Undisclosed
Neurodegenerative Diseases
Key Facts
About Modulo Bio
Modulo Bio is a private, preclinical-stage biotech founded in 2021 and based in San Francisco. The company is pioneering a novel approach to neurodegenerative diseases by targeting the neuroimmune system, specifically the function of microglia. Its integrated platform utilizes human stem cell-derived models and advanced computational analytics to discover drug targets and candidates that can enhance microglia-mediated neuroprotection. Modulo is positioned to address a massive unmet medical need in neurology by developing first-in-class small molecule therapeutics.
View full company profileAbout Vaxxinity
Vaxxinity (NASDAQ: VAXX) is a U.S.-based biotech firm with a mission to democratize healthcare through its proprietary Active Immunotherapy Medicine (AIM) platform. The company has achieved significant validation, including a successful Phase 3 trial for a COVID-19 booster and billions of doses commercialized in animal health, underpinning its strategy to develop long-acting, scalable therapies for large markets like Alzheimer's and hypercholesterolemia. Its core differentiator is the ability to safely break immune tolerance against self-antigens, a key hurdle in chronic disease treatment, using a synthetic, low-cost peptide approach that requires no frozen cold chain.
View full company profileAbout Biossil
AI-native biopharma company advancing 11 Phase 2 & 3 programs across life-threatening diseases using synthetic biology and metabolic engineering.
View full company profileAbout Biossil
AI-native biopharma company advancing 11 Phase 2 & 3 programs across life-threatening diseases using synthetic biology and metabolic engineering.
View full company profileAbout Biossil
AI-native biopharma company advancing 11 Phase 2 & 3 programs across life-threatening diseases using synthetic biology and metabolic engineering.
View full company profileAbout Biossil
AI-native biopharma company advancing 11 Phase 2 & 3 programs across life-threatening diseases using synthetic biology and metabolic engineering.
View full company profileAbout Biossil
AI-native biopharma company advancing 11 Phase 2 & 3 programs across life-threatening diseases using synthetic biology and metabolic engineering.
View full company profileAbout Biossil
AI-native biopharma company advancing 11 Phase 2 & 3 programs across life-threatening diseases using synthetic biology and metabolic engineering.
View full company profileAbout Biossil
AI-native biopharma company advancing 11 Phase 2 & 3 programs across life-threatening diseases using synthetic biology and metabolic engineering.
View full company profileAbout Biossil
AI-native biopharma company advancing 11 Phase 2 & 3 programs across life-threatening diseases using synthetic biology and metabolic engineering.
View full company profileAbout Biossil
AI-native biopharma company advancing 11 Phase 2 & 3 programs across life-threatening diseases using synthetic biology and metabolic engineering.
View full company profileAbout Biossil
AI-native biopharma company advancing 11 Phase 2 & 3 programs across life-threatening diseases using synthetic biology and metabolic engineering.
View full company profileAbout Biossil
AI-native biopharma company advancing 11 Phase 2 & 3 programs across life-threatening diseases using synthetic biology and metabolic engineering.
View full company profileAbout Corstasis Therapeutics
Corstasis Therapeutics is an early-stage biotech company targeting the significant unmet need for home-based fluid removal in patients with congestive heart failure and related organ diseases. The company is building a leadership team and advisory board with deep expertise in cardiovascular pharmaceuticals, medical devices, and clinical cardiology. While specific pipeline details are not publicly disclosed, its mission centers on shifting fluid overload management from inpatient to outpatient settings, potentially improving patient quality of life and reducing healthcare system burdens.
View full company profileAbout AAVantgarde Bio
AAVantgarde Bio is a private, pre-clinical stage biotech developing next-generation AAV vectors to overcome key limitations of current gene therapy delivery systems, such as immunogenicity, tissue specificity, and payload capacity. Its platform aims to create safer and more effective therapies for challenging neurological and rare genetic disorders, positioning it in a high-growth segment of the biopharmaceutical industry. As a young company, it is likely in a capital-intensive R&D phase, seeking partnerships and investment to advance its proprietary technology and pipeline programs toward clinical validation.
View full company profileAbout Lumen Bioscience
Lumen Bioscience is pioneering a disruptive platform for oral biologic drugs using engineered spirulina, which promises faster, cheaper, and more scalable development compared to traditional cell-culture methods. The company's lead program, LMN-801, is a Phase 2 oral hormone therapy for weight loss, with additional Phase 2/3 programs targeting C. difficile infection and oxalate malabsorption. Backed by $185 million in investment and a robust patent portfolio, Lumen aims to address large, underserved markets by making biologic therapies more accessible and affordable.
View full company profileAbout Lumen Bioscience
Lumen Bioscience is pioneering a disruptive platform for oral biologic drugs using engineered spirulina, which promises faster, cheaper, and more scalable development compared to traditional cell-culture methods. The company's lead program, LMN-801, is a Phase 2 oral hormone therapy for weight loss, with additional Phase 2/3 programs targeting C. difficile infection and oxalate malabsorption. Backed by $185 million in investment and a robust patent portfolio, Lumen aims to address large, underserved markets by making biologic therapies more accessible and affordable.
View full company profileAbout GondolaBio
GondolaBio is a private, clinical-stage biotech company based in San Diego, leveraging a decentralized model to rapidly advance a diverse pipeline of small molecule therapies for genetic diseases. Its lead asset, PORT-77, is a Phase 2 oral ABCG2 inhibitor for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), with FDA Orphan Drug and Fast Track designations. The company's strategy centers on identifying clear genetic markers to target diseases at their source, aiming to address the significant unmet need where only 5% of genetic conditions have approved therapies.
View full company profileAbout GondolaBio
GondolaBio is a private, clinical-stage biotech company based in San Diego, leveraging a decentralized model to rapidly advance a diverse pipeline of small molecule therapies for genetic diseases. Its lead asset, PORT-77, is a Phase 2 oral ABCG2 inhibitor for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), with FDA Orphan Drug and Fast Track designations. The company's strategy centers on identifying clear genetic markers to target diseases at their source, aiming to address the significant unmet need where only 5% of genetic conditions have approved therapies.
View full company profileAbout GondolaBio
GondolaBio is a private, clinical-stage biotech company based in San Diego, leveraging a decentralized model to rapidly advance a diverse pipeline of small molecule therapies for genetic diseases. Its lead asset, PORT-77, is a Phase 2 oral ABCG2 inhibitor for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), with FDA Orphan Drug and Fast Track designations. The company's strategy centers on identifying clear genetic markers to target diseases at their source, aiming to address the significant unmet need where only 5% of genetic conditions have approved therapies.
View full company profileAbout GondolaBio
GondolaBio is a private, clinical-stage biotech company based in San Diego, leveraging a decentralized model to rapidly advance a diverse pipeline of small molecule therapies for genetic diseases. Its lead asset, PORT-77, is a Phase 2 oral ABCG2 inhibitor for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), with FDA Orphan Drug and Fast Track designations. The company's strategy centers on identifying clear genetic markers to target diseases at their source, aiming to address the significant unmet need where only 5% of genetic conditions have approved therapies.
View full company profileAbout GondolaBio
GondolaBio is a private, clinical-stage biotech company based in San Diego, leveraging a decentralized model to rapidly advance a diverse pipeline of small molecule therapies for genetic diseases. Its lead asset, PORT-77, is a Phase 2 oral ABCG2 inhibitor for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), with FDA Orphan Drug and Fast Track designations. The company's strategy centers on identifying clear genetic markers to target diseases at their source, aiming to address the significant unmet need where only 5% of genetic conditions have approved therapies.
View full company profileAbout GondolaBio
GondolaBio is a private, clinical-stage biotech company based in San Diego, leveraging a decentralized model to rapidly advance a diverse pipeline of small molecule therapies for genetic diseases. Its lead asset, PORT-77, is a Phase 2 oral ABCG2 inhibitor for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), with FDA Orphan Drug and Fast Track designations. The company's strategy centers on identifying clear genetic markers to target diseases at their source, aiming to address the significant unmet need where only 5% of genetic conditions have approved therapies.
View full company profileAbout GondolaBio
GondolaBio is a private, clinical-stage biotech company based in San Diego, leveraging a decentralized model to rapidly advance a diverse pipeline of small molecule therapies for genetic diseases. Its lead asset, PORT-77, is a Phase 2 oral ABCG2 inhibitor for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), with FDA Orphan Drug and Fast Track designations. The company's strategy centers on identifying clear genetic markers to target diseases at their source, aiming to address the significant unmet need where only 5% of genetic conditions have approved therapies.
View full company profileAbout GondolaBio
GondolaBio is a private, clinical-stage biotech company based in San Diego, leveraging a decentralized model to rapidly advance a diverse pipeline of small molecule therapies for genetic diseases. Its lead asset, PORT-77, is a Phase 2 oral ABCG2 inhibitor for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), with FDA Orphan Drug and Fast Track designations. The company's strategy centers on identifying clear genetic markers to target diseases at their source, aiming to address the significant unmet need where only 5% of genetic conditions have approved therapies.
View full company profileAbout GondolaBio
GondolaBio is a private, clinical-stage biotech company based in San Diego, leveraging a decentralized model to rapidly advance a diverse pipeline of small molecule therapies for genetic diseases. Its lead asset, PORT-77, is a Phase 2 oral ABCG2 inhibitor for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), with FDA Orphan Drug and Fast Track designations. The company's strategy centers on identifying clear genetic markers to target diseases at their source, aiming to address the significant unmet need where only 5% of genetic conditions have approved therapies.
View full company profileAbout GondolaBio
GondolaBio is a private, clinical-stage biotech company based in San Diego, leveraging a decentralized model to rapidly advance a diverse pipeline of small molecule therapies for genetic diseases. Its lead asset, PORT-77, is a Phase 2 oral ABCG2 inhibitor for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), with FDA Orphan Drug and Fast Track designations. The company's strategy centers on identifying clear genetic markers to target diseases at their source, aiming to address the significant unmet need where only 5% of genetic conditions have approved therapies.
View full company profileAbout GondolaBio
GondolaBio is a private, clinical-stage biotech company based in San Diego, leveraging a decentralized model to rapidly advance a diverse pipeline of small molecule therapies for genetic diseases. Its lead asset, PORT-77, is a Phase 2 oral ABCG2 inhibitor for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), with FDA Orphan Drug and Fast Track designations. The company's strategy centers on identifying clear genetic markers to target diseases at their source, aiming to address the significant unmet need where only 5% of genetic conditions have approved therapies.
View full company profileAbout GondolaBio
GondolaBio is a private, clinical-stage biotech company based in San Diego, leveraging a decentralized model to rapidly advance a diverse pipeline of small molecule therapies for genetic diseases. Its lead asset, PORT-77, is a Phase 2 oral ABCG2 inhibitor for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), with FDA Orphan Drug and Fast Track designations. The company's strategy centers on identifying clear genetic markers to target diseases at their source, aiming to address the significant unmet need where only 5% of genetic conditions have approved therapies.
View full company profileAbout Eyconis
Eyconis is a private, pre-revenue biotech founded in 2023 as a spinout from Ascendis Pharma, focused on revolutionizing ophthalmology therapeutics through long-acting drug delivery. Its core asset is an exclusive global license to the TransCon technology platform for ophthalmology, which it aims to apply to clinically validated and emerging medicines to address high-unmet-need retinal diseases. Led by a seasoned team with deep experience in ophthalmology drug development, commercialization, and venture capital, the company is positioned to develop potentially transformational therapies. Eyconis is in the pre-clinical/early development stage, building its pipeline to improve treatment paradigms for the leading causes of blindness.
View full company profileAbout Mithrl
Mithrl is a private, preclinical-stage biotech pioneering therapies that directly target mitochondrial function to treat metabolic and cardiovascular diseases. Its core technology platform is designed to deliver therapeutic agents specifically to mitochondria, aiming to correct the energetic and oxidative stress imbalances central to these prevalent conditions. The company is in the early stages of research and development, positioning itself in a high-need, high-value therapeutic area with significant unmet medical needs. As a young, private firm, its success will depend on advancing its pipeline through preclinical validation and securing further investment to reach clinical trials.
View full company profileAbout Pragma Bio
Pragma Bio is a private, preclinical-stage biotech based in San Diego, pioneering a novel drug discovery paradigm centered on the gut-immune axis. The company's proprietary PIKASO platform integrates bioinformatics, AI, synthetic biology, and pharmacology to mine, synthesize, and test gut-derived small molecules from biosynthetic gene clusters. With backing from top-tier investors and a seasoned leadership team, Pragma Bio is building a pipeline of new chemical entities aimed at addressing high-failure-rate areas in traditional drug development, though it faces significant scientific and competitive risks inherent to the emerging microbiome therapeutics field.
View full company profileAbout R1 Therapeutics
R1 Therapeutics is a private, preclinical-stage biotech company targeting kidney disease with novel small molecule therapeutics. Founded in 2020 and headquartered in San Diego, the company is in the early stages of research and development, positioning itself to tackle a large and growing market with high unmet need. As a pre-revenue entity, its success will depend on advancing its pipeline through preclinical validation and into clinical trials, likely supported by venture capital funding.
View full company profileAbout Mitem Pharma
Mitem Pharma is an early-stage biotech targeting a fundamental biological mechanism—mitochondrial dysfunction—which is implicated in a wide range of chronic and age-related diseases. While specific pipeline details are not publicly disclosed, its focus on metabolic and neurodegenerative conditions places it in large, high-need markets with significant unmet medical need. As a private, pre-revenue company likely in the pre-clinical or early clinical stage, its success hinges on validating its platform and advancing candidates through development, requiring sustained investor support and potential partnerships.
View full company profileAbout Tezcat Biosciences
Tezcat Biosciences is pioneering a macropinocytosis-targeting drug delivery platform to treat aggressive, Ras-mutated cancers and other diseases. The company's core innovation is a protein-drug conjugate that is selectively internalized by cancer cells exhibiting high levels of macropinocytosis, a nutrient-scavenging process, while sparing healthy cells. This approach aims to overcome the limitations of receptor-dependent targeting and offers flexibility in payload selection, including small molecules, PROTACs, and nucleic acids. As a private, pre-revenue company, Tezcat is advancing an internal pipeline and is open to strategic partnerships to expand the platform's applications.
View full company profileAbout Holobiome
Holobiome is a private, preclinical-stage biotech leveraging a proprietary platform to mine the human microbiome for novel live biotherapeutic products (LBPs). Founded by research scientists from Northeastern University, the company is advancing a pipeline focused on neurological and other systemic conditions through a rigorous discovery and development process. With a strong scientific team and advisory board, Holobiome has attracted strategic partners and investors to fund its translation of cutting-edge microbiome research into potential therapies. The company operates in the high-growth but technically challenging microbiome therapeutics sector.
View full company profileAbout Holobiome
Holobiome is a private, preclinical-stage biotech leveraging a proprietary platform to mine the human microbiome for novel live biotherapeutic products (LBPs). Founded by research scientists from Northeastern University, the company is advancing a pipeline focused on neurological and other systemic conditions through a rigorous discovery and development process. With a strong scientific team and advisory board, Holobiome has attracted strategic partners and investors to fund its translation of cutting-edge microbiome research into potential therapies. The company operates in the high-growth but technically challenging microbiome therapeutics sector.
View full company profileAbout Pykus Therapeutics
Pykus Therapeutics is an early-stage biotech targeting the high-need retinal disease market with a platform-based approach to drug delivery. While specific pipeline details are not publicly disclosed, the company's focus on biologics and innovative delivery methods positions it to tackle complex conditions like age-related macular degeneration (AMD) and diabetic retinopathy. As a private, pre-revenue entity, its success will hinge on platform validation, pipeline advancement, and securing strategic partnerships or further funding. The competitive but growing ophthalmic therapeutics landscape presents both significant opportunity and risk.
View full company profileAbout PathCision Medicine
PathCision Medicine is a private, preclinical-stage biotech founded in 2021 and headquartered in Cambridge, Massachusetts. The company has developed a proprietary discovery platform that integrates computational pathology, machine learning, and deep biological understanding to identify novel therapeutic pathways from tissue data. This platform fuels an internal pipeline of targeted therapies aimed at complex diseases, with an initial focus on transforming cancer treatment. The company is led by a team with expertise in translational medicine, pathology, and AI, positioning it at the intersection of digital pathology and drug discovery.
View full company profileAbout Novasenta
Novasenta is a private, preclinical-stage biotech leveraging a unique, human tissue-centric discovery platform to identify novel therapeutic targets in the tumor microenvironment. The company's approach combines deep computational biology with experimental validation to build a pipeline of antibody-based therapies for cancer and immune disorders. Led by a team with strong industry experience in immunology and drug development, Novasenta aims to generate next-generation, safer, and more effective immunotherapies. As a pre-revenue company, its success hinges on validating its platform and advancing its internal pipeline toward clinical proof-of-concept.
View full company profileAbout Healx
Healx is a private, clinical-stage biotech applying a proprietary AI platform to transform drug discovery for rare diseases, an area traditionally neglected due to high costs and low success rates. The company has built a pipeline with programs ranging from preclinical to Phase 2, primarily in rare neurology and oncology, and actively partners with patient groups and academic consortia. By using technology to identify novel drug-disease relationships and repurpose known compounds, Healx aims to run multiple programs in parallel, increasing the speed and probability of delivering treatments to patients.
View full company profileAbout Healx
Healx is a private, clinical-stage biotech applying a proprietary AI platform to transform drug discovery for rare diseases, an area traditionally neglected due to high costs and low success rates. The company has built a pipeline with programs ranging from preclinical to Phase 2, primarily in rare neurology and oncology, and actively partners with patient groups and academic consortia. By using technology to identify novel drug-disease relationships and repurpose known compounds, Healx aims to run multiple programs in parallel, increasing the speed and probability of delivering treatments to patients.
View full company profileAbout Bluefin Biomedicine
Bluefin Biomedicine is a private, preclinical-stage biotech leveraging a human tissue-anchored discovery platform to identify novel targets for antibody therapeutics in oncology and immunology. The company's core technology integrates proteomics, transcriptomics, and bioinformatics to map target expression within complex human disease microenvironments, aiming to generate differentiated candidates with clear disease relevance. Led by an experienced team with deep expertise in drug development from companies like Sanofi and Genzyme, Bluefin is advancing a portfolio of programs toward the clinic. The company operates as a pre-revenue entity, likely funded by venture capital, and is positioned to address significant unmet medical needs in its chosen therapeutic areas.
View full company profileAbout Ollin Biosciences
Ollin Biosciences is a private, preclinical-stage biotech targeting the high-need ophthalmology market with novel biologic therapies. The company appears to be in a foundational phase, building its technology platform and pipeline, likely operating with a small team in the biotech hub of Cambridge. As a newly established entity, it is pre-revenue and faces the standard risks of early-stage drug development, including scientific validation, funding, and competition, but is positioned in a growing therapeutic area with substantial commercial potential.
View full company profileAbout Boston Immune Technologies & Therapeutics
Boston Immune Technologies & Therapeutics is a private, preclinical-to-clinical stage biotech developing a novel class of TNFR superfamily antagonists called TrapMAbs. Its platform technology aims to trap receptor molecules in an inactive state, offering potential efficacy and safety advantages over conventional antibodies for targets previously considered challenging. The company's lead asset, BITR2101 targeting TNFR2 for oncology, is in Phase I, with a second program, BITT311 targeting CD40 for autoimmunity, in IND-enabling studies. BITT operates with a lean team and is positioned in the high-potential but competitive immuno-oncology and autoimmune therapy markets.
View full company profileAbout Boston Immune Technologies & Therapeutics
Boston Immune Technologies & Therapeutics is a private, preclinical-to-clinical stage biotech developing a novel class of TNFR superfamily antagonists called TrapMAbs. Its platform technology aims to trap receptor molecules in an inactive state, offering potential efficacy and safety advantages over conventional antibodies for targets previously considered challenging. The company's lead asset, BITR2101 targeting TNFR2 for oncology, is in Phase I, with a second program, BITT311 targeting CD40 for autoimmunity, in IND-enabling studies. BITT operates with a lean team and is positioned in the high-potential but competitive immuno-oncology and autoimmune therapy markets.
View full company profileAbout Boston Immune Technologies & Therapeutics
Boston Immune Technologies & Therapeutics is a private, preclinical-to-clinical stage biotech developing a novel class of TNFR superfamily antagonists called TrapMAbs. Its platform technology aims to trap receptor molecules in an inactive state, offering potential efficacy and safety advantages over conventional antibodies for targets previously considered challenging. The company's lead asset, BITR2101 targeting TNFR2 for oncology, is in Phase I, with a second program, BITT311 targeting CD40 for autoimmunity, in IND-enabling studies. BITT operates with a lean team and is positioned in the high-potential but competitive immuno-oncology and autoimmune therapy markets.
View full company profileAbout Uniogen
Uniogen is a private, preclinical-stage biotech based in Helsinki, Finland, focused on gene therapy for rare diseases. The company's core asset is its proprietary viral vector platform, which it aims to use to develop transformative, one-time treatments. As a pre-revenue entity, its success hinges on advancing its pipeline through clinical validation and securing strategic partnerships or funding. Operating in the high-potential but risky gene therapy space, Uniogen targets a niche within the rare disease market where it can demonstrate significant clinical and economic value.
View full company profileAbout Neumirna
Neumirna is a private, pre-revenue biotech pioneering a novel miRNA-targeting platform for neurological disorders. Its lead asset, NMT.001, is a clinical-stage candidate for drug-resistant epilepsy, with a preclinical program in Parkinson's disease. Backed by leading European life science investors and founded by experts in neuroscience and RNA biology, the company is positioned to advance a new class of therapeutics that address significant unmet medical needs in neurology.
View full company profileAbout Junevity
Junevity is an early-stage biotech developing a novel class of therapeutics using siRNA to reprogram cellular gene expression and reverse age-related diseases. The company's core innovation is the RESET platform, which integrates human omics data, genetic linkages, and machine learning to identify single transcription factor targets whose repression can reset cells to a healthier state. Founded by a team with expertise in siRNA, aging biology, and bioinformatics, and backed by venture capital, Junevity is advancing a preclinical pipeline with the long-term vision of creating a new era of disease reversal.
View full company profileAbout Junevity
Junevity is an early-stage biotech developing a novel class of therapeutics using siRNA to reprogram cellular gene expression and reverse age-related diseases. The company's core innovation is the RESET platform, which integrates human omics data, genetic linkages, and machine learning to identify single transcription factor targets whose repression can reset cells to a healthier state. Founded by a team with expertise in siRNA, aging biology, and bioinformatics, and backed by venture capital, Junevity is advancing a preclinical pipeline with the long-term vision of creating a new era of disease reversal.
View full company profileAbout a:head bio
a:head bio is a private, preclinical-stage biotech pioneering a human-centric approach to neuroscience drug discovery. The company leverages advanced stem cell technology to create proprietary human brain organoid models, which it uses to identify novel drug targets and validate therapeutic candidates with greater predictive validity than traditional animal models. Focused on complex psychiatric and neurological conditions, a:head aims to de-risk the notoriously challenging field of CNS drug development and build a pipeline of first-in-class therapies. As a platform company, it operates in a pre-revenue stage, relying on venture funding to advance its research and development programs.
View full company profileAbout 7 Primus Biosciences
7 Primus Biosciences is a private, preclinical-stage biotech targeting oncology with a multi-modality approach encompassing antibodies, biologics, and small molecules. Founded in 2021, the company is distinguished by its proprietary technology platforms and a team with strong translational expertise aimed at bridging discovery and clinical development. As a typical early-stage biotech, it is pre-revenue and focused on advancing its pipeline from research towards the clinic. The company's strategy combines scientific insight with proprietary technology to address significant unmet needs in cancer therapy.
View full company profileAbout Ranok Therapeutics
Ranok Therapeutics is a private, pre-clinical stage biotech pioneering a next-generation targeted protein degradation platform for oncology. The company is leveraging its proprietary CHAMP (CHaperone-Mediated Protein degradation) platform to develop small molecule degraders against historically undruggable targets. As a pre-revenue entity, its success hinges on advancing its pipeline into clinical trials and securing strategic partnerships or further investment. The competitive landscape is intense, with numerous companies exploring TPD, but Ranok's specific technological approach could carve out a niche in this high-potential field.
View full company profileAbout Balto Pharmaceuticals
Balto Pharmaceuticals is a private, preclinical-stage biotech targeting oncology with a specialized platform for inhibiting protein-protein interactions (PPIs) using small molecules. The company is tackling a historically difficult class of targets, aiming to develop first-in-class therapies for cancers with high unmet need. As a young company, it is in the early stages of research and development, positioning itself in a high-risk, high-reward segment of drug discovery. Its success will depend on validating its platform and advancing candidates into clinical trials.
View full company profileAbout AstralBio
AstralBio is an early-stage biotech based in San Diego, CA, with a secondary office in Boston, MA, focusing on AI-driven antibody discovery for cardiometabolic and immune-mediated diseases. Founded by experienced antibody developers from ValenzaBio and AlmataBio, the company is building on a proven computational platform to identify and advance novel biologics. As a private, pre-clinical entity, it is positioned to rapidly generate pipeline candidates by integrating machine learning with traditional biologics expertise. The company's success will hinge on validating its platform and translating its computational hits into effective clinical-stage therapeutics.
View full company profileAbout AstralBio
AstralBio is an early-stage biotech based in San Diego, CA, with a secondary office in Boston, MA, focusing on AI-driven antibody discovery for cardiometabolic and immune-mediated diseases. Founded by experienced antibody developers from ValenzaBio and AlmataBio, the company is building on a proven computational platform to identify and advance novel biologics. As a private, pre-clinical entity, it is positioned to rapidly generate pipeline candidates by integrating machine learning with traditional biologics expertise. The company's success will hinge on validating its platform and translating its computational hits into effective clinical-stage therapeutics.
View full company profileAbout Virothera
Virothera is an early-stage biotechnology company pioneering a gene therapy platform based on human homologues of viral immune modifiers to reprogram immune cells. The technology targets the core problem of immune dysregulation in persistent conditions like chronic infections, cancer, and autoimmune diseases, with the goal of achieving durable control. Founded in 2020 and based in London, the private company is in the pre-clinical, pre-revenue stage, building its pipeline and team, including recent appointments to its clinical leadership. Its approach represents a potentially disruptive modality in the immunotherapy space, aiming for broader therapeutic reach compared to biologics.
View full company profileAbout Zecardio Therapeutics
Zecardio Therapeutics is a private, pre-clinical stage biotech developing small molecule drugs for cardiovascular diseases, a leading cause of global mortality with substantial unmet need. The company appears to be in the early, asset-discovery phase, supported by grants and partnerships, and is building a pipeline based on a proprietary discovery platform. As a young company, it is likely pre-revenue and focused on validating its technology and advancing lead candidates toward clinical trials to attract further investment and collaboration.
View full company profileAbout Ribocure Pharmaceuticals
Ribocure Pharmaceuticals is a Stockholm-based, clinical-stage biotech company founded in 2016, specializing in the development of innovative oligonucleotide therapeutics. As a subsidiary of the publicly traded Ribo Life Science, it benefits from an integrated R&D platform and operates a unique 'bench-to-bed' facility that includes its own Phase 2 clinical trial clinic. The company is actively seeking partnerships and talent to grow its pipeline in rare cardiovascular, renal, and oncology diseases.
View full company profileAbout Oxion Biologics
Oxion Biologics is a private, pre-revenue biotech advancing OX118, an Fc-optimized monoclonal antibody targeting OX40L, through clinical development for a broad range of autoimmune conditions. With a first-in-human trial scheduled for Q2 2025 and a granted EU Clinical Trial Application, the company is rapidly progressing towards clinical validation. Its approach aims for true disease modification by selectively depleting pro-inflammatory cells and promoting regulatory T-cell function, targeting a multi-billion dollar market with significant unmet needs.
View full company profileAbout Nuclidium
Nuclidium is a Swiss-German biotech focused on disrupting the radiopharmaceutical space with its proprietary copper-based theranostic platform. By utilizing Copper-61 and Copper-67 isotopes paired with the same targeting molecule, the company aims to overcome key limitations in current radiotheranostics, such as mismatched diagnostics/therapeutics and complex supply chains, to enable more accurate and accessible cancer treatment. Founded in 2019, the company is advancing a pipeline targeting major solid tumors and has established a secured production process and distribution network. It operates as a private, clinical-stage entity seeking to set a new standard in precision oncology.
View full company profileAbout Hillhurst Biopharmaceuticals
Hillhurst Biopharmaceuticals is pioneering the oral delivery of therapeutic gases through its GLASS technology platform, moving beyond traditional inhalation methods. Founded in 2018, the company has advanced into clinical stages, with a Phase 2a trial cleared for its Parkinson's disease candidate and a pipeline targeting sickle cell disease and acute pain. It is a privately held, pre-revenue company backed by non-dilutive grant funding from foundations and government sources, led by a team with deep experience in hematology, commercialization, and drug development.
View full company profileAbout Hillhurst Biopharmaceuticals
Hillhurst Biopharmaceuticals is pioneering the oral delivery of therapeutic gases through its GLASS technology platform, moving beyond traditional inhalation methods. Founded in 2018, the company has advanced into clinical stages, with a Phase 2a trial cleared for its Parkinson's disease candidate and a pipeline targeting sickle cell disease and acute pain. It is a privately held, pre-revenue company backed by non-dilutive grant funding from foundations and government sources, led by a team with deep experience in hematology, commercialization, and drug development.
View full company profileAbout Hillhurst Biopharmaceuticals
Hillhurst Biopharmaceuticals is pioneering the oral delivery of therapeutic gases through its GLASS technology platform, moving beyond traditional inhalation methods. Founded in 2018, the company has advanced into clinical stages, with a Phase 2a trial cleared for its Parkinson's disease candidate and a pipeline targeting sickle cell disease and acute pain. It is a privately held, pre-revenue company backed by non-dilutive grant funding from foundations and government sources, led by a team with deep experience in hematology, commercialization, and drug development.
View full company profileAbout Pinion Immunotherapeutics
Pinion Immunotherapeutics is a privately held, preclinical-stage biotech leveraging an integrated AI and mRNA/LNP platform to create immunotherapies for viral diseases and cancers. Founded in 2021 and operating as a lean, virtual company, it focuses on capital-efficient development of treatments for large markets lacking curative options. Its leadership team combines deep expertise in vaccine development, immunology, and clinical oncology, aiming to achieve functional cures through dual immune activation.
View full company profileAbout Genix Therapeutics
Genix Therapeutics is a private, pre-clinical stage biotech firm developing gene therapies for rare diseases. Its core differentiator is a proprietary delivery platform designed to improve the efficacy and safety of genetic medicines. Operating in the high-potential but challenging gene therapy space, the company is likely in a capital-intensive R&D phase, seeking partnerships and investment to advance its pipeline. As a young company with limited public information, its progress is contingent on platform validation and successful translation into clinical candidates.
View full company profileAbout SeaBeLife Biotech
SeaBeLife Biotech is pioneering a novel therapeutic approach by developing dual inhibitors of regulated necrosis pathways (necroptosis and ferroptosis), a mechanism implicated in a wide range of severe organ diseases. Its most advanced program, SBL03, is a topical ophthalmic gel for geographic atrophy (GA) in dry AMD, which has shown promising preclinical efficacy and secured an Orphan Drug Designation from the EMA. The company is privately held, pre-revenue, and operates with a capital-efficient model through strategic partnerships, such as its joint development agreement with Unither Pharmaceuticals, to advance its pipeline from preclinical to clinical stages.
View full company profileAbout NXI Therapeutics
NXI Therapeutics is a private, preclinical-stage biotech developing novel immunotherapies with an emphasis on improved specificity and safety. Operating from the European life sciences hub of Basel, the company is building a platform to target complex immune disorders and oncology. As a pre-revenue entity, it is likely reliant on venture capital and partnerships to advance its research. Its success hinges on translating its platform into differentiated clinical candidates that can address significant unmet needs in autoimmune diseases and cancer.
View full company profileAbout NXI Therapeutics
NXI Therapeutics is a private, preclinical-stage biotech developing novel immunotherapies with an emphasis on improved specificity and safety. Operating from the European life sciences hub of Basel, the company is building a platform to target complex immune disorders and oncology. As a pre-revenue entity, it is likely reliant on venture capital and partnerships to advance its research. Its success hinges on translating its platform into differentiated clinical candidates that can address significant unmet needs in autoimmune diseases and cancer.
View full company profileAbout Opprtna Therapeutics
Opprtna Therapeutics is an early-stage biotech leveraging precision DNA aptamers to target proteins involved in neurodegenerative diseases and oncology. The company's platform focuses on a historically underexplored angle of aptamer development, targeting proteins that naturally bind to RNA/DNA, which could enable high specificity and safety. With a leadership team combining scientific and operational expertise, Opprtna is in the pre-clinical stage, aiming to address large, costly markets with significant unmet need. The company is privately held and pre-revenue, building its foundational technology and pipeline.
View full company profileAbout Opprtna Therapeutics
Opprtna Therapeutics is an early-stage biotech leveraging precision DNA aptamers to target proteins involved in neurodegenerative diseases and oncology. The company's platform focuses on a historically underexplored angle of aptamer development, targeting proteins that naturally bind to RNA/DNA, which could enable high specificity and safety. With a leadership team combining scientific and operational expertise, Opprtna is in the pre-clinical stage, aiming to address large, costly markets with significant unmet need. The company is privately held and pre-revenue, building its foundational technology and pipeline.
View full company profileAbout Lyterian Therapeutics
Lyterian Therapeutics is a private, preclinical-stage biotech founded in 2020 and headquartered in South San Francisco, California. The company is pioneering a novel drug discovery platform that leverages endogenous protein homeostatic mechanisms—the body's natural systems for maintaining protein balance—to target genetically-validated drivers of disease, with an initial focus on neuroscience and rare disorders. Backed by a founding team of renowned academic scientists from Stanford, UC Berkeley, and Genentech, Lyterian is building its research team to translate fundamental biological insights into first-in-class medicines. The company is currently in a pre-revenue, asset discovery and development phase.
View full company profileAbout Rise Therapeutics
Rise Therapeutics is a private, clinical-stage biotech developing first-in-class oral immunotherapies for autoimmune diseases and cancer. Its core innovation is a proprietary oral drug delivery platform that enables the targeted delivery of biologic therapies, a significant advancement over traditional injectable biologics. The company has built integrated capabilities from discovery through in-house GMP manufacturing, accelerating multiple programs into clinical trials. With four active clinical programs and recent funding, Rise is positioned to validate its platform and address major unmet needs in immunology.
View full company profileAbout Rise Therapeutics
Rise Therapeutics is a private, clinical-stage biotech developing first-in-class oral immunotherapies for autoimmune diseases and cancer. Its core innovation is a proprietary oral drug delivery platform that enables the targeted delivery of biologic therapies, a significant advancement over traditional injectable biologics. The company has built integrated capabilities from discovery through in-house GMP manufacturing, accelerating multiple programs into clinical trials. With four active clinical programs and recent funding, Rise is positioned to validate its platform and address major unmet needs in immunology.
View full company profileAbout Signadori Bio
Signadori Bio is a French biotechnology company founded in 2021, leveraging a proprietary cell therapy platform developed at the prestigious Institut Gustave Roussy to target cancer. The company is in the preclinical stage, building its pipeline of novel immunotherapies with the goal of addressing significant unmet needs in oncology. As a private, preclinical entity, it is focused on research and development, positioning itself in the competitive but high-potential cell and gene therapy sector. Its success will hinge on validating its platform, advancing candidates into clinical trials, and securing strategic partnerships or funding.
View full company profileAbout Solid Therapeutics
SOLID Therapeutics is a private, preclinical-stage biotech focused on a novel approach to treating solid tumors through small molecules that engage both the tumor and the immune system. Originating from academic research at the University of Copenhagen, the company is in the early stages of developing its pipeline. Its core strategy is to overcome the limitations of current immuno-oncology therapies by simultaneously attacking cancer cells and modulating the tumor microenvironment.
View full company profileAbout Exogene
Exogene is a private, preclinical-stage biotechnology company applying a proprietary generative AI platform to the discovery of novel T-cell receptor (TCR) therapies for solid tumors. The company has assembled a deeply technical team with expertise in machine learning, computational biology, immunology, and drug development to build its discovery engine and advance its internal pipeline. Operating as a platform company, Exogene aims to generate its own therapeutic assets while potentially leveraging its technology through partnerships to address the significant challenge of identifying safe, effective TCRs for cancer immunotherapy.
View full company profileAbout Y-Trap
Y-Trap is a private, preclinical-stage biotech pioneering a novel approach to cancer immunotherapy. The company's platform engineers multispecific antibodies that target and reprogram tumor-promoting immune cells within the tumor microenvironment, aiming to overcome a key limitation of existing immunotherapies. By directly addressing the immunosuppressive signals that allow tumors to evade immune attack, Y-Trap's technology has the potential to treat patients unresponsive to current treatments. The company is positioned in the high-growth oncology biologics sector but faces significant technical and competitive risks inherent to drug development.
View full company profileAbout Onco3R Therapeutics
Onco3R Therapeutics is a Belgian clinical-stage biotech developing precision medicines for oncology and immunology with a portfolio of five disclosed programs. The company leverages a seasoned team with over 150 years of combined R&D experience and a dual-modality approach, utilizing both small molecules and antibodies to target clinically validated pathways. Its strategy focuses on designing best-in-class therapies by learning from the liabilities of first-generation drugs to improve safety and efficacy.
View full company profileAbout CaSRevolution
CaSRevolution is a private, preclinical-stage biotech pioneering a novel CRISPR-based gene therapy platform targeting neurodegenerative diseases. Founded in Milan in 2019, the company is developing potentially curative approaches that aim to modify disease progression, a significant unmet need in neurology. While still in early research and development, its focus on cutting-edge genetic tools positions it in a high-potential but competitive segment of the biopharma industry. The company appears to be in a foundational, pre-revenue stage, building its technology and pipeline.
View full company profileAbout Algotec Research and Development
Algotec R&D is a private, preclinical-stage biotech leveraging proprietary drug delivery platforms to enhance small molecule therapeutics. Operating from London, the company appears to be pre-revenue, focusing on R&D to build a pipeline of reformulated and novel compounds. Its strategy targets significant unmet medical needs by improving drug bioavailability and targeting, positioning it in the competitive but high-growth drug delivery sector.
View full company profileAbout Granza Bio
Granza Bio is an early-stage biotech leveraging a foundational discovery from the University of Oxford to develop a new class of immunotherapies. Its core technology platform is built around 'attack particles,' which are natural, cell-free weapons of the immune system that can be engineered for precise targeting. The company is in the pre-clinical research phase, working to translate this discovery into programmable therapeutics for oncology and inflammatory conditions. As a private, pre-revenue entity, its near-term focus is on validating its platform and advancing its first programs toward the clinic.
View full company profileAbout Macomics
Macomics is a private, pre-clinical biotech company pioneering macrophage-targeted therapies for oncology, inflammation, fibrosis, and neurology. Its core asset is the ENIGMAC™ platform, which uses human genetic and clinical evidence to identify and validate novel macrophage targets. Led by an experienced team with strong venture backing, the company aims to translate its unique understanding of macrophage biology into breakthrough treatments for diseases where current options are limited.
View full company profileAbout Macomics
Macomics is a private, pre-clinical biotech company pioneering macrophage-targeted therapies for oncology, inflammation, fibrosis, and neurology. Its core asset is the ENIGMAC™ platform, which uses human genetic and clinical evidence to identify and validate novel macrophage targets. Led by an experienced team with strong venture backing, the company aims to translate its unique understanding of macrophage biology into breakthrough treatments for diseases where current options are limited.
View full company profileAbout Macomics
Macomics is a private, pre-clinical biotech company pioneering macrophage-targeted therapies for oncology, inflammation, fibrosis, and neurology. Its core asset is the ENIGMAC™ platform, which uses human genetic and clinical evidence to identify and validate novel macrophage targets. Led by an experienced team with strong venture backing, the company aims to translate its unique understanding of macrophage biology into breakthrough treatments for diseases where current options are limited.
View full company profileAbout Human Life CORD
Japanese biotech developing umbilical cord-derived mesenchymal stromal cell therapies for inflammatory and respiratory diseases.
View full company profileAbout MapLight Therapeutics
MapLight Therapeutics is on a mission to redefine the standard of care for brain disorders by moving beyond broad neuromodulation to precise, circuit-specific therapeutics. The company has built a robust pipeline of six drug candidates, with its most advanced programs in Phase 2 trials for schizophrenia and Alzheimer's disease psychosis. Its strategy is anchored in a proprietary discovery platform co-founded by optogenetics pioneer Karl Deisseroth, which integrates cutting-edge technologies to translate deep neuroscience insights into targeted medicines for conditions like autism, Parkinson's, and schizophrenia.
View full company profileAbout MapLight Therapeutics
MapLight Therapeutics is on a mission to redefine the standard of care for brain disorders by moving beyond broad neuromodulation to precise, circuit-specific therapeutics. The company has built a robust pipeline of six drug candidates, with its most advanced programs in Phase 2 trials for schizophrenia and Alzheimer's disease psychosis. Its strategy is anchored in a proprietary discovery platform co-founded by optogenetics pioneer Karl Deisseroth, which integrates cutting-edge technologies to translate deep neuroscience insights into targeted medicines for conditions like autism, Parkinson's, and schizophrenia.
View full company profileAbout MapLight Therapeutics
MapLight Therapeutics is on a mission to redefine the standard of care for brain disorders by moving beyond broad neuromodulation to precise, circuit-specific therapeutics. The company has built a robust pipeline of six drug candidates, with its most advanced programs in Phase 2 trials for schizophrenia and Alzheimer's disease psychosis. Its strategy is anchored in a proprietary discovery platform co-founded by optogenetics pioneer Karl Deisseroth, which integrates cutting-edge technologies to translate deep neuroscience insights into targeted medicines for conditions like autism, Parkinson's, and schizophrenia.
View full company profileAbout Design Therapeutics
Design Therapeutics is a clinical-stage biotech focused on developing disease-modifying therapies for severe genetic disorders driven by nucleotide repeat expansions. Its core innovation is the GeneTAC™ platform, which creates bifunctional small molecules that can precisely upregulate or downregulate disease-causing genes without altering DNA. The company's strategy targets monogenic diseases with high unmet need, advancing a pipeline led by DT-216P2 for Friedreich's ataxia, now in Phase 1/2 trials, alongside preclinical programs in Fuchs endothelial corneal dystrophy and myotonic dystrophy type 1.
View full company profileAbout Design Therapeutics
Design Therapeutics is a clinical-stage biotech focused on developing disease-modifying therapies for severe genetic disorders driven by nucleotide repeat expansions. Its core innovation is the GeneTAC™ platform, which creates bifunctional small molecules that can precisely upregulate or downregulate disease-causing genes without altering DNA. The company's strategy targets monogenic diseases with high unmet need, advancing a pipeline led by DT-216P2 for Friedreich's ataxia, now in Phase 1/2 trials, alongside preclinical programs in Fuchs endothelial corneal dystrophy and myotonic dystrophy type 1.
View full company profileTherapeutic Areas
Other Neurodegenerative Diseases Drugs
| Drug | Company | Phase |
|---|---|---|
| PBI-01207 | Phoenix Biotechnology | Pre-clinical |
| Therapeutic Target Discovery Platform | ValtedSeq | Discovery |
| Undisclosed Neurology Program(s) | Base4 | Preclinical |
| Platform Application for Neurodegenerative Diseases | ALTUCELL | Pre-clinical/Research |
| OGG1 Activator Program | Luciole Pharmaceuticals | Pre-clinical |
| Lysosome Biology Program | Talisman Therapeutics | Preclinical |
| Microglial Health Program | Talisman Therapeutics | Preclinical |
| HSF1 Activator Program | Sisu Pharma | Pre-clinical |
| Platform-derived candidates | VRG Therapeutics | Pre-clinical |
| Neurodegenerative Biomarkers R&D | Firalis | Development |
| Neurodegeneration Pipeline | Origenis | Preclinical |
| Enhanced Brain Delivery (EBD™) Platform | Acumen Pharmaceuticals | Preclinical |