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an oral extended-release formulation. The company is focused on treating serious neurologically mediated conditions, specifically chronic pruritus, chronic cough in patients with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in individuals with Parkinson's disease. Trevi is currently conducting a Phase 2b/3 clinical trial, known as the PRISM trial, for nalbuphine ER, targeting severe pruritus associated with prurigo nodularis. The conditions being addressed share similar underlying mechanisms that involve opioid receptors in both the central and peripheral nervous systems. Founded in 2011, Trevi Therapeutics aims to provide innovative treatment options for patients suffering from these challenging health issues.

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.

InCarey is a comprehensive medical service platform based in Shanghai, China, founded in 2011. The company specializes in the management of patients with chronic and vertical diseases, integrating medical, pharmaceutical, and insurance resources to deliver a one-stop solution for long-term healthcare needs. Its cloud-based platform offers professional disease management services, facilitating one-on-one interactions between doctors and patients. Additionally, InCarey's e-commerce capabilities enable users to obtain medications for major diseases efficiently. The platform is designed to address the complexities of chronic disease management, providing innovative payment solutions and a streamlined approach to healthcare services.

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.

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.

Slope.io, Inc. is a Mobile, Alabama-based company that specializes in clinical trial logistics, catering to sponsors, contract research organizations (CROs), and research sites throughout the United States. Founded in 2016, Slope.io provides a platform that streamlines the operational processes of clinical trials by translating logistical requirements into structured schedules and workflows. The company offers customized supply kits, known as Slope.io Kitting, tailored to specific sites, patients, or study time points. By connecting clinical trials to their supply chains, Slope.io effectively manages the complexity of delivering devices, drugs, and kits from suppliers to research sites and patients, ensuring real-time coordination and complete traceability.

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.

Allay Therapeutics is focused on developing innovative pain management solutions aimed at improving post-surgical recovery for patients. The company utilizes a proprietary technology platform that integrates validated non-opioid analgesics with biopolymers to create dissolvable therapeutic products. These products are designed to deliver targeted pain relief at the surgical site over extended periods, significantly longer than conventional pain treatments. Allay's lead investigational product, ATX101, is currently undergoing evaluation in a Phase 2 clinical trial for total knee arthroplasty surgeries. With a team of experienced entrepreneurs, scientists, and clinicians based in the San Francisco Bay Area and Singapore, Allay Therapeutics is dedicated to transforming the approach to pain management and addressing the limitations associated with opioid use.

AllyAlign Health operates as a care management company dedicated to supporting patients with chronic and complex medical conditions. Founded in 2013 and based in Glen Allen, Virginia, the company offers an active care management platform that facilitates real-time, collaborative care across various boundaries. This platform connects patients with care teams, providers, and community-based services, ensuring access to essential health information, care plans, and benefits necessary for informed decision-making. Users can easily view, share, create, and update information according to their roles and responsibilities, with mobile and web-based options enhancing accessibility for patients and their families. A central, HIPAA-compliant database consolidates information from multiple sources, including electronic health records and lab tests, while semantic data modeling provides meaningful clinical insights.

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.

Strive Health, LLC, founded in 2018 and based in Denver, Colorado, specializes in solutions for chronic kidney disease. The company operates dialysis centers and employs a technology-enhanced clinical model to improve patient outcomes and experiences. Strive Health works collaboratively with innovative payers and providers to transform kidney care by focusing on the entire healthcare journey of chronic kidney disease patients, from primary care engagement to dialysis treatment. The company utilizes predictive analytics and comparative data to identify at-risk patients and aims to reinvent primary care to address kidney disease proactively, reducing the need for inpatient services and promoting home dialysis options. Backed by notable investors, Strive Health is committed to enhancing the management and care of patients with chronic kidney disease.

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.

Satsuma Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative treatments for migraine. The company's lead product candidate, STS101, is a drug-device combination that features a proprietary dry-powder formulation of dihydroergotamine mesylate. This product is designed for self-administration through a pre-filled, single-use nasal delivery device and is currently undergoing Phase III clinical trials. Founded in 2016 and based in South San Francisco, California, Satsuma aims to provide an effective therapeutic option for individuals suffering from acute migraine episodes.

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.

SetPoint Medical is a clinical-stage bioelectronic medicine company based in Valencia, California, focused on developing implantable neuromodulation devices aimed at treating chronic autoimmune diseases. The company is creating a platform that stimulates the vagus nerve to activate the body's natural inflammatory reflex, leading to a systemic immune-restorative effect. This innovative approach offers an alternative treatment option for conditions such as rheumatoid arthritis and inflammatory bowel disease, with the potential for reduced risks and costs compared to traditional drug therapies. SetPoint Medical, originally known as Innovative Metabolics Inc. until its name change in 2008, aims to provide patients and healthcare providers with effective solutions for managing debilitating inflammatory diseases.

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.

Reneo Pharmaceuticals is a clinical-stage pharmaceutical company based in San Diego, California, that focuses on developing therapies for patients with rare genetic mitochondrial diseases. These conditions are linked to defects in energy metabolism, leading to issues such as muscle weakness and wasting. The company is developing REN001, an oral drug designed to modulate genes involved in mitochondrial function, aiming to enhance adenosine triphosphate (ATP) production, which is essential for cellular energy. By improving mitochondrial activity, Reneo seeks to enhance the daily functioning and quality of life for patients suffering from these disorders. Founded in 2014, Reneo Pharmaceuticals is committed to addressing the needs of individuals with orphan metabolic diseases.

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.

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.

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.

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.

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.

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.

SetPoint Medical is a clinical-stage bioelectronic medicine company based in Valencia, California, focused on developing implantable neuromodulation devices aimed at treating chronic autoimmune diseases. The company is creating a platform that stimulates the vagus nerve to activate the body's natural inflammatory reflex, leading to a systemic immune-restorative effect. This innovative approach offers an alternative treatment option for conditions such as rheumatoid arthritis and inflammatory bowel disease, with the potential for reduced risks and costs compared to traditional drug therapies. SetPoint Medical, originally known as Innovative Metabolics Inc. until its name change in 2008, aims to provide patients and healthcare providers with effective solutions for managing debilitating inflammatory diseases.

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.

Reneo Pharmaceuticals is a clinical-stage pharmaceutical company based in San Diego, California, that focuses on developing therapies for patients with rare genetic mitochondrial diseases. These conditions are linked to defects in energy metabolism, leading to issues such as muscle weakness and wasting. The company is developing REN001, an oral drug designed to modulate genes involved in mitochondrial function, aiming to enhance adenosine triphosphate (ATP) production, which is essential for cellular energy. By improving mitochondrial activity, Reneo seeks to enhance the daily functioning and quality of life for patients suffering from these disorders. Founded in 2014, Reneo Pharmaceuticals is committed to addressing the needs of individuals with orphan metabolic diseases.

Strive Health, LLC, founded in 2018 and based in Denver, Colorado, specializes in solutions for chronic kidney disease. The company operates dialysis centers and employs a technology-enhanced clinical model to improve patient outcomes and experiences. Strive Health works collaboratively with innovative payers and providers to transform kidney care by focusing on the entire healthcare journey of chronic kidney disease patients, from primary care engagement to dialysis treatment. The company utilizes predictive analytics and comparative data to identify at-risk patients and aims to reinvent primary care to address kidney disease proactively, reducing the need for inpatient services and promoting home dialysis options. Backed by notable investors, Strive Health is committed to enhancing the management and care of patients with chronic kidney disease.

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.

Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an oral extended-release formulation. The company is focused on treating serious neurologically mediated conditions, specifically chronic pruritus, chronic cough in patients with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in individuals with Parkinson's disease. Trevi is currently conducting a Phase 2b/3 clinical trial, known as the PRISM trial, for nalbuphine ER, targeting severe pruritus associated with prurigo nodularis. The conditions being addressed share similar underlying mechanisms that involve opioid receptors in both the central and peripheral nervous systems. Founded in 2011, Trevi Therapeutics aims to provide innovative treatment options for patients suffering from these challenging health issues.

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.

Allay Therapeutics is focused on developing innovative pain management solutions aimed at improving post-surgical recovery for patients. The company utilizes a proprietary technology platform that integrates validated non-opioid analgesics with biopolymers to create dissolvable therapeutic products. These products are designed to deliver targeted pain relief at the surgical site over extended periods, significantly longer than conventional pain treatments. Allay's lead investigational product, ATX101, is currently undergoing evaluation in a Phase 2 clinical trial for total knee arthroplasty surgeries. With a team of experienced entrepreneurs, scientists, and clinicians based in the San Francisco Bay Area and Singapore, Allay Therapeutics is dedicated to transforming the approach to pain management and addressing the limitations associated with opioid use.

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.

Sleep Reset is a personalized sleep improvement program that combines scientific methods with dedicated coaching. It aims to tackle insomnia and other sleep issues by addressing their root causes, rather than relying on pills or supplements. The program, developed in collaboration with renowned sleep experts from institutions like Stanford and Yale, has been clinically proven to increase total sleep time by up to 85 minutes for those with short sleep durations, and significantly reduce sleep onset latency and nightly awakenings. Users receive a tailored plan from a dedicated sleep coach, providing a sustainable solution to improve sleep within a few weeks. Sleep Reset has gained recognition in notable publications and is backed by prestigious Silicon Valley investors.

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.

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.

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.

MEI Pharma, Inc. is a late-stage pharmaceutical company based in San Diego, California, dedicated to developing innovative therapies for cancer treatment. The company's clinical pipeline includes several key drug candidates such as Zandelisib, an oral phosphatidylinositol 3-kinase delta inhibitor currently in Phase II trials for relapsed follicular lymphoma, and Voruciclib, an oral cyclin-dependent kinase inhibitor under investigation for acute myeloid leukemia and B-cell malignancies in Phase Ib trials. Additionally, MEI Pharma is developing ME-344, a mitochondrial inhibitor targeting HER2-negative breast cancer, and Pracinostat, an oral histone deacetylase inhibitor being tested for myelodysplastic syndrome. The company has established various collaborations and agreements with notable partners, including Kyowa Kirin Company and BeiGene, to support its clinical development efforts. Founded in 2000, MEI Pharma has evolved significantly since its inception and continues to focus on advancing its promising oncology therapies.

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.

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.

Metavention, Inc., incorporated in 2012 and based in Dover, Delaware, focuses on innovative treatments aimed at addressing metabolic diseases linked to overactive sympathetic nerves. The company has developed a renal denervation procedure utilizing its IRF denervation system, which applies radio frequency energy to specifically target these nerves. This approach is designed to provide a permanent solution for conditions such as high blood pressure and complications associated with Type 2 diabetes, including elevated glucose levels and abnormal liver function. By employing trans-catheter devices that leverage standard interventional vascular techniques, Metavention equips medical practitioners with effective tools for diagnosing and treating patients suffering from these metabolic disorders.

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.

Centrexion Therapeutics Corporation is a late clinical-stage biopharmaceutical company based in Boston, Massachusetts, dedicated to developing non-opioid and non-addictive therapies aimed at addressing chronic pain. The company's product pipeline includes CNTX-4975, currently in Phase III trials for moderate to severe knee osteoarthritis pain, and in Phase II trials for Morton’s neuroma and canine osteoarthritis. Additionally, CNTX-0290 is in Phase I trials targeting chronic pain associated with inflammatory, neuropathic, and mixed conditions. Other candidates under development include CNTX-6970, for inflammatory pain, and CNTX-2022, a high-concentration topical gel formulation of lidocaine for various types of pain. CNTX-6016 is also in pre-clinical stages focusing on chronic neuropathic pain. Founded in 2013, Centrexion Therapeutics aims to provide safe and effective solutions for patients suffering from chronic pain conditions.

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.

Allakos Inc. is a clinical-stage biopharmaceutical company based in Redwood City, California, founded in 2012. The company focuses on developing antibody-based therapeutics aimed at treating various conditions linked to dysregulation of the T-helper type 2 immune response, such as allergic diseases and chronic inflammation. Its lead product, antolimab (AK002), targets conditions including eosinophilic gastritis, eosinophilic gastroenteritis, chronic urticaria, indolent systemic mastocytosis, and severe allergic conjunctivitis. Additionally, Allakos is developing AK006, an antibody that targets Siglec-6, an inhibitory receptor found on mast cells. By binding to Siglec-6, AK006 is designed to enhance the receptor's natural inhibitory function, thereby reducing mast cell activation and associated inflammatory responses.

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.

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.

SetPoint Medical is a clinical-stage bioelectronic medicine company based in Valencia, California, focused on developing implantable neuromodulation devices aimed at treating chronic autoimmune diseases. The company is creating a platform that stimulates the vagus nerve to activate the body's natural inflammatory reflex, leading to a systemic immune-restorative effect. This innovative approach offers an alternative treatment option for conditions such as rheumatoid arthritis and inflammatory bowel disease, with the potential for reduced risks and costs compared to traditional drug therapies. SetPoint Medical, originally known as Innovative Metabolics Inc. until its name change in 2008, aims to provide patients and healthcare providers with effective solutions for managing debilitating inflammatory diseases.

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.

Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an oral extended-release formulation. The company is focused on treating serious neurologically mediated conditions, specifically chronic pruritus, chronic cough in patients with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in individuals with Parkinson's disease. Trevi is currently conducting a Phase 2b/3 clinical trial, known as the PRISM trial, for nalbuphine ER, targeting severe pruritus associated with prurigo nodularis. The conditions being addressed share similar underlying mechanisms that involve opioid receptors in both the central and peripheral nervous systems. Founded in 2011, Trevi Therapeutics aims to provide innovative treatment options for patients suffering from these challenging health issues.

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.

Sleep Reset is a personalized sleep improvement program that combines scientific methods with dedicated coaching. It aims to tackle insomnia and other sleep issues by addressing their root causes, rather than relying on pills or supplements. The program, developed in collaboration with renowned sleep experts from institutions like Stanford and Yale, has been clinically proven to increase total sleep time by up to 85 minutes for those with short sleep durations, and significantly reduce sleep onset latency and nightly awakenings. Users receive a tailored plan from a dedicated sleep coach, providing a sustainable solution to improve sleep within a few weeks. Sleep Reset has gained recognition in notable publications and is backed by prestigious Silicon Valley investors.

SetPoint Medical is a clinical-stage bioelectronic medicine company based in Valencia, California, focused on developing implantable neuromodulation devices aimed at treating chronic autoimmune diseases. The company is creating a platform that stimulates the vagus nerve to activate the body's natural inflammatory reflex, leading to a systemic immune-restorative effect. This innovative approach offers an alternative treatment option for conditions such as rheumatoid arthritis and inflammatory bowel disease, with the potential for reduced risks and costs compared to traditional drug therapies. SetPoint Medical, originally known as Innovative Metabolics Inc. until its name change in 2008, aims to provide patients and healthcare providers with effective solutions for managing debilitating inflammatory diseases.

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 Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to the development and commercialization of therapeutics for rare genetic disorders, particularly those that lead to life-threatening metabolic conditions. The company’s primary product candidate, setmelanotide, is a first-in-class melanocortin-4 receptor agonist currently undergoing Phase III clinical trials for the treatment of obesity related to pro-opiomelanocortin (POMC) and leptin receptor deficiencies, as well as Bardet-Biedl and Alström syndromes. Additionally, it is in Phase II trials for various other genetic obesity disorders. Rhythm Pharmaceuticals is also advancing RM-853, an orally available ghrelin o-acyltransferase inhibitor in preclinical development aimed at addressing Prader-Willi syndrome. Founded in 2008 and based in Boston, Massachusetts, the company previously operated under the name Rhythm Metabolic, Inc. and rebranded in October 2015.

Cartiva, Inc. is a medical device company based in Alpharetta, Georgia, focused on developing and marketing innovative solutions for orthopedic surgeons and patients suffering from osteoarthritis and cartilage injuries. Founded in 2011 as a spin-off from Carticept Medical, the company offers the Cartiva Synthetic Cartilage Implant (SCI), designed to replace damaged cartilage surfaces in joints such as the foot, ankle, and knee. This organic polymer-based biomaterial aims to restore natural joint structure, alleviate pain, and enhance functionality. In addition to the SCI, Cartiva provides the ProxiFuse Hammertoe Correction System, which assists in the fixation and reconstruction of lesser toes after correction procedures. The company's products are distributed internationally, including regions such as Brazil, Canada, and the United Kingdom. Cartiva is dedicated to improving patient quality of life through effective treatment options and is backed by a team of experienced professionals in the orthopedic field.

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.

Trillium Therapeutics Inc. is a clinical-stage immuno-oncology company based in Mississauga, Canada, that specializes in developing therapies for cancer treatment. The company focuses on harnessing the immune system to combat malignancies through innovative protein therapeutics. Its lead program, TTI-621, is a SIRPaFc fusion protein currently undergoing Phase I clinical trials for advanced relapsed or refractory hematologic malignancies, solid tumors, and mycosis fungoides. Additionally, Trillium is advancing TTI-622, another SIRPaFc protein in Phase I trials, while also exploring TTI-10001, a small molecule stimulator of interferon genes agonist in the discovery phase. Originally founded in 2004, the company has undergone several name changes, reflecting its evolving focus within the biopharmaceutical sector.

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.