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35Pharma is a biopharmaceutical company that designs and develops innovative biologics to address diseases with high unmet medical need. The company applies protein engineering and a deep understanding of transforming growth factor-beta structure-function relationships to create potent ligand traps for cardio-pulmonary and metabolic diseases, including pulmonary hypertension and musculoskeletal disorders.

Adcendo is a biotechnology company focused on developing novel antibody-drug conjugates (ADCs) for cancer treatment. These ADCs target specific receptors overexpressed in various cancer forms with significant unmet needs, such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer.

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

OnKure Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing precision medicines that target biologically validated drivers of cancers underserved by existing therapies. The company develops epigenetic therapies, including selective histone deacetylase inhibitors, and advances candidates such as OKI-179 and OKI-422 in preclinical and clinical development. Its approach aims to directly target malignant cells, combine with other targeted therapies, and prime the tumor microenvironment to enhance immunotherapies, with a pipeline of tumor-agnostic candidates designed for optimal efficacy and tolerability.

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

Cartesian Therapeutics is a clinical-stage biopharmaceutical company developing cell and gene immunotherapies to treat cancer and autoimmune diseases. The company designs chimeric antigen receptor therapies for multiple myeloma and other cancers and uses its ImmTOR immune tolerance platform to mitigate antigen-specific immune responses. It operates a proprietary technology and manufacturing platform to deliver one or more mRNA molecules into cells to enhance their function, aiming to improve therapeutic performance.

Founded in 2017, Aardvark Therapeutics is a biopharmaceutical company dedicated to developing small molecule therapeutics targeting metabolic diseases. Its lead product candidate, ARD-101, is an oral gut-restricted therapy designed to selectively activate bitter taste receptors for hunger-associated conditions.

BridgeBio Oncology Therapeutics is a biopharmaceutical company specializing in the development of precision oncology therapeutics. It focuses on targeting RAS-dependent cancers, leveraging a profound understanding of RAS signaling biology to create drugs that selectively inhibit RAS-driven PI3K activation and a wide spectrum of KRAS mutants. The company aims to provide patients with tailored treatments that address both active and inactive cancer-driving states.

Cullinan Therapeutics is a developer of an externally sourced cancer therapeutics intended to end a drug program quickly if the early research suggests it won't work. The company's therapeutics are developed via assets sourced internally through dry lab or externally through business, academic and pharma collaborations, enabling researchers to get a highly diversified portfolio of oncology therapeutics with a unique, cost-efficient business model.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on developing precision oncology therapeutics. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond positively to specific treatments, thereby enhancing the effectiveness of its drug development efforts. Acrivon's pipeline features its lead program, ACR-368 (prexasertib), which is currently in Phase 2 trials, alongside several preclinical programs targeting key components of DNA damage response and cell cycle regulation, including WEE1 and PKMYT1. By concentrating on complex oncology drug targets that are often not addressed by traditional therapies, Acrivon aims to expedite the advancement of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials.

Founded in 2015 and based in Cambridge, Massachusetts, Obsidian Therapeutics develops next-generation controllable cell and gene therapies designed to extend adoptive immunotherapy to every cancer patient. Its innovative approach uses pharmacologic operating systems to precisely control protein activity in cells, enabling physicians to improve upon current cell therapies using simple, safe, orally-active marketed drugs.

Ventyx Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral, selective TYK2 inhibitor in Phase 1 trials for various autoimmune diseases.

Sudo Biosciences is a biopharmaceutical company focused on developing innovative medicines targeting the tyrosine kinase 2 (TYK2) pseudokinase domain, which plays a critical role in immune-mediated inflammatory conditions. Its pipeline includes next-generation TYK2 inhibitors for treating multiple sclerosis and other neuroinflammatory diseases, as well as topical candidates for immune-mediated dermatologic conditions.

Attralus, Inc. is a biopharmaceutical company dedicated to addressing the systemic nature of amyloidosis and enhancing the quality of life for affected patients. Incorporated in 2010 and headquartered in South San Francisco, California, Attralus develops proprietary peptide-based pan-amyloid targeting agents aimed at diagnosing and treating various forms and stages of systemic amyloidosis. Its product pipeline includes AT-01, a polybasic peptide radiotracer for diagnostic and monitoring purposes; AT-02, a peptide-immunoglobulin fusion for therapeutic use; and AT-03, an Fc-fusion protein designed for therapeutic applications. By targeting the common pathology present in all systemic amyloidosis diseases, Attralus seeks to create effective treatments for all subtypes, particularly those that currently lack available treatment options. The company was previously known as Aurora Bio, Inc., before rebranding in August 2020.

Sudo Biosciences is a biopharmaceutical company focused on developing innovative medicines targeting the tyrosine kinase 2 (TYK2) pseudokinase domain, which plays a critical role in immune-mediated inflammatory conditions. Its pipeline includes next-generation TYK2 inhibitors for treating multiple sclerosis and other neuroinflammatory diseases, as well as topical candidates for immune-mediated dermatologic conditions.

Arvinas, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, that specializes in the discovery, development, and commercialization of innovative therapies aimed at degrading disease-causing proteins. The company's lead product candidates include ARV-110, a proteolysis targeting chimera (PROTAC) currently in phase I clinical trials for metastatic castration-resistant prostate cancer, and ARV-471, which targets the estrogen receptor for patients with metastatic ER-positive/HER2-negative breast cancer. In addition to these, Arvinas is developing various other PROTACs aimed at degrading clinically relevant androgen receptor mutations and treatments for neurodegenerative diseases, including tauopathies. The company has established collaborations with prominent pharmaceutical firms such as Pfizer Inc., Genentech, and Bayer AG, enhancing its research capabilities and reach within the biopharmaceutical landscape. Founded in 2015, Arvinas aims to improve patient outcomes by eliminating harmful proteins from the body through its proprietary technology.

VYNE Therapeutics Inc. is a biopharmaceutical company specializing in the development and commercialization of innovative therapies for dermatological conditions. It offers AMZEEQ, a topical minocycline treatment for non-nodular moderate-to-severe acne vulgaris in patients aged 9 and older. The company is advancing its pipeline with FMX103, currently in Phase III clinical trials for moderate-to-severe papulopustular rosacea in adults, and FCD105, a topical combination foam under investigation in Phase II trials for moderate-to-severe acne vulgaris. Additionally, VYNE is developing Serlopitant, an oral NK1 receptor antagonist aimed at addressing pruritus associated with prurigo nodularis, as well as exploring therapies for other immuno-inflammatory conditions. Formerly known as Menlo Therapeutics Inc., the company rebranded in September 2020 and is headquartered in Bridgewater, New Jersey.

Harpoon Therapeutics, Inc. is a clinical-stage immunotherapy company based in South San Francisco, California, focused on developing a novel class of T cell engagers to harness the body's immune system for treating cancer and other diseases. The company utilizes its proprietary TriTAC (Tri-specific T cell Activating Construct) platform to create engineered proteins that direct T cells to target and kill cells expressing specific antigens. Harpoon's lead product candidate, HPN424, is currently undergoing Phase I clinical trials for metastatic castration-resistant prostate cancer. Additionally, the company is advancing other candidates, including HPN536, which is in Phase I/IIa trials for ovarian cancer and MSLN-expressing tumors, as well as HPN217 for multiple myeloma and HPN328 for small cell lung cancer. Harpoon Therapeutics was founded in 2015 and is committed to addressing unmet medical needs in oncology through innovative therapies.

VistaGen Therapeutics is a clinical-stage biopharmaceutical company focused on developing medicines for central nervous system disorders. Its product candidates include AV-101, an orally available prodrug in Phase 2 for major depressive disorder and related symptoms; PH94B, a neuroactive nasal spray in preparation for Phase III clinical development for acute treatment of anxiety in adults with social anxiety disorder; and PH10, a neuroactive nasal spray planned for Phase 2b development for major depressive disorder. The company pursues licensing and collaboration arrangements to support development and commercialization with partners such as Pherin Pharmaceuticals, BlueRock Therapeutics, Cato Research, and EverInsight Therapeutics for PH94B in Asia. VistaGen was founded in 1998 and is headquartered in South San Francisco, California.

Avalyn Pharma is a biopharmaceutical company focused on developing therapies for severe respiratory diseases, notably idiopathic pulmonary fibrosis (IPF). Established in 2011, the company is headquartered in Seattle, Washington.

Mariana Oncology is a biotechnology company focused on developing innovative radiopharmaceuticals aimed at targeting cancer cells with radioactive drugs. The company employs a multidisciplinary approach that includes ligand discovery, radiochemistry, radiobiology, and translational and clinical research in oncology. By building a diverse pipeline of treatments for various solid tumor types, Mariana Oncology seeks to enhance the efficacy of radio medicines in cancer therapy, ultimately aiding medical professionals in their efforts to improve patient outcomes.

Rapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing precision medicines for neurological disorders. The company focuses on creating transformational small-molecule therapies aimed at patients with central nervous system conditions. Leveraging foundational research in neuronal receptor biology, Rapport Therapeutics maps and targets specific neuronal receptor complexes, which are complex assemblies of proteins that include principal receptor subunits and receptor-associated proteins. These receptor-associated proteins are essential for regulating receptor expression and functionality, allowing the company to innovate in the treatment of neurological diseases.

COMPASS Pathways is a UK-based mental health care company dedicated to accelerating patient access to innovative treatments for mental health challenges. The company focuses on developing psilocybin therapy, administered in conjunction with psychological support, for patients with treatment-resistant depression who have not responded to conventional treatments.

AN2 Therapeutics is a biopharmaceutical company dedicated to researching, developing, and commercializing innovative medicines targeting infectious diseases. Established in 2017, the company focuses on advancing a clinical-stage antibacterial compound and has a strategic partnership with Brii Biosciences.

Lyra Therapeutics is a clinical-stage therapeutics company focused on the development and commercialization of localized treatments for ear, nose, and throat diseases. Its XTreo platform is designed to deliver medicines directly to affected tissue for sustained effect with a single administration. The company’s initial product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices intended for in-office administration to provide up to six months of continuous therapy to the sinonasal passages for chronic rhinosinusitis, with mometasone furoate as the active ingredient. Founded in 2005 and headquartered in Watertown, Massachusetts.

OnKure Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing precision medicines that target biologically validated drivers of cancers underserved by existing therapies. The company develops epigenetic therapies, including selective histone deacetylase inhibitors, and advances candidates such as OKI-179 and OKI-422 in preclinical and clinical development. Its approach aims to directly target malignant cells, combine with other targeted therapies, and prime the tumor microenvironment to enhance immunotherapies, with a pipeline of tumor-agnostic candidates designed for optimal efficacy and tolerability.

Quanta Therapeutics is a biotechnology company focused on developing novel cancer therapeutic candidates. It specializes in allosteric modulation to directly inhibit RAS-driven cancer types by restoring conformational control of active RAS signaling at the cell membrane.

Boundless Bio develops innovative cancer therapeutics focused on understanding and treating previously untreatable cancers. It specializes in targeting extrachromosomal DNA (ecDNA) biology, a key driver of aggressive cancers.

Karyopharm Therapeutics Inc. is a clinical-stage pharmaceutical company based in Newton, Massachusetts, focused on the discovery and development of innovative drug therapies for cancer and other significant diseases. The company specializes in small molecule selective inhibitors of nuclear export (SINE) compounds, targeting the nuclear export protein exportin 1. Its lead product, XPOVIO, is approved in the U.S. for treating multiple myeloma in various settings. Karyopharm is advancing several clinical trials, including BOSTON and STORM for multiple myeloma, SADAL for diffuse large B-cell lymphoma, and SEAL for liposarcoma. Additionally, it is exploring the treatment of endometrial cancer through the SIENDO trial and glioblastoma multiforme in the KING trial. The company maintains collaborations to facilitate clinical trials and research efforts, including projects related to COVID-19. Karyopharm continues to develop various investigational programs aimed at expanding its therapeutic portfolio.

VectivBio AG is a clinical-stage biotechnology company based in Basel, Switzerland, founded in 2019 as a spinout from Therachon. The company specializes in the discovery, development, and commercialization of innovative treatments for severe rare diseases, particularly those with significant unmet medical needs. Its lead product candidate, Apraglutide, is a synthetic GLP-2 analog designed to enhance the intestine’s ability to absorb fluids and nutrients, thereby reducing the reliance on parenteral support in patients with short bowel syndrome. VectivBio is dedicated to developing best-in-disease therapies that aim to meaningfully improve the lives of patients and their families, providing more than just incremental advancements over existing standard-of-care options.

Allakos is a clinical-stage biopharmaceutical company developing antibody-based therapeutics targeting immunomodulatory receptors on immune effector cells. Its pipeline focuses on allergic, inflammatory, and proliferative diseases, with lead programs targeting Siglec-6 (AK006) and Siglec-8.

Acelyrin is a biopharmaceutical company dedicated to advancing treatments for diseases involving excessive immune system activation. It achieves this by discovering, acquiring, and expediting the development and market launch of innovative drug candidates.

Triana Biomedicines develops a molecular glue discovery platform targeting challenging disease targets. Its drug discovery engine combines high-resolution structural insights, advanced computational tools, and proprietary chemical libraries.

MoonLake Immunotherapeutics is a clinical-stage biopharmaceutical company developing novel therapies for inflammatory skin and joint diseases. Its flagship product, SLK, is an investigational Nanobody targeting IL-17A and IL-17F, showing potential for disease modification in dermatology and rheumatology patients.

Founded in 2018, Neuron23 is a biotechnology company based in South San Francisco, California, with an additional office in Munich, Germany. It specializes in developing and manufacturing medicines for genetic disorders using artificial intelligence technology and genetics.

Metagenomi is a genetic medicines company headquartered in Emeryville, California. It leverages metagenomics and machine learning to discover novel genome editing systems from diverse organisms, then convert these natural systems into compact, highly efficient, and specific tools aimed at reducing immune responses. The company maintains a proprietary, metagenomics-derived genome editing toolbox that includes programmable nucleases, base editors, small RNA-mediated integration systems (RIGS), large template RIGS, and CRISPR-associated transposases (CASTs). Its development focus is in vivo gene editing with a wholly owned pipeline, while exploring technology out-licensing for ex vivo cell therapies to pursue curative therapeutics for genetic diseases.

Founded in 2021, Solve Therapeutics is a biopharmaceutical company based in Belmont, California. It specializes in the development of first-in-class Antibody Drug Conjugates and Bispecific Therapeutics using its proprietary biotechnology platform.

Founded in 2019, Enliven Therapeutics is a precision oncology company based in Boulder, Colorado. It focuses on developing small molecule therapies to extend and improve patient lives by targeting clinically validated biological targets with industry-leading chemistry.

AN2 Therapeutics is a biopharmaceutical company dedicated to researching, developing, and commercializing innovative medicines targeting infectious diseases. Established in 2017, the company focuses on advancing a clinical-stage antibacterial compound and has a strategic partnership with Brii Biosciences.

Ambagon Therapeutics is a biotechnology company focused on developing small-molecule therapeutics for cancer treatment. The company specializes in research aimed at stabilizing the interactions of oncogenic proteins, which are often difficult to modulate with traditional methods. By selectively enhancing the activity of tumor-suppressing proteins associated with transcription factors and oncoproteins, Ambagon aims to provide new therapeutic options for addressing significant unmet clinical needs in oncology. Founded by Christian Ottmann, Lucas Brunsveld, and Michelle Arkin, the company is dedicated to advancing innovative solutions in cancer care.

Emergence Therapeutics AG is a biopharmaceutical company based in Duisburg, Germany, focused on developing innovative antibody-drug conjugates (ADCs) to address high-need cancers. Established in 2019, the company leverages advanced technologies in antibody design, linker systems, and therapeutic payloads to create targeted treatments. Its lead program specifically targets Nectin-4, a clinically validated marker for various cancers, utilizing a highly specific antibody in combination with optimized linker technology and amanitin as the payload. This approach aims to provide effective therapeutic options for challenging cancer types. Emergence Therapeutics is also exploring opportunities to develop additional first- or best-in-class ADCs to meet unmet medical needs in oncology.

Acelyrin is a biopharmaceutical company dedicated to advancing treatments for diseases involving excessive immune system activation. It achieves this by discovering, acquiring, and expediting the development and market launch of innovative drug candidates.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on developing precision oncology therapeutics. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond positively to specific treatments, thereby enhancing the effectiveness of its drug development efforts. Acrivon's pipeline features its lead program, ACR-368 (prexasertib), which is currently in Phase 2 trials, alongside several preclinical programs targeting key components of DNA damage response and cell cycle regulation, including WEE1 and PKMYT1. By concentrating on complex oncology drug targets that are often not addressed by traditional therapies, Acrivon aims to expedite the advancement of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials.

Quanta Therapeutics is a biotechnology company focused on developing novel cancer therapeutic candidates. It specializes in allosteric modulation to directly inhibit RAS-driven cancer types by restoring conformational control of active RAS signaling at the cell membrane.

Structure Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing oral small-molecule medicines for chronic diseases with high unmet medical needs. Its platform combines structure-based drug design, computational chemistry, and data integration to create orally available therapies that overcome limitations of biologics and peptide drugs. The company concentrates on targeting G-protein coupled receptors and other mechanisms, leveraging structural insights to identify differentiated, efficacious, and safe treatments. Its programs address cardiovascular, metabolic, and pulmonary conditions, with a focus on delivering convenient, effective medicines that can offer broad patient impact and commercial potential. Structure Therapeutics conducts its research and development activities across its pipeline within a single organization and seeks to advance clinical candidates through development.

Science 37 specializes in decentralized clinical trials, offering a technology-driven approach that enables patients to participate from their homes. The company's platform connects researchers with patients via telemedicine investigators and mobile nurses, accelerating trial enrollment and retention while reaching a more diverse patient population.

Expansion Therapeutics is a drug discovery and development company focused on creating small molecule medicines for RNA-mediated diseases. The company specializes in developing novel RNA-targeted drug candidates that aim to address a wide range of severe medical conditions. By leveraging its innovative technology, Expansion Therapeutics seeks to provide transformative oral treatment options, enhancing the therapeutic landscape for patients suffering from these complex diseases.

Ventyx Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral, selective TYK2 inhibitor in Phase 1 trials for various autoimmune diseases.

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

Founded in 2015 and based in Cambridge, Massachusetts, Obsidian Therapeutics develops next-generation controllable cell and gene therapies designed to extend adoptive immunotherapy to every cancer patient. Its innovative approach uses pharmacologic operating systems to precisely control protein activity in cells, enabling physicians to improve upon current cell therapies using simple, safe, orally-active marketed drugs.

Attralus, Inc. is a biopharmaceutical company dedicated to addressing the systemic nature of amyloidosis and enhancing the quality of life for affected patients. Incorporated in 2010 and headquartered in South San Francisco, California, Attralus develops proprietary peptide-based pan-amyloid targeting agents aimed at diagnosing and treating various forms and stages of systemic amyloidosis. Its product pipeline includes AT-01, a polybasic peptide radiotracer for diagnostic and monitoring purposes; AT-02, a peptide-immunoglobulin fusion for therapeutic use; and AT-03, an Fc-fusion protein designed for therapeutic applications. By targeting the common pathology present in all systemic amyloidosis diseases, Attralus seeks to create effective treatments for all subtypes, particularly those that currently lack available treatment options. The company was previously known as Aurora Bio, Inc., before rebranding in August 2020.

Renovacor, Inc., established in 2013 and headquartered in Philadelphia, Pennsylvania, is a biopharmaceutical company dedicated to developing transformative gene therapies for cardiovascular diseases. Currently in the preclinical stage, Renovacor's primary focus is on creating a recombinant adeno-associated virus (AAV)-based gene therapy targeting patients with dilated cardiomyopathy (DCM) caused by mutations in the Bcl2-associated athanogene 3 (BAG3) gene. This condition affects approximately 35,000 individuals in the United States and Europe, qualifying it as an orphan disease. Renovacor aims to improve patient outcomes by potentially preventing disease progression through a BAG3 gene replacement therapy, given the current five-year survival rate of only 50% for DCM patients despite standard care.

Tango Therapeutics is a biotechnology company focused on developing cancer therapies based on the genetic principle of synthetic lethality. It aims to counteract tumor suppressor gene loss, reverse immune evasion in cancer cells, and identify effective drug combinations.

Zentera Therapeutics is a biopharmaceutical company.

Nimbus Therapeutics, LLC is a biotechnology company based in Cambridge, Massachusetts, focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. Founded in 2009, the company utilizes a computational technology-driven approach to advance a pipeline of novel small-molecule therapeutics. Nimbus Therapeutics specializes in designing allosteric inhibitors, notably targeting Acetyl-CoA Carboxylase for conditions such as non-alcoholic steatohepatitis and hepatocellular carcinoma. The company has established strategic partnerships with organizations like Charles River Laboratories and Celgene Corporation to enhance its drug development efforts. In March 2015, Nimbus Discovery rebranded itself as Nimbus Therapeutics to better reflect its transition into a clinical-stage enterprise.

Point Biopharma Global Inc., established in 2019 and headquartered in Toronto, Canada, specializes in the development and commercialization of radioligand therapies for cancer treatment. The company's portfolio comprises several assets, including PNT2002, PNT2004, PNT2003, and PNT2001. Point Biopharma leverages its expertise in radioisotopes like Actinium-225 and Lutetium-177, along with advanced manufacturing technology and a patient-centric approach, to revolutionize theragnostic drug development and radioligand commercialization on a global scale.

BioTheryX is a clinical-stage biopharmaceutical company focused on restoring protein homeostasis through targeted protein degradation. It specializes in developing innovative small molecule degraders, initially targeting cancer, using its proprietary PRODEGY platform built upon expertise in Cereblon binding.

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

Forge Biologics, Inc. is a contract development and manufacturing organization (CDMO) specializing in gene therapies and genetic medicines. Founded in 2019 and based in Columbus, Ohio, the company focuses on developing and manufacturing therapeutics, particularly for viral vector gene therapies aimed at treating genetic diseases. Notably, it offers a novel approach combining adeno-associated virus (AAV) and umbilical cord transplant to address infantile Krabbe disease, a severe neurodegenerative disorder. Additionally, Forge Biologics collaborates with scientists, physicians, biotech and pharmaceutical companies, and patient groups to facilitate the progression of gene therapy programs from preclinical development through to clinical and commercial-scale manufacturing. The company is committed to enabling access to transformative medicines for patients suffering from rare genetic diseases.

Boundless Bio develops innovative cancer therapeutics focused on understanding and treating previously untreatable cancers. It specializes in targeting extrachromosomal DNA (ecDNA) biology, a key driver of aggressive cancers.

Janux Therapeutics develops immunotherapies that target tumors without harming healthy tissue. Its proprietary technology enables two distinct bispecific platforms: Tumor Activated T Cell Engagers (TRACTr) and Tumor Activated Immunomodulators (TRACIr), designed to stimulate the immune system's response against cancer.

Arcellx is a clinical-stage biotechnology company developing immunotherapies and cell therapies for cancer and other serious diseases. It seeks to make cell therapies safer, more effective, and more broadly accessible. Its lead candidate CART-ddBCMA is in a Phase 1 trial for relapsed or refractory multiple myeloma and has FDA Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy designations. The company is also advancing ARC-SparX, a dosable and controllable CAR-T platform, into clinical programs ACLX-001 for relapsed or refractory multiple myeloma and ACLX-002 for relapsed or refractory acute myeloid leukemia. Arcellx's pipeline covers multiple myeloma, acute myeloid leukemia, and other cancer indications, with research extending into additional oncology areas and other serious diseases.

GH Research is a clinical-stage biopharmaceutical company focused on developing therapies for psychiatric and neurological disorders, with an emphasis on treatment-resistant depression. It is advancing novel and proprietary mebufotenin (5-MeO-DMT) therapies, including inhalable and intravenous product candidates, to provide transformative treatment options for patients who do not respond to existing therapies. The portfolio includes GH001, an inhalable mebufotenin candidate, and GH002, an intravenous candidate. The company concentrates its activities in research and development to create practice-changing solutions for depression.

Ventyx Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral, selective TYK2 inhibitor in Phase 1 trials for various autoimmune diseases.

OnKure Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing precision medicines that target biologically validated drivers of cancers underserved by existing therapies. The company develops epigenetic therapies, including selective histone deacetylase inhibitors, and advances candidates such as OKI-179 and OKI-422 in preclinical and clinical development. Its approach aims to directly target malignant cells, combine with other targeted therapies, and prime the tumor microenvironment to enhance immunotherapies, with a pipeline of tumor-agnostic candidates designed for optimal efficacy and tolerability.

DTx Pharma, LLC is a biotechnology company headquartered in San Diego, California, specializing in the development and commercialization of fatty acid-conjugated peptide therapeutics. Founded in 2017, the company focuses on delivering RNA-based medicines to treat patients with rare and chronic diseases, including retinitis pigmentosa, Duchenne muscular dystrophy, and various central nervous system disorders. DTx Pharma's innovative delivery technology addresses challenges faced by previous-generation platforms, such as poor pharmacokinetics and limited cellular uptake, thereby enhancing the efficiency of nucleic acid drug delivery across diverse tissues and cell types. The company aims to establish RNA therapeutics as a leading option for personalized treatment in multiple therapeutic areas.

Satsuma Pharmaceuticals, Inc. 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 incorporates a proprietary dry-powder formulation of dihydroergotamine mesylate. Designed for self-administration, STS101 utilizes a pre-filled, single-use nasal delivery device, enhancing patient convenience and accessibility. The product is currently undergoing Phase III clinical trials, marking a significant step in its development. Founded in 2016, Satsuma is headquartered in South San Francisco, California.

Artiva Biotherapeutics, Inc. is a San Diego-based biotechnology company focused on developing and manufacturing cellular immunotherapies for cancer treatment. Founded in 2019, Artiva specializes in off-the-shelf, allogeneic natural killer (NK) cell therapies aimed at treating both hematologic cancers and solid tumors. The company's product pipeline includes therapies that target CD20 and CD19 in B-cell lymphomas, as well as HER2 in various solid tumors. Artiva is committed to harnessing the therapeutic potential of NK cells while addressing challenges in their scalability and manufacturing. Its mission centers on delivering effective and safe cell therapies that are readily accessible to cancer patients in need.

ASLAN Pharmaceuticals, established in 2010 and based in Singapore, is a clinical-stage biopharmaceutical company focused on immunology and oncology. It develops innovative treatments, including a monoclonal therapy for atopic dermatitis and a small molecule inhibitor for cancer, aiming to transform patients' lives. The company collaborates with prominent pharmaceutical partners and operates as a subsidiary of ASLAN Pharmaceuticals Limited.

Viracta Therapeutics, Inc. is a clinical-stage drug development company based in Cardiff, California, that specializes in precision oncology focused on virus-associated malignancies. The company utilizes a viral activation therapy platform to develop treatments for cancers linked to the Epstein-Barr Virus (EBV) and other virus-related diseases. Its lead investigational drug, nanatinostat, is being evaluated in combination with the antiviral valganciclovir as a potential oral therapy for EBV-positive lymphoma, including post-transplant lymphoproliferative disorder, plasmablastic lymphoma, and angioimmunoblastic T cell lymphoma. Viracta is also exploring the application of this approach to other EBV-associated conditions, such as nasopharyngeal carcinoma and gastric carcinoma. Founded in 2007 and rebranded from HemaQuest Pharmaceuticals in 2015, Viracta Therapeutics is actively conducting multiple clinical trials to advance its therapeutic candidates.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

Verve Therapeutics is a biotechnology company developing genetic medicines for cardiovascular disease, aiming to transform treatment from chronic management to single-course gene editing therapies. It leverages human genetics analysis and gene-editing technology, with expertise in cardiovascular medicine, human genetics, gene editing, delivery technologies, drug development, and commercialization.

Vera Therapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2016 and formerly known as Trucode Gene Repair, Inc. The company focuses on developing innovative biologic therapeutics with the potential to transform treatment for patients suffering from serious immunological diseases and genetic disorders. Its lead product candidate, atacicept, is a fusion protein administered as a subcutaneous injection once weekly, designed to inhibit factors that stimulate the production of autoantibodies associated with autoimmune diseases. Additionally, Vera Therapeutics is utilizing gene-editing technology aimed at addressing conditions such as sickle cell disease and cystic fibrosis, striving to enhance gene detection and regulation for improved patient outcomes.

Affinivax, Inc. is a biotechnology company based in Lexington, Massachusetts, specializing in the development of vaccines using its innovative Multiple Antigen Presenting System (MAPS) technology. Founded in 2014, the company aims to enhance the protection against a range of infectious diseases that affect both children and adults globally. MAPS technology allows for the effective binding of protective polysaccharides and proteins within a single vaccine, inducing robust immune responses. Affinivax is advancing vaccine programs targeting significant pathogens, including Streptococcus pneumoniae, which contributes to considerable morbidity and mortality worldwide. The company leverages insights from leading experts in infectious diseases and has secured exclusive intellectual property rights related to its MAPS platform from Boston Children’s Hospital, with notable backing from the Bill & Melinda Gates Foundation.

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

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

Immuneering Corporation is a biotechnology company based in Cambridge, Massachusetts, with an additional office in New York, New York. Founded in 2008, it specializes in genetic, genomic, and proteomic data analysis for pharmaceutical clients, aiming to enhance the clinical and commercial success of medicines. The company utilizes advanced techniques such as gene expression analysis, proteomics, and next-generation sequencing to uncover biological insights that inform drug development. Immuneering's expertise in translational bioinformatics is applied throughout the drug development process, particularly for oncologic and neurologic diseases, and is integral to its proprietary computational platform, known as Disease Cancelling Technology. This platform supports drug discovery initiatives and provides computational biology services to both pharmaceutical and biotechnology companies, contributing to the creation of therapies for serious illnesses, including cancer and autoimmune diseases.

Founded in 2018, Neuron23 is a biotechnology company based in South San Francisco, California, with an additional office in Munich, Germany. It specializes in developing and manufacturing medicines for genetic disorders using artificial intelligence technology and genetics.

Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for severe, rare muscle disorders with significant unmet medical need. It employs a holistic drug discovery approach targeting the muscle as an organ, utilizing its proprietary platform to identify precision medicines regulating key proteins in muscle tissue.

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

Nuvation Bio is a global biopharmaceutical company dedicated to developing innovative therapies for oncology, focusing on patients with difficult-to-treat cancers where conventional treatments have proven ineffective.

VectivBio AG is a clinical-stage biotechnology company based in Basel, Switzerland, founded in 2019 as a spinout from Therachon. The company specializes in the discovery, development, and commercialization of innovative treatments for severe rare diseases, particularly those with significant unmet medical needs. Its lead product candidate, Apraglutide, is a synthetic GLP-2 analog designed to enhance the intestine’s ability to absorb fluids and nutrients, thereby reducing the reliance on parenteral support in patients with short bowel syndrome. VectivBio is dedicated to developing best-in-disease therapies that aim to meaningfully improve the lives of patients and their families, providing more than just incremental advancements over existing standard-of-care options.

Rezolute Inc., established in 2010 and headquartered in Redwood City, California, is a clinical-stage biopharmaceutical company focused on developing therapies for rare and metabolic diseases. The company's primary product candidate, RZ358, is a human monoclonal antibody currently in Phase 2b trials for treating congenital hyperinsulinism, an ultra-rare pediatric genetic disorder. Additionally, Rezolute is advancing RZ402, a small molecule plasma kallikrein inhibitor in preclinical stages for diabetic macular edema treatment. The company aims to improve existing standards of care by applying proprietary formulation and manufacturing capabilities to known molecules.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

Flame Biosciences is a clinical-stage biotechnology company based in New York, founded in 2017. The company specializes in developing innovative therapies for cancer and other inflammatory conditions. Its portfolio includes FL-101 therapy, designed to treat lung cancers driven by inflammation, and FL-201, aimed at reducing chronic inflammation in patients. Flame Biosciences is dedicated to improving the lives of individuals suffering from debilitating and life-threatening diseases through its transformative therapeutic approaches.

BioShin Limited is a clinical-stage biopharmaceutical company focused on developing innovative medicines for neurological, neuropsychiatric diseases, and rare disorders. Established in 2018 and based in Pudong, China, BioShin operates as a wholly owned subsidiary of Biohaven Pharmaceutical Holding Company Ltd. The company is engaged in developing late-stage product candidates and offers clinical trial platforms that explore various therapeutic approaches, including calcitonin gene-related peptide receptor antagonism for migraine relief, glutamate modulation for Alzheimer’s disease and anxiety disorders, and myeloperoxidase inhibition targeting inflammation and neurodegeneration. By addressing significant unmet medical needs in neurological care, BioShin aims to make a substantial impact in the Asia-Pacific market.

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

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

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company based in San Mateo, California, focused on the discovery and development of innovative cancer therapeutics. Established in 2017, the company aims to address historically challenging cancer targets through precision medicine. Its primary product candidates include entospletinib, a selective inhibitor of spleen tyrosine kinase intended for acute myeloid leukemia patients, and KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9 for treating MYC-amplified solid tumors. Kronos Bio employs advanced technologies, such as high-throughput small-molecule microarrays and targeted protein degradation, to identify potent and selective compounds that target dysregulated transcription factors and other key drivers of oncogenic signaling.

Cerevel Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neuroscience diseases. Founded in 2018 and headquartered in Cambridge, Massachusetts, the company is advancing a diverse pipeline that includes five clinical-stage investigational therapies and several preclinical compounds targeting conditions such as schizophrenia, epilepsy, Parkinson's disease, and substance use disorder. Key products under development include CVL-231, a positive allosteric modulator for schizophrenia; CVL-865, which is being tested for drug-resistant focal onset epilepsy and acute anxiety; and Tavapadon, a selective dopamine D1/D5 partial agonist for Parkinson's disease. Cerevel Therapeutics employs a unique approach that leverages expertise in neurocircuitry and receptor selectivity to address unmet medical needs in the field of neuroscience.

Praxis Precision Medicines is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders marked by neuronal imbalance. Established in 2015 and headquartered in Cambridge, Massachusetts, the company leverages genetic insights to create innovative treatments for neurological and psychiatric conditions. Its leading product candidates include PRAX-114, an allosteric modulator targeting GABAA receptors, currently undergoing Phase IIa trials for major depressive disorder and perimenopausal depression, and PRAX-944, a selective inhibitor of T-type calcium channels in Phase IIa trials for essential tremor. Additionally, Praxis is advancing PRAX-562, a sodium current blocker in Phase I trials for severe pediatric epilepsy and adult cephalgia, along with PRAX-222, an antisense oligonucleotide aimed at treating patients with SCN2A epilepsy, and a program focused on KCNT1-related epilepsy. The company has established collaborations and licensing agreements with various organizations to enhance its research and development efforts.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

VelosBio, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-drug conjugates (ADCs) to treat various hematological cancers and solid tumors. Established in 2017, the company specializes in therapies that target receptor tyrosine kinase-like orphan receptor 1 (ROR1), a protein present on many cancerous cells but absent in normal tissues, making it a promising target for cancer treatment. VelosBio's lead product, VLS-101, is an ROR1-directed ADC designed for patients with both hematologic and solid tumor malignancies. The company's approach aims to provide novel targeted therapies that can be utilized as monotherapy or in combination with other treatments across a wide array of cancer types.

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with classical complement-mediated disorders affecting the body, brain, and eye. The company's pipeline is built on a platform technology that targets well-researched autoimmune and neurodegenerative disease processes triggered by the aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody designed for intravenous administration to treat autoimmune and neurodegenerative disorders. Another key candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration to address neurodegenerative ophthalmic disorders. The company employs a biomarker-driven development strategy to ensure that its product candidates effectively engage the target at therapeutic doses within the intended patient tissue, while also exploring additional orphan and large market indications.

Athira Pharma is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for brain disorders. Its lead product candidate, ATH-1017, aims to treat neurodegenerative diseases like Alzheimer's and Parkinson's by restoring neuronal health.

Mereo Biopharma, established in London in 2015, specializes in acquiring and advancing late-stage drug candidates from larger pharmaceutical companies. These candidates, often overlooked due to resource constraints, target rare and specialized diseases with significant unmet medical needs. Mereo's strategy involves rapidly progressing these assets through clinical development, either by partnering or divesting them, or directly commercializing them, particularly in orphan disease indications. The company combines operational efficiency with financial resources, leveraging a unique partnership with a leading global CRO, ICON, to conduct comprehensive clinical studies. Mereo's focus is on transforming the lives of patients worldwide by developing innovative medicines that can significantly improve their quality of life.

Pliant Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in South San Francisco, California, dedicated to discovering and developing innovative therapies for fibrotic diseases. Its lead product candidate, PLN-74809, is an oral small-molecule dual selective inhibitor of avß6 and avß1 integrins, targeting idiopathic pulmonary fibrosis and primary sclerosing cholangitis. PLN-74809 has received Orphan Drug Designation from the U.S. Food and Drug Administration for both indications and is currently undergoing Phase 2a clinical trials. The company is also developing PLN-1474, a small-molecule selective inhibitor of avß1, for the treatment of liver fibrosis associated with nonalcoholic steatohepatitis, which is in Phase 1 trials. Founded in 2015, Pliant Therapeutics is also advancing preclinical programs aimed at addressing oncology and muscular dystrophies.

Spruce Biosciences is a biopharmaceutical company focused on developing therapies for rare endocrine disorders. Its pipeline includes tildacerfont for classic congenital adrenal hyperplasia in adults and children, with ongoing clinical development, and Tralesinidase Alfa in its portfolio. The company collaborates with Eli Lilly to research, develop, and commercialize compounds. Founded in 2014, Spruce Biosciences is headquartered in Daly City, California, and aims to deliver meaningful therapies for patients with rare endocrine diseases.

Zentalis Pharmaceuticals is a clinical-stage biopharmaceutical company dedicated to developing small molecule therapeutics targeting cancer's underlying biological pathways. Its pipeline includes two lead product candidates: ZN-c5 for advanced estrogen receptor-positive breast cancer, and ZN-c3 for advanced solid tumors and platinum-resistant ovarian cancer.

Pieris Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on the development of anticalin-based therapeutics for treating various medical conditions, including cancer and severe asthma. Anticalins are engineered proteins derived from lipocalins, naturally occurring human proteins that can bind and transport a variety of molecules. The company's lead programs include PRS-060, a drug candidate in Phase I clinical trials targeting IL-4Ra for asthma and inflammatory diseases, and PRS-343, a bispecific protein in Phase I trials for oncology. Pieris is also advancing PRS-344, a bispecific anticalin-antibody fusion protein in preclinical development, and PRS-080, currently in Phase IIa trials for targeting hepcidin in patients with functional iron deficiency. Pieris collaborates with strategic partners and research institutions to enhance its development efforts, showcasing a robust pipeline aimed at addressing significant unmet medical needs. The company was founded in 2000 and is recognized for its innovative approach in biotherapeutics.