Takeda Ventures

Harness Therapeutics is a private biotechnology company focused on developing SINEUPs, a novel class of therapeutic RNAs that upregulate protein expression with precise control and specificity. The company’s platform uses mRNA-targeted oligonucleotides to modulate post-transcriptional protein synthesis, enabling disease-modifying protein upregulation for conditions that have resisted traditional approaches. Its work centers on central nervous system and ophthalmologic disorders, with potential applications across multiple therapy areas by expanding the druggable proteome beyond what conventional small molecules, biologics, or gene therapies can address.

Degron Therapeutics is a drug discovery company focused on developing a new class of therapeutics centered around targeted protein degradation. The company specializes in molecular glue degrader drugs, which are small molecules designed to selectively target and degrade specific proteins associated with various diseases. Degron Therapeutics aims to lead the field of small molecule molecular glue degrader medicines by leveraging its expertise to identify and validate challenging disease targets that traditional small molecule inhibitors cannot effectively address. The company is committed to addressing unmet medical needs across a broad range of therapeutic areas by developing innovative protein degradation drugs, utilizing its advanced molecular glue platform technology to enhance treatment efficacy.

Founded in 2020, Be Biopharma specializes in developing innovative therapeutics using engineered B-cells to treat diseases such as cancer, autoimmune disorders, and infectious diseases. Its approach aims to overcome limitations of current cell and gene therapies.

Founded in 2020, Be Biopharma specializes in developing innovative therapeutics using engineered B-cells to treat diseases such as cancer, autoimmune disorders, and infectious diseases. Its approach aims to overcome limitations of current cell and gene therapies.

Ascentage Pharma is a clinical-stage biotechnology company focused on developing novel small-molecule therapies for cancers, hepatitis B virus (HBV), and age-related diseases. Its pipeline comprises seven drug candidates targeting protein-protein interactions and tyrosine kinases.

Nabla Bio is a biotechnology company that uses artificial intelligence and lab technologies to rationally design novel proteins for drug development. They aim to create medicines and improve nutrition by engineering proteins with enhanced qualities.

EvolveImmune Therapeutics is a biotechnology company focused on developing innovative immunotherapies aimed at addressing unmet needs in oncology and autoimmune diseases. The company has created an advanced immunobiological platform that combines sustained immune activation with targeted tumor cell therapy in a single agent. Through its unique approach, EvolveImmune's biotherapeutics utilize engineered extracellular vesicles to directly target tumor cells and enhance cytokine secretion at the tumor site, thereby improving tumor destruction while minimizing toxicity. The company is actively building a pipeline of novel therapeutic candidates that leverage its proprietary in vivo target discovery platform, enabling more effective treatment options for patients suffering from various diseases.

Crescendo Biologics develops innovative antibody therapeutics using its proprietary platforms. The company focuses on generating high-affinity, human VH antibody fragments for oncology and other indications. Its technology is based on a transgenic mouse platform that enables the creation of fully human VHH domain building blocks.

Larkspur Biosciences specializes in developing precision immunotherapies for specific patient groups with molecularly defined cancers. Its focus is on targeting the interaction between tumors and the immune system to stimulate robust, long-lasting responses.

OncoResponse is an immuno‑oncology biotechnology company that uses a proprietary human antibody platform to discover novel targets and develop fully human monoclonal antibodies for cancer treatment. By interrogating the adaptive immune system of patients who respond exceptionally well to checkpoint inhibition, the company identifies genuine human antibodies against high‑value targets linked with immunosuppressive myeloid biology. Its pipeline aims to alleviate tumor‑microenvironment immunosuppression and enhance immune activation, converting cold tumors into hot ones and providing new therapeutic options for patients.

Cerevance develops novel therapeutics for neurological disorders such as Alzheimer's disease, Parkinson's disease, and other chronic neurodegenerative conditions. It uses a proprietary platform to profile specific brain cell types in human tissue samples, enabling the identification of promising targets for new treatments.

Ensoma is a biotechnology company focused on advancing genomic medicine through its innovative in vivo approach, utilizing proprietary Engenious vectors. These vectors are engineered to deliver a wide array of gene modification technologies directly to blood and immune cells, including T cells, B cells, and myeloid cells, without the necessity for stem cell collection or prior myeloablative conditioning, which can pose risks to patients. This unique method allows for the administration of therapies via a single injection, enhancing accessibility and convenience in various healthcare settings, even where resources may be limited. Ensoma aims to expand the curative potential of genomic medicine, making it more accessible to patients in need.

Nimbus Lakshmi operates as a wholly-owned subsidiary of Nimbus Therapeutics and offers tyrosine kinase 2 (TYK2) inhibitor NDI-034858. NDI-034858, an orally administered, selective allosteric inhibitor of TYK2, is currently undergoing assessment for its potential in treating various autoimmune diseases, following promising Phase 2b outcomes in psoriasis.

Zelluna Immunotherapy is a Norwegian biopharmaceutical company focused on developing T-cell receptor based cancer immunotherapies. Founded in 2016 and based in Oslo, it pursues a pipeline of tumor-specific TCRs, many derived from patients who showed long-term survival after peptide-based cancer vaccine trials at the Norwegian Radium Hospital/Oslo University Hospital. The company aims to enable immune cells to recognize a broad range of cancers with safety and durability. It operates in collaboration with Oslo Cancer Cluster and is located at the Oslo Cancer Cluster Innovation Park near major hospital facilities and GMP-certified cellular therapy capabilities.

Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies to treat serious and life-threatening genetic diseases. Code Bio leverages its novel multivalent synthetic DNA delivery platform, 3DNA, which has been engineered to overcome many of the challenges inherent with viral-based gene therapies such as immunogenicity, size and delivery limitations, re-dosability, and manufacturing complexity. The company is advancing an internal pipeline focused on select rare disease programs as well as establishing partnerships to take forward programs in both rare and prevalent diseases.

Founded in 2020, Be Biopharma specializes in developing innovative therapeutics using engineered B-cells to treat diseases such as cancer, autoimmune disorders, and infectious diseases. Its approach aims to overcome limitations of current cell and gene therapies.

Koneksa Health Inc. is a healthcare data analytics company that specializes in designing and developing software solutions for pharmaceutical and biotech firms. The company’s flagship product, Koneksa Compare, facilitates the collection, monitoring, and analysis of patient-generated data to support clinical studies and regulatory claims. By integrating emerging technologies such as bio-sensors, activity trackers, and mobile-based questionnaires, Koneksa enhances the accuracy and breadth of data that researchers can analyze, thereby minimizing subjective biases in clinical endpoints. Additionally, the platform provides a dashboard that offers electronic clinical outcome assessments and reminders for study participants. Established in 2013 and headquartered in New York, Koneksa also maintains a presence in London, United Kingdom, and is recognized for its contributions to improving the efficiency of drug development through innovative data analysis.

Adaptate Biotherapeutics is a London-based biotherapeutics company founded in 2019 by Adrian Hayday. The company specializes in developing antibody-based therapies aimed at modulating gamma delta T cells in situ. By leveraging innovative technologies, Adaptate focuses on creating immunotherapies that target cancer and autoimmune diseases. Its approach has the potential to significantly influence treatment options for patients with these conditions, providing access to effective and novel therapeutic modalities.

Ambys Medicines, Inc. is a biotechnology company based in Redwood City, California, focused on discovering and developing innovative therapies for severe liver diseases. Founded in 2016, Ambys aims to transform the lives of individuals suffering from liver conditions by creating advanced gene, drug, and cell therapies. The company is dedicated to restoring liver function, halting the progression of liver disease, and addressing the serious complications associated with liver failure. Through its regenerative medical therapies, Ambys seeks to provide relief to patients with chronic liver diseases, ultimately improving their quality of life.

Integra Therapeutics is a biotechnology company focused on advancing gene editing technologies to address various diseases. It develops innovative gene writing tools that enhance the capabilities of gene therapy, aiming to meet the specific needs of patients. By integrating the precision of CRISPR systems with the efficiency of viral integrases and transposases, Integra Therapeutics seeks to overcome existing limitations in gene editing. The company's approach enables scientists to introduce DNA into genomes using sequence-specific DNA binding proteins, facilitating the development of more effective gene therapies.

Cardurion Pharmaceuticals, LLC, established in 2017 and headquartered in Boston, Massachusetts, is a biotechnology company dedicated to developing innovative therapies for heart failure and other cardiovascular diseases. The company is advancing clinical programs that target PDE9 and CaMKII inhibition, utilizing its expertise in cardiovascular signaling pathways to address significant unmet patient needs.

Founded in 2020, Egle Therapeutics is a biotechnology company based in Paris, France. It specializes in developing immunotherapies targeting immune suppressor regulatory T-cells (Tregs) for oncology and autoimmune diseases.

Turnstone Biologics is a clinical-stage biotechnology company focused on developing viral immunotherapies to improve cancer survival. Its lead candidate, RIVAL-01, combines three immunomodulators to stimulate immune activity and reprogram the tumor microenvironment.

Ribon Therapeutics, Inc. is a biotechnology company founded in 2015 and headquartered in Lexington, Massachusetts. The company focuses on developing novel therapeutics that target monoPARP proteins, which are critical regulators of cancer cell survival mechanisms. By leveraging insights from its scientific founders, Ribon is establishing a proprietary drug discovery platform aimed at investigating the molecular actions and biological functions of a specific subset of the PARP protein family. This platform enables Ribon to create a pipeline of innovative small molecule inhibitors designed to disrupt the fundamental survival capabilities of cancer cells under stress. In addition to cancer treatment, the company's research may have implications for addressing other diseases. Ribon is supported by notable life science investors, which enhances its capacity to advance its therapeutic developments.

Harness Therapeutics is a private biotechnology company focused on developing SINEUPs, a novel class of therapeutic RNAs that upregulate protein expression with precise control and specificity. The company’s platform uses mRNA-targeted oligonucleotides to modulate post-transcriptional protein synthesis, enabling disease-modifying protein upregulation for conditions that have resisted traditional approaches. Its work centers on central nervous system and ophthalmologic disorders, with potential applications across multiple therapy areas by expanding the druggable proteome beyond what conventional small molecules, biologics, or gene therapies can address.

BridGene Biosciences is a biotechnology company that employs chemoproteomic technology to discover and develop small molecules. It uses covalent small molecules for proteome-wide screening in live cells, enabling the targeting of traditionally undruggable proteins.

Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies to treat serious and life-threatening genetic diseases. Code Bio leverages its novel multivalent synthetic DNA delivery platform, 3DNA, which has been engineered to overcome many of the challenges inherent with viral-based gene therapies such as immunogenicity, size and delivery limitations, re-dosability, and manufacturing complexity. The company is advancing an internal pipeline focused on select rare disease programs as well as establishing partnerships to take forward programs in both rare and prevalent diseases.

Adaptate Biotherapeutics is a London-based biotherapeutics company founded in 2019 by Adrian Hayday. The company specializes in developing antibody-based therapies aimed at modulating gamma delta T cells in situ. By leveraging innovative technologies, Adaptate focuses on creating immunotherapies that target cancer and autoimmune diseases. Its approach has the potential to significantly influence treatment options for patients with these conditions, providing access to effective and novel therapeutic modalities.

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

StrideBio, Inc. is a gene therapy company based in Durham, North Carolina, focused on developing innovative genetic medicines for rare diseases. Founded in 2015, the company utilizes a proprietary structure-inspired adeno-associated virus (AAV) vector engineering platform to create unique and differentiated vectors. These vectors are designed to improve upon naturally occurring AAV serotypes, addressing limitations of first-generation gene therapies. StrideBio's technologies facilitate gene addition, silencing, and editing, providing potential curative options for patients with severe medical conditions. By combining structural insights with accelerated evolution, StrideBio aims to produce novel AAV capsids capable of evading neutralizing antibodies, ultimately enhancing treatment outcomes for those affected by rare diseases.

Maverick Therapeutics, Inc. is a biotechnology company based in Brisbane, California, that specializes in developing T-cell engagement therapeutics aimed at treating solid tumor cancers. Founded in 2016, the company focuses on creating innovative immunotherapies that enhance safety and efficacy for patients. Its proprietary platform, COBRA™, is recognized as a leading bispecific T-cell engaging technology, engineered to target solid tumors with high specificity and potency. This novel platform addresses toxicity challenges associated with cancer treatments by remaining inactive upon administration and becoming fully active within the tumor microenvironment, thereby optimizing T-cell redirection therapy. Through its advancements, Maverick Therapeutics aims to improve patient outcomes and facilitate more effective cancer care.

Presage Biosciences, Inc. is an oncology company based in Seattle, Washington, that focuses on enhancing drug development and clinical trials through innovative technology. Founded in 2008, the company has developed the CIVO arrayed microinjection platform, which allows for the simultaneous assessment of multiple drugs or drug combinations directly within a patient's tumor. This method provides insights into drug efficacy, resistance, and potential synergies in the tumor's native microenvironment, thereby overcoming limitations associated with systemic drug administration. By using this clinical surrogate approach, Presage enables drug developers to identify ineffective compounds early in the process, ultimately streamlining the development of effective cancer treatments. The technology also facilitates the identification of drug targets and effective combinations, allowing for more accurate measurement of therapeutic responses.

Xilio Therapeutics is a clinical-stage biotechnology company focused on developing innovative immunotherapies to enhance the immune system's ability to combat cancer. The company specializes in tumor-selective immunotherapies, utilizing its proprietary technology to maximize the effectiveness of immuno-oncology treatments while minimizing side effects outside the tumor. Among its key products is XTX201, which aims to induce immune activity directly within tumors, demonstrating improved antitumor effects. Founded in 2015 and based in Waltham, Massachusetts, Xilio Therapeutics was previously known as Akrevia Therapeutics until its rebranding in March 2020. The company is dedicated to advancing a pipeline of novel immuno-oncology molecules, including tumor-activated cytokines and antibodies, with the goal of significantly improving patient outcomes in cancer treatment.

Ensoma is a biotechnology company focused on advancing genomic medicine through its innovative in vivo approach, utilizing proprietary Engenious vectors. These vectors are engineered to deliver a wide array of gene modification technologies directly to blood and immune cells, including T cells, B cells, and myeloid cells, without the necessity for stem cell collection or prior myeloablative conditioning, which can pose risks to patients. This unique method allows for the administration of therapies via a single injection, enhancing accessibility and convenience in various healthcare settings, even where resources may be limited. Ensoma aims to expand the curative potential of genomic medicine, making it more accessible to patients in need.

Catamaran Bio, Inc. is a biotechnology company focused on developing allogeneic CAR-NK immune cell therapies for the treatment of solid tumors and other cancers. Founded in 2019 and based in Cambridge, Massachusetts, the company utilizes its Tailwind platform to engineer, expand, and process natural killer (NK) cells into off-the-shelf CAR-NK cell therapy products. By leveraging genetic engineering, Catamaran Bio aims to address significant unmet medical needs in cancer treatment, providing transformative therapies for patients.

Bridge Medicines LLC is a biotechnology company specializing in drug discovery and development. Founded in 2016 and based in New York, it aims to translate academic research into effective therapeutics for various human diseases. The company focuses on developing innovative treatments, including inhibitors for ENL-YEATS to target acute leukemias and small molecule inhibitors for activated factor XII to address hereditary angioedema and inflammatory disorders. Bridge Medicines collaborates with notable institutions such as Memorial Sloan Kettering Cancer Center and The Rockefeller University, providing comprehensive support from preclinical studies to clinical trials. By streamlining the path from concept to drug candidate, Bridge Medicines seeks to efficiently develop new therapies in oncology, neuropsychiatry, and other rare diseases, ultimately enhancing treatment options for physicians and patients.

Founded in 2020, Be Biopharma specializes in developing innovative therapeutics using engineered B-cells to treat diseases such as cancer, autoimmune disorders, and infectious diseases. Its approach aims to overcome limitations of current cell and gene therapies.

XFOREST Therapeutics specializes in developing RNA-targeted small molecule therapeutics for diseases previously considered undruggable. The company employs a structure-based discovery platform that combines high-throughput RNA motif screening and splicing modulation analysis, enabling the identification of selective binders to accelerate drug development in areas such as oncology, neuroscience, immunology, and rare diseases.

Palleon Pharmaceuticals is a biotechnology company developing drugs that target glycan-mediated immune regulation to treat cancer. Its platform integrates insights from glycoscience and human immunology, enabling it to create novel therapies targeting multiple immune cell types.

ImmPACT Bio USA Inc. is a cell therapy company focused on developing engineered T-cells for the treatment of solid tumors. The company specializes in creating chimeric antigen receptor (CAR) T-cell therapies that target specific gene loss features in tumor cells, allowing for the selective destruction of cancerous tissues while preserving healthy ones. This technology aims to enhance tumor specificity, enabling the CAR-T cells to induce cytotoxicity only when engaging with tumor cells, thereby minimizing the safety risks commonly associated with existing CAR-T therapies. ImmPACT Bio's innovative approach seeks to improve treatment outcomes for cancer patients by offering a more precise and safer therapeutic option.

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

Enterome develops innovative cancer treatments focusing on generating powerful, long-lasting immune responses using its proprietary OncoMimic technology. The company aims to overcome immune tolerance against cancer cells, with applications across various solid tumors. Its most advanced programs are in Phase 2 clinical trials for hard-to-treat tumors such as glioblastoma, adrenal malignancies, and colorectal cancer.

Cerevance develops novel therapeutics for neurological disorders such as Alzheimer's disease, Parkinson's disease, and other chronic neurodegenerative conditions. It uses a proprietary platform to profile specific brain cell types in human tissue samples, enabling the identification of promising targets for new treatments.

Redpin Therapeutics, Inc. is a preclinical stage gene therapy company founded in 2017 and based in New York, New York. The company specializes in developing a proprietary chemogenetics platform for targeted cell therapies aimed at addressing intractable diseases of the nervous system. Redpin's innovative approach utilizes ion channels as neuromodulation tools, allowing for selective activation or inhibition of disease-causing neurons while preserving normal functioning cells. By controlling these neuronal circuits, Redpin enables physicians to precisely target dysfunctional neural activity associated with specific conditions. The company's technology integrates principles from synthetic biology, gene therapy, and traditional pharmacotherapy to advance its mission of addressing complex neurological disorders.

Xilio Therapeutics is a clinical-stage biotechnology company focused on developing innovative immunotherapies to enhance the immune system's ability to combat cancer. The company specializes in tumor-selective immunotherapies, utilizing its proprietary technology to maximize the effectiveness of immuno-oncology treatments while minimizing side effects outside the tumor. Among its key products is XTX201, which aims to induce immune activity directly within tumors, demonstrating improved antitumor effects. Founded in 2015 and based in Waltham, Massachusetts, Xilio Therapeutics was previously known as Akrevia Therapeutics until its rebranding in March 2020. The company is dedicated to advancing a pipeline of novel immuno-oncology molecules, including tumor-activated cytokines and antibodies, with the goal of significantly improving patient outcomes in cancer treatment.

PVP Biologics, Inc. is a biotechnology company based in San Diego, California, focused on developing innovative therapeutic products for celiac disease. Established in 2015, the company specializes in creating an oral enzyme designed to break down immuno-reactive components of gluten in the stomach. This approach aims to alleviate the painful symptoms and intestinal damage caused by accidental gluten ingestion in individuals with celiac disease. By advancing this product candidate, PVP Biologics seeks to significantly reduce the burden of living with this condition and improve the quality of life for those affected. As of early 2020, PVP Biologics operates as a subsidiary of Takeda Pharmaceutical Company Limited.

Emendo Biotherapeutics is a biotechnology company focused on advancing genome editing technologies to develop genetic medicines. Established by a team of experts with backgrounds in protein engineering and DNA repair from prestigious institutions, Emendo aims to tackle the challenges associated with gene therapy. By innovating and transforming existing tools, the company seeks to address diseases and disorders that are presently deemed untreatable. Emendo's approach fuses diverse scientific disciplines with creative problem-solving, enabling the development of advanced gene editing solutions that enhance the efficacy of gene therapy and expand its potential applications. Through this commitment to pushing the boundaries of science, Emendo Biotherapeutics strives to fulfill the promise of gene editing for improved patient outcomes.

EvolveImmune Therapeutics is a biotechnology company focused on developing innovative immunotherapies aimed at addressing unmet needs in oncology and autoimmune diseases. The company has created an advanced immunobiological platform that combines sustained immune activation with targeted tumor cell therapy in a single agent. Through its unique approach, EvolveImmune's biotherapeutics utilize engineered extracellular vesicles to directly target tumor cells and enhance cytokine secretion at the tumor site, thereby improving tumor destruction while minimizing toxicity. The company is actively building a pipeline of novel therapeutic candidates that leverage its proprietary in vivo target discovery platform, enabling more effective treatment options for patients suffering from various diseases.

Harness Therapeutics is a private biotechnology company focused on developing SINEUPs, a novel class of therapeutic RNAs that upregulate protein expression with precise control and specificity. The company’s platform uses mRNA-targeted oligonucleotides to modulate post-transcriptional protein synthesis, enabling disease-modifying protein upregulation for conditions that have resisted traditional approaches. Its work centers on central nervous system and ophthalmologic disorders, with potential applications across multiple therapy areas by expanding the druggable proteome beyond what conventional small molecules, biologics, or gene therapies can address.

Turnstone Biologics is a clinical-stage biotechnology company focused on developing viral immunotherapies to improve cancer survival. Its lead candidate, RIVAL-01, combines three immunomodulators to stimulate immune activity and reprogram the tumor microenvironment.

Avidity Biosciences is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

ProteKt Therapeutics is a drug development company focused on creating potent and selective oral inhibitors of the kinase PKR, targeting neurodegenerative and neuroinflammatory diseases. The company utilizes unique computational methods to develop a series of novel and selective molecules, which are validated through clinically relevant assays. This approach aims to address issues such as memory consolidation impairment and enhance long-term memory in patients. In 2019, ProteKt Therapeutics graduated from the FutuRx accelerator and subsequently raised $4 million in a pre-A funding round to support its innovative research and development efforts.

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

Phathom Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Buffalo Grove, Illinois, that specializes in developing and commercializing innovative treatments for gastrointestinal diseases. Incorporated in 2018, the company has rights in the United States, Europe, and Canada for vonoprazan, a potassium-competitive acid blocker (P-CAB) designed to inhibit acid secretion in the stomach. Currently, vonoprazan is undergoing Phase III clinical trials for the treatment of erosive gastroesophageal reflux disease and is also being evaluated in combination with antibiotics for addressing Helicobacter pylori infection. Phathom Pharmaceuticals aims to meet the unmet medical needs of patients suffering from acid-related disorders by leveraging the expertise of its team, which comprises seasoned professionals from the gastrointestinal and pharmaceutical sectors.

HOOKIPA Pharma is a clinical-stage biopharmaceutical company developing immune-therapeutics targeting infectious diseases and cancers using its proprietary arenavirus platform. Its lead product candidates include HB-101 for cytomegalovirus prevention in transplant patients, and HB-201/HB-202 for human papillomavirus-positive cancers.

BiomX develops bacteriophage-based therapies targeting bacteria linked to skin conditions and chronic diseases such as inflammatory bowel disease, liver disorders, and cancer. It collaborates with renowned institutions for research and development, aiming to create innovative microbiome therapeutics.

Ribon Therapeutics, Inc. is a biotechnology company founded in 2015 and headquartered in Lexington, Massachusetts. The company focuses on developing novel therapeutics that target monoPARP proteins, which are critical regulators of cancer cell survival mechanisms. By leveraging insights from its scientific founders, Ribon is establishing a proprietary drug discovery platform aimed at investigating the molecular actions and biological functions of a specific subset of the PARP protein family. This platform enables Ribon to create a pipeline of innovative small molecule inhibitors designed to disrupt the fundamental survival capabilities of cancer cells under stress. In addition to cancer treatment, the company's research may have implications for addressing other diseases. Ribon is supported by notable life science investors, which enhances its capacity to advance its therapeutic developments.

Avidity Biosciences is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

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

Bsense Bio Therapeutics develops small-molecule therapeutics for sensory hyperexcitability-related disorders. It pursues a novel approach that targets multiple hyperexcitability mechanisms with a single compound, focusing on two cation channels: a ligand-gated cation channel and a voltage-gated potassium channel, to improve efficacy and safety.

Ramino Bio is a private biotech company that develops an oral drug to treat metabolic diseases. Ramino has received funding totaling about $3.3 million since its beginning from the FutuRx biotech incubator, which is supported by the Israeli Innovation Authority, Takeda, J&J, OrbiMed, and RMPG. By speeding up their disintegration, our exclusive technology lowers the extra BCAA that is floating around.

Ambys Medicines, Inc. is a biotechnology company based in Redwood City, California, focused on discovering and developing innovative therapies for severe liver diseases. Founded in 2016, Ambys aims to transform the lives of individuals suffering from liver conditions by creating advanced gene, drug, and cell therapies. The company is dedicated to restoring liver function, halting the progression of liver disease, and addressing the serious complications associated with liver failure. Through its regenerative medical therapies, Ambys seeks to provide relief to patients with chronic liver diseases, ultimately improving their quality of life.

Ambys Medicines, Inc. is a biotechnology company based in Redwood City, California, focused on discovering and developing innovative therapies for severe liver diseases. Founded in 2016, Ambys aims to transform the lives of individuals suffering from liver conditions by creating advanced gene, drug, and cell therapies. The company is dedicated to restoring liver function, halting the progression of liver disease, and addressing the serious complications associated with liver failure. Through its regenerative medical therapies, Ambys seeks to provide relief to patients with chronic liver diseases, ultimately improving their quality of life.

GEXVal Inc. is a drug discovery company based in Fujisawa, Japan, founded in 2018. The company focuses on the research and development of innovative treatments for rare and incurable diseases, particularly in the fields of psychiatry, neurology, and pulmonary arterial hypertension. GEXVal is dedicated to addressing the needs of patients and families affected by these conditions by providing cost-effective treatment options that aim to enhance health and improve quality of life. Through its specialized research efforts, GEXVal seeks to develop compounds that target intractable diseases, enabling physicians to offer better therapeutic solutions to their patients.

Presage Biosciences, Inc. is an oncology company based in Seattle, Washington, that focuses on enhancing drug development and clinical trials through innovative technology. Founded in 2008, the company has developed the CIVO arrayed microinjection platform, which allows for the simultaneous assessment of multiple drugs or drug combinations directly within a patient's tumor. This method provides insights into drug efficacy, resistance, and potential synergies in the tumor's native microenvironment, thereby overcoming limitations associated with systemic drug administration. By using this clinical surrogate approach, Presage enables drug developers to identify ineffective compounds early in the process, ultimately streamlining the development of effective cancer treatments. The technology also facilitates the identification of drug targets and effective combinations, allowing for more accurate measurement of therapeutic responses.

ARTham Therapeutics Inc is a biopharmaceutical company based in Naka-Ku, Japan, founded in 2018. The company focuses on delivering innovative biological medicines aimed at treating debilitating diseases. Its portfolio includes programs such as art-648, art-ep, art-pde, and art-001, which are designed to address specific medical needs in areas including inflammatory skin diseases, vascular malformations, and immuno-oncology endometriosis. Operating under a virtual research and development model, ARTham Therapeutics collaborates with academic and business partners to drive innovation and create disease-modifying therapies that enhance patient health and quality of life.

Reborna Biosciences specializes in researching and developing treatments for genetic diseases, focusing on areas with unmet medical needs. The company's core technology enables low-molecular compounds to target ribonucleic acid (RNA), leading to the creation of new disease-modifying drugs for patients suffering from rare genetic diseases such as spinal muscular atrophy.

StrideBio, Inc. is a gene therapy company based in Durham, North Carolina, focused on developing innovative genetic medicines for rare diseases. Founded in 2015, the company utilizes a proprietary structure-inspired adeno-associated virus (AAV) vector engineering platform to create unique and differentiated vectors. These vectors are designed to improve upon naturally occurring AAV serotypes, addressing limitations of first-generation gene therapies. StrideBio's technologies facilitate gene addition, silencing, and editing, providing potential curative options for patients with severe medical conditions. By combining structural insights with accelerated evolution, StrideBio aims to produce novel AAV capsids capable of evading neutralizing antibodies, ultimately enhancing treatment outcomes for those affected by rare diseases.

Quince Therapeutics is a preclinical-stage biopharmaceutical company focused on developing precision therapeutics for debilitating and rare diseases. Its core technology is a bone-targeting drug platform designed to deliver small molecules, peptides, or large molecules directly to the site of bone fractures and diseases, promoting rapid healing with fewer off-target safety concerns compared to non-targeted therapeutics.

Crescendo Biologics develops innovative antibody therapeutics using its proprietary platforms. The company focuses on generating high-affinity, human VH antibody fragments for oncology and other indications. Its technology is based on a transgenic mouse platform that enables the creation of fully human VHH domain building blocks.

Shire is a global biopharmaceutical company focused on developing and marketing therapies for rare diseases. It maintains a broad portfolio across hematology, immunology, neuroscience, ophthalmology, internal medicine and endocrine disorders, and hereditary angioedema. The company operates a worldwide sales and marketing network with direct marketing in the United States, Canada, the United Kingdom, Ireland, France, Germany, Italy and Spain. It emphasizes patient-focused care and aims to deliver innovative therapies to patients with rare diseases worldwide.

Fimecs is a biotechnology company that specializes in drug discovery focused on protein degradation technology. The firm aims to develop first-in-class therapeutics targeting the degradation of proteins associated with cancers and other challenging diseases. Utilizing a proprietary platform based on ubiquitin E3 ligase binders, Fimecs designs small molecules that can induce the degradation of specific proteins, many of which are considered undruggable. This innovative approach targets proteins linked to various diseases, expanding the potential for effective treatment options beyond the traditionally druggable targets identified in small molecule drug discovery. Through its emphasis on targeted protein degradation, Fimecs is positioned to contribute significantly to the advancement of anti-cancer therapies and the treatment of other hard-to-treat conditions.

Cerevance develops novel therapeutics for neurological disorders such as Alzheimer's disease, Parkinson's disease, and other chronic neurodegenerative conditions. It uses a proprietary platform to profile specific brain cell types in human tissue samples, enabling the identification of promising targets for new treatments.

Wave Life Sciences is a clinical-stage biotechnology company focused on RNA medicines, designing, optimizing, and producing stereopure oligonucleotides through its PRISM platform. The company aims to address genetically defined diseases by reducing harmful protein expression or correcting dysfunctional proteins, employing modalities such as RNA editing, antisense silencing, and RNA interference. Its programs span neurological and neuromuscular conditions, reflecting an emphasis on central nervous system targets. Wave collaborates with Pfizer and Takeda on research, development, and commercialization of stereopure oligonucleotide therapeutics and antisense therapies. Founded in 2012 and based in Singapore, the company seeks to unlock the broader potential of RNA medicines to improve patient outcomes.

TiGenix NV is a Belgium-based biomedical company specializing in innovative treatments for damaged and osteoarthritic joints. Founded as a spin-off from the Catholic University of Leuven and the University of Ghent, TiGenix leverages regenerative medicine to create durable therapies validated through controlled clinical studies. The company focuses on developing a portfolio of products aimed at addressing specific musculoskeletal issues, with a primary emphasis on cartilage damage, which significantly impairs patient mobility and function. TiGenix also explores the anti-inflammatory properties of stem cells to create novel therapies for serious medical conditions, particularly in areas where there is a high unmet medical need.

SEEDSUPPLY is a research and development company based in Fujisawa, Kanagawa, Japan, that specializes in creating medical and agricultural chemical products. Established in May 2017 as a spin-off from Takeda Pharmaceutical Company Limited, SEEDSUPPLY operates a drug discovery platform focused on the development of small molecule drugs. The company utilizes binder selection technology to address drug targets that are typically difficult to analyze with conventional high-throughput screening methods. This innovative approach enables medical professionals to explore new possibilities in the discovery of drug candidate compounds, contributing to advancements in medical treatments.

HOOKIPA Pharma is a clinical-stage biopharmaceutical company developing immune-therapeutics targeting infectious diseases and cancers using its proprietary arenavirus platform. Its lead product candidates include HB-101 for cytomegalovirus prevention in transplant patients, and HB-201/HB-202 for human papillomavirus-positive cancers.

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

Chordia Therapeutics Inc. is a clinical-stage biotechnology company based in Fujisawa, Japan, founded in 2017. The company specializes in the research and development of innovative oncology drugs, focusing on delivering first-in-class therapies for cancers that currently have high unmet medical needs. Chordia Therapeutics is committed to advancing new therapeutic options to patients as swiftly as possible, aiming to address significant gaps in cancer treatment.

Palleon Pharmaceuticals is a biotechnology company developing drugs that target glycan-mediated immune regulation to treat cancer. Its platform integrates insights from glycoscience and human immunology, enabling it to create novel therapies targeting multiple immune cell types.

Founded in 2015 and based in Minato-ku, Japan, Noile-Immune Biotech specializes in developing CAR-T cell therapies for cancer immunotherapies. Its pipeline includes candidates like NIB102, NIB103, and NIB104.

Molecular Templates, Inc. is a clinical-stage biopharmaceutical company based in Austin, Texas, dedicated to the discovery and development of biologic therapeutics aimed at treating cancer and other serious diseases. The company utilizes its proprietary engineered toxin body (ETB) platform to create therapies with unique mechanisms of action that differ from traditional antibody-drug conjugates. Its lead candidate, MT-3724, is currently undergoing Phase II clinical trials for relapsed and refractory diffuse large B-cell lymphoma and non-Hodgkin’s lymphoma. Additionally, Molecular Templates is developing other candidates, including MT-4019, which targets CD38, and has a partnership with Takeda Pharmaceutical Company to create CD38-targeted therapies for multiple myeloma. The company also collaborates with Vertex Pharmaceuticals to enhance the hematopoietic stem cell transplant process through novel conditioning regimens.

BiomX develops bacteriophage-based therapies targeting bacteria linked to skin conditions and chronic diseases such as inflammatory bowel disease, liver disorders, and cancer. It collaborates with renowned institutions for research and development, aiming to create innovative microbiome therapeutics.

GammaDelta Therapeutics focuses on developing innovative immunotherapies utilizing gamma delta (γδ) T cells to treat cancer and autoimmune diseases. The company aims to harness the unique properties of tissue resident γδ T cells to enhance the effectiveness of immunotherapy. By leveraging these specialized immune cells, GammaDelta Therapeutics seeks to provide patients with improved treatment options that can significantly impact their health outcomes in the face of serious diseases.

Cerevance develops novel therapeutics for neurological disorders such as Alzheimer's disease, Parkinson's disease, and other chronic neurodegenerative conditions. It uses a proprietary platform to profile specific brain cell types in human tissue samples, enabling the identification of promising targets for new treatments.

Quince Therapeutics is a preclinical-stage biopharmaceutical company focused on developing precision therapeutics for debilitating and rare diseases. Its core technology is a bone-targeting drug platform designed to deliver small molecules, peptides, or large molecules directly to the site of bone fractures and diseases, promoting rapid healing with fewer off-target safety concerns compared to non-targeted therapeutics.

Scohia Pharma is a Fujisawa, Japan-based biopharmaceutical company focused on researching, designing, and developing therapies for metabolic, cardiovascular, and renal diseases. Its work targets lifestyle-related conditions such as obesity, diabetes, dyslipidemia, and hypertension, with an emphasis on delivering treatments that improve patient outcomes through research and development across metabolic and renal disorders.

Ariad Pharmaceuticals, Inc. is a biotechnology company based in Cambridge, Massachusetts, specializing in the discovery, development, and commercialization of innovative cancer therapies. The company’s flagship product, Iclusig (ponatinib), is a tyrosine kinase inhibitor approved for the treatment of adult patients with chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia in multiple regions, including the United States and Europe. Additionally, Ariad is developing Brigatinib, an investigational treatment targeting anaplastic lymphoma kinase for non-small cell lung cancer, as well as AP32788, aimed at treating non-small cell lung cancer and other solid tumors. The company markets Iclusig through specialty pharmacies in the U.S. Established in 1991, Ariad Pharmaceuticals operates as a subsidiary of Takeda Pharmaceuticals U.S.A., Inc., and remains committed to advancing therapies that transform the lives of cancer patients.

PVP Biologics, Inc. is a biotechnology company based in San Diego, California, focused on developing innovative therapeutic products for celiac disease. Established in 2015, the company specializes in creating an oral enzyme designed to break down immuno-reactive components of gluten in the stomach. This approach aims to alleviate the painful symptoms and intestinal damage caused by accidental gluten ingestion in individuals with celiac disease. By advancing this product candidate, PVP Biologics seeks to significantly reduce the burden of living with this condition and improve the quality of life for those affected. As of early 2020, PVP Biologics operates as a subsidiary of Takeda Pharmaceutical Company Limited.

Maverick Therapeutics, Inc. is a biotechnology company based in Brisbane, California, that specializes in developing T-cell engagement therapeutics aimed at treating solid tumor cancers. Founded in 2016, the company focuses on creating innovative immunotherapies that enhance safety and efficacy for patients. Its proprietary platform, COBRA™, is recognized as a leading bispecific T-cell engaging technology, engineered to target solid tumors with high specificity and potency. This novel platform addresses toxicity challenges associated with cancer treatments by remaining inactive upon administration and becoming fully active within the tumor microenvironment, thereby optimizing T-cell redirection therapy. Through its advancements, Maverick Therapeutics aims to improve patient outcomes and facilitate more effective cancer care.

Cerevance develops novel therapeutics for neurological disorders such as Alzheimer's disease, Parkinson's disease, and other chronic neurodegenerative conditions. It uses a proprietary platform to profile specific brain cell types in human tissue samples, enabling the identification of promising targets for new treatments.

HOOKIPA Pharma is a clinical-stage biopharmaceutical company developing immune-therapeutics targeting infectious diseases and cancers using its proprietary arenavirus platform. Its lead product candidates include HB-101 for cytomegalovirus prevention in transplant patients, and HB-201/HB-202 for human papillomavirus-positive cancers.

Bridge Medicines LLC is a biotechnology company specializing in drug discovery and development. Founded in 2016 and based in New York, it aims to translate academic research into effective therapeutics for various human diseases. The company focuses on developing innovative treatments, including inhibitors for ENL-YEATS to target acute leukemias and small molecule inhibitors for activated factor XII to address hereditary angioedema and inflammatory disorders. Bridge Medicines collaborates with notable institutions such as Memorial Sloan Kettering Cancer Center and The Rockefeller University, providing comprehensive support from preclinical studies to clinical trials. By streamlining the path from concept to drug candidate, Bridge Medicines seeks to efficiently develop new therapies in oncology, neuropsychiatry, and other rare diseases, ultimately enhancing treatment options for physicians and patients.

Emendo Biotherapeutics is a biotechnology company focused on advancing genome editing technologies to develop genetic medicines. Established by a team of experts with backgrounds in protein engineering and DNA repair from prestigious institutions, Emendo aims to tackle the challenges associated with gene therapy. By innovating and transforming existing tools, the company seeks to address diseases and disorders that are presently deemed untreatable. Emendo's approach fuses diverse scientific disciplines with creative problem-solving, enabling the development of advanced gene editing solutions that enhance the efficacy of gene therapy and expand its potential applications. Through this commitment to pushing the boundaries of science, Emendo Biotherapeutics strives to fulfill the promise of gene editing for improved patient outcomes.

Bioniz Therapeutics, Inc. is a biopharmaceutical company based in Irvine, California, established in 2009. It specializes in discovering and developing innovative peptide therapeutics aimed at treating immune diseases and cancer. The company's pipeline includes BNZ-1, a selective inhibitor targeting interleukin cytokines IL2, IL9, and IL15, which are significant contributors to conditions such as HTLV-1 Associated Myelopathy and T-cell leukemias. Additionally, Bioniz is developing IL-15 and IL-21 inhibitors, as well as BNZ-3, an early-stage asset focused on inhibiting IL-4, IL-9, and IL-21 cytokines. Bioniz Therapeutics is committed to advancing its lead technologies through proof of concept and preclinical studies, with the goal of partnering for clinical trials and eventual commercialization.

Mersana Therapeutics is a clinical-stage biopharmaceutical company that develops antibody-drug conjugates (ADCs) for cancer patients with unmet medical needs. It leverages proprietary ADC platforms, including Dolaflexin, Dolasynthen, and Immunosynthen, to create a diverse pipeline of product candidates designed to improve therapeutic benefit and address oncology indications underserved by traditional ADCs. The lead candidate XMT-1536 targets NaPi2b and is in Phase I trials for ovarian cancer, non-small cell lung cancer, and other indications. The company also advances additional programs such as Emi-Le (XMT-1660), XMT-2056, XMT-2068, and XMT-2175, reflecting its focus on next-generation ADCs. Mersana is headquartered in Cambridge, Massachusetts, and was founded in 2005.

Ultragenyx Pharmaceutical develops therapeutics for rare diseases, focusing on serious genetic conditions with limited treatment options. The company leverages its team's experience to create life-changing medicines by advancing underdeveloped scientific research in these areas.

Quince Therapeutics is a preclinical-stage biopharmaceutical company focused on developing precision therapeutics for debilitating and rare diseases. Its core technology is a bone-targeting drug platform designed to deliver small molecules, peptides, or large molecules directly to the site of bone fractures and diseases, promoting rapid healing with fewer off-target safety concerns compared to non-targeted therapeutics.

Prosetta Biosciences, Inc. is a biotechnology company based in San Francisco, California, founded in 2002. The company specializes in developing novel small-molecule antiviral therapeutics through its innovative drug discovery platform, which utilizes cell-free protein synthesizing systems. This platform enables the identification of small molecule drugs that may be overlooked by traditional pharmaceutical screening methods. Prosetta Biosciences is engaged in collaborative efforts with both commercial and non-profit partners to advance compounds targeting a variety of human diseases, including oncology, neurodegenerative disorders, and infectious diseases. The company's focus on modulating the assembly of multi-protein complexes within cells positions it as a significant player in the biopharmaceutical sector.

Univercells is a company specializing in high-density, low-cost biomanufacturing solutions aimed at enhancing the production of biologics, including antibodies, proteins, and vaccines. The company leverages innovative engineering and proprietary technologies, such as single-use systems and continuous process intensification, to significantly reduce capital investments by over 75% and the cost of goods by up to 90%. Univercells offers a range of services, including scale-X hydro, scale-X carbo, and NevoLine, which support both research and development and commercial-scale production of biologics. Additionally, the company provides integrated services like equipment confirmation, capacity and facility design, and bioprocess excellence, facilitating the scalability of cell and gene therapy manufacturers from clinical trials to commercial manufacturing. Through its commitment to making biologics more accessible and affordable, Univercells addresses critical global health challenges.