F-Prime Capital

Galatea Bio is a biotechnology company based in the United States, founded by Carlos Bustamante, Nicholas Katsanis, and Alexander Ioannidis. The company focuses on improving diagnostic testing and accelerating drug discovery, particularly in underserved populations that have been historically underrepresented in genetics research. Galatea Bio operates a biobank that serves as a biosample-to-bioinformatics platform, utilizing advanced algorithms and innovative analyses to enhance genomic discovery. By identifying patients at risk for severe multi-inflammatory syndromes and infections, Galatea Bio aims to develop new tests, treatments, and cures that can significantly improve healthcare outcomes for diverse populations.

Genomics plc is focused on transforming healthcare through advanced genomic analysis. The company employs sophisticated methodologies to analyze large integrated datasets of genomic and phenotypic information, aiming to enhance understanding of human biology. By leveraging genetic inference technology, Genomics plc seeks to identify new drug targets for serious diseases and assess individual disease risk. Their analytical platform enables the exploration of genomic sequence data, providing valuable insights that support pharmaceutical companies in reducing risks associated with drug development. This approach not only aims to improve healthcare outcomes but also sets a standard for how organizations and patients can benefit from genomic data.

Nvelop Therapeutics is engaged in the development of advanced genetic medicines aimed at addressing severe genetic diseases. The company specializes in creating programmable delivery systems that enhance in vivo gene editing and facilitate the delivery of therapeutic cargo. By integrating epigenetic editing technologies with innovative delivery methods, Nvelop Therapeutics targets unmet medical needs, particularly in therapeutically relevant cells and tissues. Their focus on novel gene editing delivery technologies enables medical professionals to effectively deliver a range of therapeutic payloads, thereby improving treatment options for patients suffering from these challenging conditions.

Ensoma is a biotechnology company focused on advancing genomic medicine through its innovative in vivo approach using Engenious vectors. These vectors are engineered to facilitate the delivery of gene modification technologies directly to blood and immune cells, including T cells, B cells, and myeloid cells, without the need for stem cell collection or harmful pre-treatment procedures like chemotherapy. This unique capability allows Ensoma to administer therapies through a single injection, making it possible to deliver effective treatments in outpatient settings and areas with limited access to advanced healthcare systems. By streamlining the process of gene therapy, Ensoma aims to enhance the accessibility and curative potential of genomic medicine for patients with various diseases.

AviadoBio is focused on developing innovative gene therapies aimed at transforming the lives of individuals affected by neurodegenerative disorders, specifically frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The company leverages advanced research from King's College London and the UK Dementia Research Institute to create its therapeutic platform. By integrating a comprehensive understanding of brain biology with proprietary gene therapy techniques, AviadoBio seeks to address the challenges of effectively delivering treatments to targeted areas in the brain. This approach aims to enhance the therapeutic potential of gene therapies, offering hope for halting or potentially reversing the progression of these debilitating diseases.

Galatea Bio is a biotechnology company based in the United States, founded by Carlos Bustamante, Nicholas Katsanis, and Alexander Ioannidis. The company focuses on improving diagnostic testing and accelerating drug discovery, particularly in underserved populations that have been historically underrepresented in genetics research. Galatea Bio operates a biobank that serves as a biosample-to-bioinformatics platform, utilizing advanced algorithms and innovative analyses to enhance genomic discovery. By identifying patients at risk for severe multi-inflammatory syndromes and infections, Galatea Bio aims to develop new tests, treatments, and cures that can significantly improve healthcare outcomes for diverse populations.

Embark offers dog DNA testing that ends preventable diseases in dogs. It was founded by top experts in genomics, computational biology, and software design to sell an industry-leading dog DNA test. They use a clinical-grade CLIA-certified SNP chip to provide the most accurate and comprehensive results (200,000+ genetic markers) available on the market. Their data contributes to proprietary genetics research that will help dog owners optimize nutrition, exercise, allergies, and prevent a variety of heritable conditions. Their technological advantage, intellectual capital, and research-focus enable them to push canine health toward our shared vision of preventative veterinary care.

Prime Medicine is a biotechnology company focused on developing genetic therapies using its proprietary Prime Editing technology. This next-generation gene editing method allows for precise "search and replace" modifications in the genome, aiming to restore normal genetic function and address the underlying causes of various diseases. By targeting a broad spectrum of conditions with significant unmet medical needs, Prime Medicine seeks to revolutionize the field of genetic medicine through effective and efficient therapeutic solutions.

Genomics plc is focused on transforming healthcare through advanced genomic analysis. The company employs sophisticated methodologies to analyze large integrated datasets of genomic and phenotypic information, aiming to enhance understanding of human biology. By leveraging genetic inference technology, Genomics plc seeks to identify new drug targets for serious diseases and assess individual disease risk. Their analytical platform enables the exploration of genomic sequence data, providing valuable insights that support pharmaceutical companies in reducing risks associated with drug development. This approach not only aims to improve healthcare outcomes but also sets a standard for how organizations and patients can benefit from genomic data.

Ensoma is a biotechnology company focused on advancing genomic medicine through its innovative in vivo approach using Engenious vectors. These vectors are engineered to facilitate the delivery of gene modification technologies directly to blood and immune cells, including T cells, B cells, and myeloid cells, without the need for stem cell collection or harmful pre-treatment procedures like chemotherapy. This unique capability allows Ensoma to administer therapies through a single injection, making it possible to deliver effective treatments in outpatient settings and areas with limited access to advanced healthcare systems. By streamlining the process of gene therapy, Ensoma aims to enhance the accessibility and curative potential of genomic medicine for patients with various diseases.

Sana Biotechnology is a biotechnology company based in Seattle, Washington, with additional offices in South San Francisco, California, and Cambridge, Massachusetts. Founded in 2018, the company specializes in developing engineered cells as medicines to treat a variety of diseases. Sana Biotechnology focuses on reprogramming cells in the body and replacing damaged cells and tissues, leveraging recent scientific advancements to create new therapeutic options. The company's research spans multiple therapeutic areas with significant unmet needs, including oncology, diabetes, B-cell-mediated autoimmune disorders, and central nervous system conditions. Its pipeline includes various product candidates aimed at addressing these challenges through innovative cell engineering programs.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

NFlection Therapeutics, Inc. is a biotechnology company dedicated to the discovery and development of targeted therapies for rare disorders. Based in Wayne, Pennsylvania, the company primarily addresses conditions such as neurofibromatosis type 1, immunosuppressant-mediated squamous cell carcinoma, and congenital birthmarks like keratinocytic epidermal nevi and nevus sebaceous. NFlection specializes in treatments that target the Ras/Raf/MEK/ERK signaling pathway, which is often aberrantly activated in these disorders. One of its notable developments includes a soft mitogen-activated protein kinase (MEK) formulated as a cosmetically elegant gel for topical application, allowing for localized treatment while minimizing systemic side effects typically associated with orally administered MEK inhibitors. Founded in 2014, NFlection Therapeutics aims to improve patient outcomes through innovative therapeutic solutions.

Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.

AviadoBio is focused on developing innovative gene therapies aimed at transforming the lives of individuals affected by neurodegenerative disorders, specifically frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The company leverages advanced research from King's College London and the UK Dementia Research Institute to create its therapeutic platform. By integrating a comprehensive understanding of brain biology with proprietary gene therapy techniques, AviadoBio seeks to address the challenges of effectively delivering treatments to targeted areas in the brain. This approach aims to enhance the therapeutic potential of gene therapies, offering hope for halting or potentially reversing the progression of these debilitating diseases.

Prime Medicine is a biotechnology company focused on developing genetic therapies using its proprietary Prime Editing technology. This next-generation gene editing method allows for precise "search and replace" modifications in the genome, aiming to restore normal genetic function and address the underlying causes of various diseases. By targeting a broad spectrum of conditions with significant unmet medical needs, Prime Medicine seeks to revolutionize the field of genetic medicine through effective and efficient therapeutic solutions.

Created by co-founders of the $40B DNA sequencing leader Illumina, Luna DNA incentivizes the sharing of health and DNA data for research. LunaDNA rewards people for sharing the data they already own while contributing to medical research and discovery that saves lives. DNA sequencing data has become siloed. As personal DNA sequencing has risen in popularity, the data has become siloed as each company looks to maximize their individual profit from this data. There is currently little incentive for consumers to contribute their DNA and health information to a third party database. There are also multiple privacy, security, and trust issues that limit broad participation. The solution is LunaDNA. LunaDNA is a community owned database that rewards individuals shares in the database and rewards for contributing their health and DNA data. Shares entitle members to a share in the profits from medical research and discoveries. The proceeds flow back to the community like dividends as researchers pay to access the data for discovery.

LunaPBC, Inc. is a Public Benefit Corporation based in San Diego, California, that develops a community-owned platform for genomic and medical research. The company's primary offering, LunaDNA, serves as a knowledge database where individuals can share their health and DNA data to contribute to health research and drive medical discoveries. This platform is designed to benefit both non-profit and for-profit health research projects, facilitating collaboration among pharmaceutical, insurance, and healthcare IT companies. In addition to advancing medical research, LunaPBC allows its members to share in the financial benefits derived from medical breakthroughs, promoting a model of shared community ownership.

Embark offers dog DNA testing that ends preventable diseases in dogs. It was founded by top experts in genomics, computational biology, and software design to sell an industry-leading dog DNA test. They use a clinical-grade CLIA-certified SNP chip to provide the most accurate and comprehensive results (200,000+ genetic markers) available on the market. Their data contributes to proprietary genetics research that will help dog owners optimize nutrition, exercise, allergies, and prevent a variety of heritable conditions. Their technological advantage, intellectual capital, and research-focus enable them to push canine health toward our shared vision of preventative veterinary care.

Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing precision genetic medicines using its innovative base editing technology. Founded in 2017, the company aims to provide lifelong cures for patients with serious diseases. Its therapeutic portfolio includes treatments for sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, pediatric acute myeloid leukemia, alpha-1 antitrypsin deficiency, and glycogen storage disorder 1A, as well as therapies for ocular and central nervous system disorders. Beam's base editing technology allows for targeted modifications at a single base in the genome without creating double-stranded breaks in DNA, positioning it as a pioneer in the field of genetic medicine. The company's pipeline includes several programs, such as BEAM-101, ESCAPE, BEAM-302, BEAM-301, and BEAM-201, reflecting its commitment to advancing genetic therapies.

Singular Genomics is a life science technology company focused on advancing next generation sequencing (NGS) and multiomics technologies to support researchers and clinicians. The company's core platform, the Singular Sequencing Engine, underpins its product offerings, emphasizing accuracy, speed, flexibility, and scalability. Singular Genomics has developed two primary integrated solutions: the G4 Integrated Solution, which targets the NGS market with a combination of instrument and consumable kits, and the PX Integrated Solution, which integrates single cell analysis, spatial analysis, genomics, and proteomics into a versatile platform. These solutions cater to various market segments, including basic biology, oncology, and immunology, enhancing research capabilities across diverse applications.

Sana Biotechnology is a biotechnology company based in Seattle, Washington, with additional offices in South San Francisco, California, and Cambridge, Massachusetts. Founded in 2018, the company specializes in developing engineered cells as medicines to treat a variety of diseases. Sana Biotechnology focuses on reprogramming cells in the body and replacing damaged cells and tissues, leveraging recent scientific advancements to create new therapeutic options. The company's research spans multiple therapeutic areas with significant unmet needs, including oncology, diabetes, B-cell-mediated autoimmune disorders, and central nervous system conditions. Its pipeline includes various product candidates aimed at addressing these challenges through innovative cell engineering programs.

Geneception is a company focused on the development of breakthrough in gene and cell therapies that are fully-integrated.

Genomics plc is focused on transforming healthcare through advanced genomic analysis. The company employs sophisticated methodologies to analyze large integrated datasets of genomic and phenotypic information, aiming to enhance understanding of human biology. By leveraging genetic inference technology, Genomics plc seeks to identify new drug targets for serious diseases and assess individual disease risk. Their analytical platform enables the exploration of genomic sequence data, providing valuable insights that support pharmaceutical companies in reducing risks associated with drug development. This approach not only aims to improve healthcare outcomes but also sets a standard for how organizations and patients can benefit from genomic data.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

Nebula Genomics is a company that focuses on human genome sequencing and health data, aiming to create a comprehensive online marketplace for genomic information. Founded in 2016 by Harvard genomics pioneer George Church and his colleagues, the company is headquartered in San Francisco, California, with additional offices in Boston, Massachusetts. Nebula Genomics utilizes blockchain technology to empower consumers by allowing them to maintain control over their genomic data and receive compensation for its use. The platform aggregates extensive genetic information, enabling researchers to analyze data for accelerated drug development and personalized medicine. The company’s DNA sequencing technology covers all genes, regulatory regions, the Y chromosome, and mitochondrial DNA, facilitating in-depth genetic analysis and variant exploration.

Modis Therapeutics is bringing disease-modifying therapies to patients with rare genetic diseases.

Benchling, Inc. is a developer of a cloud-based informatics platform tailored for life sciences research and development. The platform includes a suite of integrated applications that allow scientists to design, share, and document experiments, as well as manage biological entities like plasmids and antibodies. Key features include Benchling Insights, which enables users to visualize and collaborate on structured data, and a biological registration system for inventory tracking. Benchling's solutions cater to a diverse range of applications such as gene therapy, vaccines, and industrial biotechnology, serving scientists in biotech, pharmaceutical, academic, and government sectors. Established in 2012 and based in San Francisco, the company aims to enhance the speed and collaboration of life science research by providing tools that streamline workflows and facilitate critical R&D decisions.

Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing precision genetic medicines using its innovative base editing technology. Founded in 2017, the company aims to provide lifelong cures for patients with serious diseases. Its therapeutic portfolio includes treatments for sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, pediatric acute myeloid leukemia, alpha-1 antitrypsin deficiency, and glycogen storage disorder 1A, as well as therapies for ocular and central nervous system disorders. Beam's base editing technology allows for targeted modifications at a single base in the genome without creating double-stranded breaks in DNA, positioning it as a pioneer in the field of genetic medicine. The company's pipeline includes several programs, such as BEAM-101, ESCAPE, BEAM-302, BEAM-301, and BEAM-201, reflecting its commitment to advancing genetic therapies.

Biopalette is a company focused on developing advanced gene-editing techniques aimed at creating innovative therapies. The business operates a platform that combines genome editing and microbiome engineering, allowing for the cultivation of bacteria through targeted genome modifications. This technology enables scientists to explore and test various applications in gene editing, specifically tailored to different types of cells. Biopalette's approach positions it at the intersection of genetics and microbiome science, facilitating breakthroughs in therapeutic development.

Orchard Therapeutics is a biopharmaceutical company based in the United Kingdom that specializes in developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which modifies a patient's own hematopoietic stem cells to create a targeted treatment administered in a single dose. Orchard's portfolio includes Strimvelis, the first autologous gene therapy approved by the European Medicines Agency for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). Additionally, it is advancing clinical programs for various conditions, including metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has an extensive preclinical pipeline targeting mucopolysaccharidosis types I, IIIA, and IIIB. Orchard Therapeutics collaborates with leading institutions in gene therapy research, enhancing its efforts to transform the lives of patients with rare diseases in the UK, European Union, and the United States. Founded in 2015 and headquartered in London, it aims to provide sustainable clinical benefits through its innovative therapies.

Biopalette is a company focused on developing advanced gene-editing techniques aimed at creating innovative therapies. The business operates a platform that combines genome editing and microbiome engineering, allowing for the cultivation of bacteria through targeted genome modifications. This technology enables scientists to explore and test various applications in gene editing, specifically tailored to different types of cells. Biopalette's approach positions it at the intersection of genetics and microbiome science, facilitating breakthroughs in therapeutic development.

Biopalette is a company focused on developing advanced gene-editing techniques aimed at creating innovative therapies. The business operates a platform that combines genome editing and microbiome engineering, allowing for the cultivation of bacteria through targeted genome modifications. This technology enables scientists to explore and test various applications in gene editing, specifically tailored to different types of cells. Biopalette's approach positions it at the intersection of genetics and microbiome science, facilitating breakthroughs in therapeutic development.

Denali Therapeutics is a biotechnology company focused on the discovery and development of therapies for patients with neurodegenerative diseases, including Alzheimer's disease, Parkinson's disease, ALS and others. Denali was founded by scientists, industry experts and investors who share the vision that recent scientific insights into the genetic causes and biological processes underlying neurodegenerative disease, together with new translational medicine tools, offer an unprecedented opportunity to discover and develop effective medicines. Denali is rigorously pursuing a science-driven approach to translational medicine and clinical development.

Caribou Biosciences, Inc. is a biotechnology company focused on cellular engineering and genome editing using CRISPR technology. Established in 2011 and based in Berkeley, California, the company leverages its CRISPR-Cas gene editing platform to facilitate precise modifications in DNA across various applications. These include developing therapeutics for human and animal health, creating disease models, enhancing agricultural traits, and advancing industrial biotechnology. Caribou Biosciences is particularly dedicated to the development of genome-edited cell therapies, including allogeneic CAR-T and CAR-NK cell therapies, aimed at improving outcomes for patients with severe diseases. The company’s innovative approach combines CRISPR with guide RNA to enable targeted DNA alterations, thereby contributing to advancements in both therapeutic development and biological research.

Orchard Therapeutics is a biopharmaceutical company based in the United Kingdom that specializes in developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which modifies a patient's own hematopoietic stem cells to create a targeted treatment administered in a single dose. Orchard's portfolio includes Strimvelis, the first autologous gene therapy approved by the European Medicines Agency for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). Additionally, it is advancing clinical programs for various conditions, including metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has an extensive preclinical pipeline targeting mucopolysaccharidosis types I, IIIA, and IIIB. Orchard Therapeutics collaborates with leading institutions in gene therapy research, enhancing its efforts to transform the lives of patients with rare diseases in the UK, European Union, and the United States. Founded in 2015 and headquartered in London, it aims to provide sustainable clinical benefits through its innovative therapies.

Proteostasis Therapeutics is discovering and developing novel small molecule therapeutics designed to control the body's protein homeostasis, or Proteostasis Network. The Proteostasis Network maintains the body's natural balance of proteins to protect us from numerous diseases. These novel therapies, or Proteostasis Regulators, are designed to treat multiple genetic and degenerative disorders associated with deficiencies of the Proteostasis Network, such as emphysema, type II diabetes, Alzheimer's Disease and Huntington's Disease.

Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

REGENXBIO Inc. is a clinical-stage biotechnology company specializing in gene therapy aimed at addressing genetic defects and enabling cells to produce therapeutic proteins and antibodies. The company utilizes its proprietary NAV Technology Platform, which involves recombinant adeno-associated virus vectors for gene delivery. REGENXBIO’s lead candidate, RGX-314, is currently in Phase I/IIa clinical trials for the treatment of wet age-related macular degeneration. Additional product candidates under development include RGX-121 for mucopolysaccharidosis type II, RGX-111 for mucopolysaccharidosis type I, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia. Beyond its own product candidates, REGENXBIO also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and has a collaboration agreement with Neurimmune AG to develop vectorized antibodies for neurodegenerative diseases. Founded in 2008 and headquartered in Rockville, Maryland, the company was previously known as ReGenX Biosciences, LLC before rebranding in 2014.

Denali Therapeutics is a biotechnology company focused on the discovery and development of therapies for patients with neurodegenerative diseases, including Alzheimer's disease, Parkinson's disease, ALS and others. Denali was founded by scientists, industry experts and investors who share the vision that recent scientific insights into the genetic causes and biological processes underlying neurodegenerative disease, together with new translational medicine tools, offer an unprecedented opportunity to discover and develop effective medicines. Denali is rigorously pursuing a science-driven approach to translational medicine and clinical development.

Precision BioSciences, Inc. is a biotechnology company based in Durham, North Carolina, focused on genome editing and the development of therapeutic products. Utilizing its proprietary ARCUS genome editing platform, the company aims to create innovative solutions for human diseases and food applications. Precision BioSciences operates through two primary segments: Therapeutic and Food. The Therapeutic segment is engaged in developing allogeneic CAR T immunotherapies, with several candidates in clinical trials targeting various types of cancers, including acute lymphoblastic leukemia and multiple myeloma. Additionally, the company is involved in in vivo gene correction activities and has collaborations for developing treatments for chronic Hepatitis B. The Food segment concentrates on creating nutrition products. Founded in 2006, Precision BioSciences is dedicated to translating advanced gene editing technology into impactful products across the life sciences.

Dimension Therapeutics is a gene therapy company focused on developing novel treatments for rare diseases. Their team comprises biotech industry veterans and thought leaders in gene therapy and rare diseases. The company is focused on advancing its platform of gene therapy programs in rare diseases through clinical development, starting with lead programs in hemophilia, and building out a world-class product engine for adeno-associated virus (AAV) therapeutics. In conjunction with its launch, Dimension has entered into an exclusive license and collaboration with REGENX Biosciences. REGENX holds exclusive rights to a portfolio of over 100 patents and patent applications pertaining to its NAV vector technology and related applications. Through its license and collaboration with REGENX, Dimension has acquired preferred access to NAV vector technology and rights within REGENX product programs in multiple rare disease indications. Dimension has completed a Series A financing led by Fidelity Biosciences.

Caribou Biosciences, Inc. is a biotechnology company focused on cellular engineering and genome editing using CRISPR technology. Established in 2011 and based in Berkeley, California, the company leverages its CRISPR-Cas gene editing platform to facilitate precise modifications in DNA across various applications. These include developing therapeutics for human and animal health, creating disease models, enhancing agricultural traits, and advancing industrial biotechnology. Caribou Biosciences is particularly dedicated to the development of genome-edited cell therapies, including allogeneic CAR-T and CAR-NK cell therapies, aimed at improving outcomes for patients with severe diseases. The company’s innovative approach combines CRISPR with guide RNA to enable targeted DNA alterations, thereby contributing to advancements in both therapeutic development and biological research.

REGENXBIO Inc. is a clinical-stage biotechnology company specializing in gene therapy aimed at addressing genetic defects and enabling cells to produce therapeutic proteins and antibodies. The company utilizes its proprietary NAV Technology Platform, which involves recombinant adeno-associated virus vectors for gene delivery. REGENXBIO’s lead candidate, RGX-314, is currently in Phase I/IIa clinical trials for the treatment of wet age-related macular degeneration. Additional product candidates under development include RGX-121 for mucopolysaccharidosis type II, RGX-111 for mucopolysaccharidosis type I, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia. Beyond its own product candidates, REGENXBIO also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and has a collaboration agreement with Neurimmune AG to develop vectorized antibodies for neurodegenerative diseases. Founded in 2008 and headquartered in Rockville, Maryland, the company was previously known as ReGenX Biosciences, LLC before rebranding in 2014.

Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

Dimension Therapeutics is a gene therapy company focused on developing novel treatments for rare diseases. Their team comprises biotech industry veterans and thought leaders in gene therapy and rare diseases. The company is focused on advancing its platform of gene therapy programs in rare diseases through clinical development, starting with lead programs in hemophilia, and building out a world-class product engine for adeno-associated virus (AAV) therapeutics. In conjunction with its launch, Dimension has entered into an exclusive license and collaboration with REGENX Biosciences. REGENX holds exclusive rights to a portfolio of over 100 patents and patent applications pertaining to its NAV vector technology and related applications. Through its license and collaboration with REGENX, Dimension has acquired preferred access to NAV vector technology and rights within REGENX product programs in multiple rare disease indications. Dimension has completed a Series A financing led by Fidelity Biosciences.

Proteostasis Therapeutics is discovering and developing novel small molecule therapeutics designed to control the body's protein homeostasis, or Proteostasis Network. The Proteostasis Network maintains the body's natural balance of proteins to protect us from numerous diseases. These novel therapies, or Proteostasis Regulators, are designed to treat multiple genetic and degenerative disorders associated with deficiencies of the Proteostasis Network, such as emphysema, type II diabetes, Alzheimer's Disease and Huntington's Disease.