Arbor Biotechnologies, Inc., founded in 2016 and based in Cambridge, Massachusetts, operates within the biotechnology sector, focusing on bio-discovery and human diagnostic development. The company utilizes a range of advanced technologies, including artificial intelligence, genome sequencing, gene synthesis, and high-throughput screening, to facilitate the discovery of proteins aimed at enhancing human health and sustainability. Arbor's proprietary genomic tools leverage AI and machine learning to analyze genomic sequence data, thereby improving the efficiency of developing curative treatments for genetic diseases. These innovations enable drug developers to replace entire genes and precisely correct mutations, ultimately addressing previously untreatable genetic conditions.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.
Aviceda Therapeutics is a late-stage, pre-clinical biotechnology company focused on developing innovative treatments for glyco-immune diseases. By leveraging glycobiology and a proprietary cell-based high-throughput screening platform, the company aims to create next-generation immunomodulators that target the innate immune system and address chronic non-resolving inflammation. Aviceda's product pipeline includes candidates for a variety of conditions, such as eye diseases, fibrosis, neurology, and oncology. Key products under development include AVD-104, AVD-302, and AVD-401 for dry eye, diabetic retinopathy, and retinoblastoma, as well as AVD-601, AVD-701, and AVD-801 targeting oncology, neurology, and fibrosis. Through its advanced technologies and biological insights, Aviceda seeks to provide breakthrough glycol-therapeutics that improve patient outcomes.
RAPT Therapeutics is a clinical-stage biopharmaceutical company specializing in immunology. It focuses on discovering, developing, and commercializing oral small-molecule therapies aimed at addressing significant unmet medical needs in oncology and inflammatory diseases. RAPT's proprietary discovery engine has led to the advancement of two drug candidates: FLX475, which selectively inhibits the migration of immunosuppressive regulatory T cells into tumors, and RPT193, which targets type 2 T helper cells in inflamed tissues. Both candidates have progressed to clinical trials within a short time frame since the company's inception. Additionally, RAPT is exploring further therapeutic targets, including general control nonderepressible 2 and hematopoietic progenitor kinase 1, which are currently in the discovery phase.
ADCendo ApS is a biotechnology company focused on developing innovative anti-cancer drugs through the use of antibody-drug conjugates (ADCs). The company targets novel receptors that are overexpressed in various cancer types, addressing significant unmet medical needs in the field of oncology. Its ADCs aim to provide targeted therapy for patients suffering from aggressive cancers such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer, as well as other carcinomas including those of the breast, colon, pancreas, prostate, ovary, and kidney. Through its research and development efforts, ADCendo strives to create effective treatment options for patients facing challenging cancer diagnoses.
Cidara Therapeutics is a biotechnology company based in San Diego, California, established in 2012. The company specializes in the discovery and development of innovative anti-infective therapies for serious diseases, particularly those that affect individuals with compromised immune systems. Its lead candidate, rezafungin acetate, is an antifungal agent designed to treat and prevent severe invasive fungal infections, such as candidemia and invasive candidiasis, which carry significant mortality risks. In addition to its antifungal efforts, Cidara is advancing its proprietary Cloudbreak platform, which focuses on developing antiviral Fc-conjugates aimed at preventing and treating various viral infections, including influenza, RSV, HIV, and coronavirus. Cidara's mission is to transform treatment and prevention strategies for life-threatening illnesses.
ViceBio is a biopharmaceutical company focused on developing innovative vaccines to combat respiratory viral infectious diseases. Utilizing its patented Molecular Clamp Technology, ViceBio stabilizes viral glycoproteins in their prefusion conformation, which allows for the creation of effective and ready-to-use liquid vaccines. This technology enables long-term storage and easy administration of vaccines targeting various respiratory viruses, including respiratory syncytial virus (RSV), metapneumovirus, influenza, and SARS-CoV-2. Through its commitment to advancing vaccination strategies, ViceBio aims to prevent potentially fatal infections caused by these viruses.
Candid Therapeutics is a biotechnology company that focuses on the treatment of a range of immunological conditions.
Pathalys is a clinical biopharmaceutical company that develops therapeutics that improve late-stage chronic kidney disease. Pathalys' initial focus will be on improving treatment for secondary hyperparathyroidism, which is an unmet need (SHPT). SHPT is a condition in which a disease outside of the parathyroid glands causes all of the parathyroid glands to swell and become hyperactive, producing an excessive amount of parathyroid hormone (PTH).
Savara is an orphan lung disease company. Savara’s pipeline comprises Molgradex, an inhaled granulocyte-macrophage colony-stimulating factor, or GM-CSF, in Phase 3 development for autoimmune pulmonary alveolar proteinosis, or aPAP, in Phase 2a development for nontuberculous mycobacteria, or NTM, lung infection, and in preparation for Phase 2a development in cystic fibrosis, or CF, affected individuals with chronic NTM lung infection; and AeroVanc, a Phase 3 stage inhaled vancomycin for treatment of persistent methicillin resistant staphylococcus aureus, or MRSA, lung infection in CF. Savara’s strategy involves expanding its pipeline of potentially best-in-class products through indication expansion, strategic development partnerships and product acquisitions, with the goal of becoming a leading company in its field. The most recent acquisition is aerosolized amikacin/fosfomycin, a Phase 2 ready, proprietary combination antibiotic, which has demonstrated potent and broad-spectrum antibacterial activity against highly drug resistant pathogens. Savara’s management team has significant experience in orphan drug development and pulmonary medicine, identifying unmet needs, developing and acquiring new product candidates, and effectively advancing them to approvals and commercialization.
Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, founded in 2017. The company focuses on developing human genetics-based therapeutics aimed at addressing ineffective red blood cell production in hematologic diseases. Its innovative pipeline includes therapies such as bitopertin for treating erythropoietic porphyrias and Diamond-Blackfan anemia. Disc Medicine targets the hepcidin metabolism axis and aims to modify fundamental biological pathways related to red blood cell formation and function, including heme biosynthesis and iron homeostasis. Through its portfolio of clinical and preclinical product candidates, the company strives to transform the lives of patients suffering from severe hematologic disorders.
Pheon Therapeutics is an Antibody-Drug Conjugate (ADC) specialist that creates ADCs for a variety of difficult-to-treat cancers. Its lead program is a first-in-class ADC against a novel target that is highly expressed in solid tumors across a broad range of hard-to-treat cancer types. The company was founded in 2022 and is based in London, England.
Obsidian Therapeutics is a developer of a next-generation cell and gene therapies created to extend adoptive immunotherapy to every patient with cancer.The company's next-generation cell and gene therapies with pharmacologic operating systems provide exquisite control of protein activity in cells and create adoptive cell therapies with new functions that are under control of the treating physician using simple, safe, orally-active, marketed drugs, enabling patients to improve on the current generation of cell therapies.
Avalo Therapeutics is a clinical-stage biopharmaceutical company that employs a precision medicine approach to discover, develop, and commercialize highly targeted therapeutics in areas of significant unmet clinical need.
Mirador Therapeutics envisions precision medicine for immune-mediated inflammatory and fibrotic diseases.
Ocular Therapeutix, Inc. is a biopharmaceutical company focused on developing and commercializing innovative therapies for eye diseases using its proprietary bioresorbable hydrogel platform technology. The company offers several products, including ReSure Sealant, a hydrogel ophthalmic wound sealant designed to protect corneal incisions after cataract surgery, and DEXTENZA, an ophthalmic insert for the treatment of post-surgical ocular pain and inflammation, as well as allergic conjunctivitis and dry eye diseases. Ocular Therapeutix is also developing OTX-TP, an insert aimed at reducing intraocular pressure in glaucoma patients, and OTX-TIC, an intracameral implant for similar indications, along with OTX-TKI, an intravitreal implant targeting wet age-related macular degeneration. In addition, the company has preclinical programs focused on various ocular conditions. Ocular Therapeutix collaborates with Regeneron Pharmaceuticals to integrate its hydrogel technology with Regeneron's VEGF-targeting compounds for retinal disease treatments. Founded in 2006, the company is headquartered in Bedford, Massachusetts.
Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.
Mineralys Therapeutics is a biopharmaceutical company that provides hypertension treatment. They also bring a targeted approach to the management of hypertension.
Adverum Biotechnologies is a clinical-stage gene therapy company focused on developing innovative treatments for ocular and rare diseases. The company's pipeline includes several product candidates, notably ADVM-022, which targets wet age-related macular degeneration through a single intravitreal injection designed to deliver long-lasting therapeutic effects. Other candidates include ADVM-043 for alpha-1 antitrypsin deficiency and ADVM-053 for hereditary angioedema. Adverum collaborates with Editas Medicine to utilize its proprietary AAV vectors for gene editing in inherited retinal diseases, and with Regeneron Pharmaceuticals for developing various ocular therapies. Founded in 2006 and headquartered in Redwood City, California, Adverum aims to establish gene therapy as a new standard of care, aspiring to restore vision and prevent blindness in affected patients.
Silence Therapeutics is a biotechnology company based in London, focused on the discovery, development, and delivery of innovative RNA therapeutics for various medical conditions, including hematology, cardiovascular diseases, and rare metabolic disorders. The company specializes in designing short interfering RNA (siRNA) molecules that activate the RNA interference (RNAi) pathway to selectively degrade target messenger RNAs. Its product pipeline includes SLN124, aimed at treating iron overload disorders, SLN360, which targets cardiovascular diseases associated with elevated lipoprotein levels, and SLN500 for complement-mediated diseases. Silence Therapeutics has established strategic collaborations with AstraZeneca to develop small interfering RNA therapeutics for a range of diseases, and with Genomics England Limited. The company was previously known as SR Pharma before rebranding in 2007.
Astria Therapeutics is a biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative therapies for rare and niche allergic and immunological diseases. The company focuses on bringing life-changing treatments to patients and families affected by these conditions. Its lead program, STAR-0215, is a monoclonal antibody that inhibits plasma kallikrein and is currently in preclinical development for hereditary angioedema. Additionally, Astria is developing STAR-0310, a monoclonal antibody OX40 antagonist, which is also in preclinical stages and targets atopic dermatitis, an immune disorder characterized by compromised skin barrier function and associated itching.
LB Pharmaceuticals Inc is a New York-based life sciences company focused on discovering and developing drugs for central nervous system disorders, particularly schizophrenia. Established in 2015, the company aims to commercialize novel and improved versions of existing CNS treatments that have not been developed or marketed in the United States. Its primary asset, LB-102 (n-Methyl amisulpride), is a patented reformulation of amisulpride, a drug recognized for its effectiveness in treating schizophrenia. By employing a pro-drug approach, LB Pharmaceuticals modifies the chemical structure of amisulpride to enhance its efficacy and safety, thereby offering improved treatment options for patients who do not respond well to current medications. The company is dedicated to generating innovative intellectual property and advancing its research-focused agenda in the pharmaceutical landscape.
Tourmaline Bio is a late-stage clinical biotechnology company focused on developing transformative medicines aimed at improving the lives of patients with immune diseases. Its primary product candidate, TOUR006, is a fully human monoclonal antibody that targets interleukin-6 (IL-6), a significant proinflammatory cytokine implicated in various autoimmune and inflammatory disorders. The company leverages over twenty years of clinical and commercial experience associated with the anti-IL-6 and anti-IL-6 receptor antibody class, which has effectively treated over a million patients suffering from a range of autoimmune conditions. Tourmaline Bio is committed to addressing the needs of individuals with life-altering immune diseases through its innovative therapeutic approaches.
VistaGen Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for diseases and disorders of the central nervous system, particularly those related to psychiatric conditions such as depression and anxiety. The company's main product candidates include PH94B, a neuroactive nasal spray poised for Phase III trials aimed at treating acute anxiety in adults with social anxiety disorder, and PH10, another nasal spray in preparation for Phase 2b development for major depressive disorder. Additionally, AV-101, an oral prodrug candidate, is being developed for multiple indications, including major depressive disorder, suicidal ideation, and neuropathic pain. VistaGen has established various licensing and collaboration agreements with other firms to enhance its research and development efforts, including a strategic partnership for the clinical development of PH94B in select Asian markets. Founded in 1998, VistaGen is headquartered in South San Francisco, California.
Inhibrx, Inc. is a clinical-stage biotechnology company based in La Jolla, California, dedicated to developing a diverse pipeline of novel biologic therapeutic candidates. The company employs advanced protein engineering techniques, including its proprietary sdAb platform, to target complex disease biology, with a focus on oncology, orphan diseases, and infectious diseases. Inhibrx's lead candidates include INBRX-109, a multivalent agonist in Phase 1 trials for solid tumors; INBRX-105, an antagonist of PD-L1 also in Phase 1 trials; INBRX-101, an Fc-fusion protein for alpha-1 antitrypsin deficiency; and INBRX-103, a monoclonal antibody targeting CD47. Additionally, the company is developing preclinical programs such as INBRX-106, an agonist of OX40, and INBRX-111, an antibody targeting Pseudomonas aeruginosa. Inhibrx has established collaborations with organizations like Celgene and Bluebird Bio and has received funding from various granting agencies, including the NIH and CARB-X.
COMPASS Pathways plc is a mental health care company based in the United Kingdom and the United States, focusing on developing innovative therapies for treatment-resistant depression. The company is conducting Phase IIb clinical trials for its psilocybin formulation, COMP360, which aims to provide a new treatment option for patients who have not responded to conventional therapies. By researching psilocybin-assisted therapy, COMPASS Pathways seeks to assess its safety and efficacy, while combining the psychedelic compound with psychological support. The company was incorporated in 2020 and aims to address the significant unmet need for effective and accessible mental health treatments, striving to empower individuals facing mental health challenges.
AN2 Therapeutics, Inc. is a biopharmaceutical company based in Menlo Park, California, founded in 2017. The company is dedicated to the research, development, and commercialization of innovative medicines aimed at addressing infectious diseases. AN2 Therapeutics is focused on developing epetraborole, a once-daily oral treatment for patients suffering from chronic non-tuberculous mycobacterial lung disease, which represents a significant unmet medical need. Additionally, the company has established a strategic partnership with Brii Biosciences to enhance its development efforts.
Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.
ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.
Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company based in Shanghai, China, focused on discovering and developing cell therapies for cancer treatment. The company’s lead product candidates include GC012F, a dual-target autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia and is also being investigated for relapsed or refractory B cell non-Hodgkin’s lymphoma. Additionally, Gracell is developing GC007F for B cell non-Hodgkin's lymphoma and GC027, an allogeneic CAR-T product candidate for adult T cell acute lymphoblastic leukemia. The company also has GC007g, a donor-derived therapy for relapsed or refractory B cell acute lymphoblastic leukemia, and a pipeline of earlier stage candidates targeting other cancer types, including ovarian cancer and breast cancer. Founded in 2017, Gracell aims to provide innovative cellular therapeutics that enhance treatment options for patients suffering from various hematological malignancies and solid tumors.
CG Oncology is a clinical-stage biopharmaceutical company dedicated to developing innovative immunotherapies for cancer treatment, particularly focusing on bladder cancer. The company specializes in oncolytic immunotherapies, with its lead candidate, cretostimogene grenadenorepvec, designed as a targeted, intravesical immunotherapy. Currently, CG Oncology is conducting multiple clinical trials, including two phase three trials aimed at high-risk patients with non-muscle invasive bladder cancer (NMIBC) who are unresponsive to BCG therapy, as well as an additional phase two study in combination with a checkpoint inhibitor. The company aims to provide effective bladder-sparing treatment options for patients while advancing the development of its proprietary therapies.
Upstream Bio is a clinical-stage biotechnology company dedicated to developing antibody therapies for inflammatory diseases, particularly severe asthma and other respiratory disorders. The company's lead candidate, verekitug, is a monoclonal antibody that acts as an antagonist to the Thymic Stromal Lymphopoietin (TSLP) receptor. TSLP is a key cytokine that drives inflammatory responses and is strategically positioned upstream of various signaling pathways influencing immune cells. Upstream Bio aims to address unmet medical needs in the treatment of severe respiratory conditions by leveraging this validated target to create effective therapies.
Alkeus Pharmaceuticals is a Boston-based clinical-stage biopharmaceutical company dedicated to developing treatments for serious and untreatable ophthalmic conditions. The company's primary focus is on its lead drug candidate, an orally delivered compound aimed at addressing Stargardt disease and age-related macular degeneration. This innovative treatment has the potential to help medical professionals manage symptoms of irreversible vision loss early in life, offering hope to patients affected by these debilitating eye diseases.
Carmot Therapeutics, Inc. is a clinical-stage biotechnology company based in Berkeley, California, with an additional office in San Francisco. Founded in 2008, the company focuses on discovering and developing therapies for metabolic diseases, including obesity and diabetes. Carmot employs a proprietary drug discovery approach known as Chemotype Evolution, which enables the exploration of novel chemical and biological frontiers. The company is advancing a portfolio of clinical-stage drug candidates, including CT-388, a once-weekly injectable dual GLP-1/GIP receptor agonist; CT-996, a once-daily oral GLP-1 receptor agonist; and CT-868, a once-daily injectable dual GLP-1/GIP receptor agonist for treating type 1 diabetes in patients with overweight or obesity. These innovative therapies aim to enhance glucose clearance and promote weight loss, addressing the fundamental causes of metabolic diseases.
Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines using its innovative virus-like particle (VLP) technology platform, which allows for the multivalent display of complex viral antigens. This technology aims to provide broad and durable protection against various infectious diseases, with an initial focus on life-threatening respiratory illnesses. Icosavax's pipeline includes vaccine candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and SARS-CoV-2. The company is dedicated to advancing its VLP technology to discover, develop, and ultimately commercialize effective vaccines against these infectious diseases.
Abivax is a clinical-stage biotechnology company based in France that develops therapeutics aimed at harnessing the body's natural immune response to treat autoimmune diseases, viral infections, and cancer. The company is currently evaluating its drug candidate, obefazimod, in Phase 3 clinical trials for the treatment of adults with moderately to severely active ulcerative colitis. Abivax focuses on addressing significant unmet medical needs in chronic inflammatory diseases, particularly inflammatory bowel diseases, which include ulcerative colitis and Crohn's disease. Through its innovative drug development platforms, Abivax aims to provide novel and effective treatment options for patients suffering from these conditions.
Tourmaline Bio is a late-stage clinical biotechnology company focused on developing transformative medicines aimed at improving the lives of patients with immune diseases. Its primary product candidate, TOUR006, is a fully human monoclonal antibody that targets interleukin-6 (IL-6), a significant proinflammatory cytokine implicated in various autoimmune and inflammatory disorders. The company leverages over twenty years of clinical and commercial experience associated with the anti-IL-6 and anti-IL-6 receptor antibody class, which has effectively treated over a million patients suffering from a range of autoimmune conditions. Tourmaline Bio is committed to addressing the needs of individuals with life-altering immune diseases through its innovative therapeutic approaches.
VectivBio AG is a clinical-stage biotechnology company based in Basel, Switzerland, founded in 2019 as a spinout from Therachon. The company specializes in developing innovative treatments for severe rare diseases with significant unmet medical needs. Its primary product candidate, Apraglutide, is a long-acting synthetic analog of glucagon-like peptide-2 (GLP-2) designed to enhance the intestine's ability to absorb fluids and nutrients, thereby reducing the reliance on parenteral support. VectivBio aims to deliver transformational therapies that significantly improve the lives of patients and their families, rather than offering only incremental benefits over existing treatments.
Immunovant is a clinical-stage biopharmaceutical company focused on enabling normal lives for patients with autoimmune diseases. Its investigational product candidate, IMVT-1401 (formerly known as RVT-1401), is a novel, fully human monoclonal antibody targeting the neonatal Fc receptor (FcRn). We believe that this product has the potential to address a variety of IgG-mediated autoimmune diseases as a subcutaneous injection.
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.
Abivax is a clinical-stage biotechnology company based in France that develops therapeutics aimed at harnessing the body's natural immune response to treat autoimmune diseases, viral infections, and cancer. The company is currently evaluating its drug candidate, obefazimod, in Phase 3 clinical trials for the treatment of adults with moderately to severely active ulcerative colitis. Abivax focuses on addressing significant unmet medical needs in chronic inflammatory diseases, particularly inflammatory bowel diseases, which include ulcerative colitis and Crohn's disease. Through its innovative drug development platforms, Abivax aims to provide novel and effective treatment options for patients suffering from these conditions.
Aktis Oncology is a biotechnology company pioneering the discovery and development of a new class of targeted radiopharmaceuticals to treat a broad range of solid tumor cancers. Founded and incubated by MPM Capital, the company has developed proprietary platforms to generate tumor-targeting agents with ideal properties for alpha radiotherapy. Designed for high tumor penetration and long residence time, Aktis Oncology's molecules will quickly clear other areas of the body, thereby maximizing tumor elimination while minimizing the side effects of treatment. This approach would also enable clinicians to visualize and verify target engagement prior to exposure to therapeutic radioisotopes.
Upstream Bio is a clinical-stage biotechnology company dedicated to developing antibody therapies for inflammatory diseases, particularly severe asthma and other respiratory disorders. The company's lead candidate, verekitug, is a monoclonal antibody that acts as an antagonist to the Thymic Stromal Lymphopoietin (TSLP) receptor. TSLP is a key cytokine that drives inflammatory responses and is strategically positioned upstream of various signaling pathways influencing immune cells. Upstream Bio aims to address unmet medical needs in the treatment of severe respiratory conditions by leveraging this validated target to create effective therapies.
Plexium, Inc. is a biotechnology company based in San Diego, California, that specializes in developing targeted protein degradation therapies aimed at treating cancer and neurodegenerative diseases. Founded in 2017, Plexium utilizes a platform that identifies small molecules capable of modulating E3 ligases, which play a crucial role in protein degradation. This platform includes the DELPhe screening technology, designed for high-content readouts from cell-based assays. By harnessing E3 ligases, Plexium's approach enables selective targeting of various cancers, including small cell lung and breast tumors. The company's innovative research extends beyond existing therapies, exploring new drug-like protein degraders and molecular glues to enhance treatment options for cancer and inflammatory diseases.
Arbor Biotechnologies, Inc., founded in 2016 and based in Cambridge, Massachusetts, operates within the biotechnology sector, focusing on bio-discovery and human diagnostic development. The company utilizes a range of advanced technologies, including artificial intelligence, genome sequencing, gene synthesis, and high-throughput screening, to facilitate the discovery of proteins aimed at enhancing human health and sustainability. Arbor's proprietary genomic tools leverage AI and machine learning to analyze genomic sequence data, thereby improving the efficiency of developing curative treatments for genetic diseases. These innovations enable drug developers to replace entire genes and precisely correct mutations, ultimately addressing previously untreatable genetic conditions.
Structure Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel oral therapeutics for chronic diseases with significant unmet medical needs. Utilizing advanced computational and structure-based technology, the company designs orally available small molecules that aim to overcome the limitations of traditional biologic and peptide drugs. Its lead product candidate, GSBR-1290, is a small molecule agonist targeting the glucagon-like peptide-1 receptor, which is relevant for treating type-2 diabetes and obesity. Additionally, Structure Therapeutics is advancing other oral therapeutics targeting G-protein-coupled receptors for conditions such as pulmonary and cardiovascular diseases, including candidates ANPA-0073 and LTSE-2578. The company combines expertise in drug design and development to create differentiated and effective treatments that can have a profound impact on patient care.
Alpine Immune Sciences is a clinical-stage biopharmaceutical company based in Seattle, Washington, specializing in the discovery and development of protein-based immunotherapies for cancer, autoimmune, and inflammatory disorders. The company employs a proprietary platform to transform native immune system proteins into multi-targeted therapeutics. Its lead programs include ALPN-101, a dual antagonist targeting the inducible T cell costimulator (ICOS) and CD28 pathways, aimed at treating autoimmune and inflammatory diseases, and ALPN-303, a dual antagonist of the B cell activating factor (BAFF) and the proliferation-inducing ligand (APRIL), which are crucial for B cell activation and survival. Additionally, Alpine has developed ALPN-202, an antagonist targeting programmed cell death protein ligand 1 (PD-L1) and cytotoxic T-lymphocyte associated protein 4 (CTLA-4), designed to enhance cancer treatment. The company also collaborates with Kite Pharma to advance immunotherapy approaches targeting the immune synapse in cancer therapy.
Obsidian Therapeutics is a developer of a next-generation cell and gene therapies created to extend adoptive immunotherapy to every patient with cancer.The company's next-generation cell and gene therapies with pharmacologic operating systems provide exquisite control of protein activity in cells and create adoptive cell therapies with new functions that are under control of the treating physician using simple, safe, orally-active, marketed drugs, enabling patients to improve on the current generation of cell therapies.
Versanis Bio is focused on discovering and developing medicines that target medical conditions commonly found in older adults. The company is advancing Bimagrumab, a human monoclonal antibody that inhibits activin type II receptors, blocking the action of ligands such as activin A and myostatin. Previous studies conducted by Novartis involved over 1,500 participants and demonstrated that Bimagrumab consistently induced significant fat loss while preserving lean mass. Additionally, the treatment has shown improvements in HbA1c levels and other cardiometabolic parameters, offering a promising option for overweight and obese adults aiming to achieve and maintain a healthier body composition.
Artios Pharma Limited is a biotechnology company based in Cambridge, United Kingdom, that specializes in developing innovative cancer treatments targeting DNA damage response (DDR) pathways. Founded in 2015, the company is dedicated to creating first-in-class therapies aimed at selectively killing cancer cells. Artios's product pipeline features several promising programs, including a focus on DNA polymerase theta (Polθ), which plays a crucial role in DNA repair processes. Additionally, the company is advancing an in-licensed program that targets a newly identified protein linked to DDR and is developing a novel treatment aimed at DNA nucleases involved in these pathways. Artios collaborates with Cancer Research Technology and prominent DNA repair researchers globally to enhance its research and development efforts.
RayzeBio, Inc. is a biotechnology company based in San Diego, California, founded in 2020. It specializes in developing tumor-targeted small molecule medicines that utilize the therapeutic potential of radioisotopes, particularly Actinium-225, an alpha-emitting radioisotope aimed at treating solid tumors. RayzeBio is focused on enhancing cancer treatment outcomes by creating a robust pipeline of radiopharmaceutical candidates targeting validated oncology drug targets. The company's portfolio includes a range of drug candidates at various stages of development, from discovery to late-stage clinical programs, addressing significant market opportunities in the field of cancer therapeutics.
Synthekine Inc. is a biotechnology company focused on developing innovative cytokine therapies and immunotherapies for cancer and autoimmune disorders. Established in 2018 and located in Menlo Park, California, the company employs advanced platforms such as engineered partial agonists and orthogonal cell therapies. Its Synthekine Platform utilizes surrogate agonists instead of traditional mutant cytokines, allowing for the creation of therapeutics that enhance efficacy while minimizing side effects. The product pipeline includes STK-009, an orthogonal ligand, and SYNCAR-001, a CAR-T therapy targeting CD-19, along with STK-012, which consists of partial agonists of IL-2. Synthekine aims to revolutionize treatment paradigms by harnessing the principles of cytokine partial agonism and immunological specificity, leading to novel immunotherapies that improve patient outcomes.
Magenta Therapeutics is a clinical-stage biotechnology company focused on developing innovative therapies to enhance stem cell transplantation for patients with blood cancers, genetic diseases, and autoimmune disorders. The company aims to improve the safety and efficacy of stem cell transplants, which are currently high-risk procedures often considered only as a last resort. Its pipeline includes several key product candidates, such as targeted antibody-drug conjugates for transplant conditioning, a novel stem cell mobilization agent, and allogeneic stem cell therapies. Magenta is also exploring collaborations to assess the effectiveness of its therapies in combination with existing treatments, such as gene therapies for sickle cell disease. Founded in 2015 and headquartered in Cambridge, Massachusetts, Magenta Therapeutics seeks to expand the curative potential of stem cell therapies to a broader patient population by addressing the toxicity and long-term side effects associated with current transplant methods.
Eikon Therapeutics, Inc. is a biopharmaceutical company based in Hayward, California, focused on developing innovative treatments for chronic and life-threatening diseases. Founded in 2019, the company employs advanced super-resolution microscopy and live-cell imaging techniques to discover drugs targeting previously intractable proteins. By integrating biology, engineering, and chemistry, Eikon Therapeutics aims to enhance the understanding of cellular processes and improve drug discovery efficiency. Its proprietary platforms allow for the sensitive identification of compound-protein interactions, enabling the direct measurement of how chemical compounds affect individual proteins in live cells. This novel approach seeks to unlock new therapeutic opportunities and expand the company's pipeline of potential medications.
Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.
Entrada Therapeutics, Inc. is a biotechnology company focused on treating diseases through the intracellular delivery of biologics, such as proteins, peptides, and nucleic acids. Founded in 2016 and based in Boston, Massachusetts, the company aims to transform patient care by developing a new class of medicines that target intracellular mechanisms previously deemed inaccessible. Entrada's technology addresses challenges associated with delivering both large and small molecules, significantly enhancing the efficiency of therapeutic delivery into the cytosol. Its pipeline includes innovative therapies for neuromuscular diseases and other conditions, utilizing proprietary Endosomal Escape Vehicle (EEV) therapeutics to improve treatment outcomes across various organs and tissues.
Aktis Oncology is a biotechnology company pioneering the discovery and development of a new class of targeted radiopharmaceuticals to treat a broad range of solid tumor cancers. Founded and incubated by MPM Capital, the company has developed proprietary platforms to generate tumor-targeting agents with ideal properties for alpha radiotherapy. Designed for high tumor penetration and long residence time, Aktis Oncology's molecules will quickly clear other areas of the body, thereby maximizing tumor elimination while minimizing the side effects of treatment. This approach would also enable clinicians to visualize and verify target engagement prior to exposure to therapeutic radioisotopes.