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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

ArmaGen, Inc. is a clinical-stage biotechnology company based in San Diego, California, specializing in the development of therapies for severe neurological diseases. The company focuses on lysosomal storage disorders and neurodegenerative diseases, including Hunter syndrome, Hurler syndrome, Sanfilippo A syndrome, and metachromatic leukodystrophy. ArmaGen's innovative product candidates, such as AGT-182 and AGT-181, are designed to penetrate the blood-brain barrier, allowing for more effective treatment of neurological complications associated with these conditions. Founded in 2001, ArmaGen aims to improve the quality of life for patients suffering from these challenging disorders through its targeted therapeutic approaches.

Juventas Therapeutics is a privately-held clinical-stage biotechnology company developing regenerative therapies. Founded in 2007, it focuses on treating life-threatening diseases using non-viral gene therapies that activate natural processes to repair the body.

Xenon Pharmaceuticals is a biopharmaceutical company dedicated to developing novel medicines for neurological disorders. It focuses on rare genetic defects as drug targets, leveraging its proprietary discovery platform called Extreme Genetics. The company's pipeline includes XEN1101 and XEN496 for epilepsy treatment.

Receptor BioLogix is a biotechnology company based in Palo Alto, California, focused on developing receptor-based biological therapeutics aimed at treating cancer, inflammatory, and autoimmune diseases. The company specializes in creating Hermodulins, which are pan-HER ligand traps designed to inhibit the compensatory feedback mechanisms that can reduce the effectiveness and longevity of various cancer treatments. Additionally, Receptor BioLogix utilizes its Intron Fusion Protein platform to discover novel splice variants of human proteins, which hold therapeutic potential. Through these innovative approaches, the company aims to enhance treatment options and improve outcomes for patients with challenging medical conditions.