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.

Rhygaze is focused on developing a novel gene therapy aimed at restoring vision in patients affected by diseases that lead to blindness. The company's innovative technology specifically targets cone cells, which are crucial for light detection but may lose sensitivity due to various conditions. By delivering a light-sensor gene directly to these compromised cone cells, Rhygaze seeks to repair their ability to detect light, thereby offering potential vision restoration solutions for individuals suffering from blindness.

Shift Bioscience is focused on using advanced simulation techniques to address the challenges of aging and age-related diseases. The company has developed a high-throughput aging biomarker that facilitates the identification of rejuvenation gene factors through AI-driven cell simulations. By employing deep learning technology, Shift tests extensive gene combinations to discover potential drugs that can reverse aging effects and repair mitochondrial genes. The ultimate goal is to create a single family of drugs capable of treating various diseases associated with aging, thereby promoting sustained health into old age through accessible prescriptions.

Genomics PLC, established in 2014 and headquartered in Oxford, UK, specializes in developing algorithms and software solutions that harness the power of genomics. The company focuses on cancer, microbes, and rare diseases, offering solutions that transform healthcare provisions for governments and providers, de-risk drug development for pharmaceutical clients, enhance diagnoses and treatment options for clinicians, and ensure accurate result processing for research labs and genome analysts. By applying advanced analytics to massive genomic and phenotypic datasets, Genomics PLC aims to lead the genomic transformation of healthcare, revealing the human wiring diagram and setting standards for genomic data utilization.

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, 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.

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.

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.

Genomics PLC, established in 2014 and headquartered in Oxford, UK, specializes in developing algorithms and software solutions that harness the power of genomics. The company focuses on cancer, microbes, and rare diseases, offering solutions that transform healthcare provisions for governments and providers, de-risk drug development for pharmaceutical clients, enhance diagnoses and treatment options for clinicians, and ensure accurate result processing for research labs and genome analysts. By applying advanced analytics to massive genomic and phenotypic datasets, Genomics PLC aims to lead the genomic transformation of healthcare, revealing the human wiring diagram and setting standards for genomic data utilization.

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.

Sana Biotechnology, Inc. is a biotechnology company focused on developing engineered cells as therapies to treat a wide range of diseases. Established in 2018 and headquartered in Seattle, Washington, with additional offices in South San Francisco and Cambridge, the company utilizes advanced scientific techniques to reprogram cells or replace damaged cells and tissues. This innovative approach aims to create a new class of medicines that address unmet treatment needs across various therapeutic areas, including oncology, diabetes, autoimmune disorders, and central nervous system conditions. Sana's pipeline includes several product candidates designed to provide meaningful medical solutions and transform disease treatment methodologies.

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.

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.

LunaDNA is a community-owned platform that facilitates the sharing of health and DNA data for medical research. Founded by the creators of Illumina, a leader in DNA sequencing, LunaDNA addresses the issue of siloed genomic data by providing individuals with a compelling incentive to share their health information. Participants are rewarded with shares in the database, allowing them to benefit from the profits generated by medical research and discoveries. This model not only empowers users to contribute their health records, genomic data, and personal health experiences but also fosters a sense of community ownership. As researchers pay to access the data for their studies, the proceeds are distributed back to the community, akin to dividends. By prioritizing privacy and trust, LunaDNA aims to increase participation in genomic research while facilitating advancements in healthcare that can ultimately save lives.

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.

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, specializing in the development of precision genetic medicines utilizing its innovative base editing technology. Founded in 2017, the company aims to provide lifelong cures for patients suffering from serious diseases by targeting specific bases in the genome without inducing double-stranded breaks in DNA. Beam Therapeutics is actively developing therapies for a range of conditions, including sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, pediatric acute myeloid leukemia, alpha-1 antitrypsin deficiency, glycogen storage disorder type 1A, as well as ocular and central nervous system disorders. The company’s diverse pipeline includes several programs such as BEAM-101, BEAM-201, BEAM-301, BEAM-302, and ESCAPE, reflecting its commitment to advancing gene correction, gene modification, gene activation, gene silencing, and multiplex editing technologies.

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

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, Inc. is a biotechnology company focused on developing engineered cells as therapies to treat a wide range of diseases. Established in 2018 and headquartered in Seattle, Washington, with additional offices in South San Francisco and Cambridge, the company utilizes advanced scientific techniques to reprogram cells or replace damaged cells and tissues. This innovative approach aims to create a new class of medicines that address unmet treatment needs across various therapeutic areas, including oncology, diabetes, autoimmune disorders, and central nervous system conditions. Sana's pipeline includes several product candidates designed to provide meaningful medical solutions and transform disease treatment methodologies.

Genomics PLC, established in 2014 and headquartered in Oxford, UK, specializes in developing algorithms and software solutions that harness the power of genomics. The company focuses on cancer, microbes, and rare diseases, offering solutions that transform healthcare provisions for governments and providers, de-risk drug development for pharmaceutical clients, enhance diagnoses and treatment options for clinicians, and ensure accurate result processing for research labs and genome analysts. By applying advanced analytics to massive genomic and phenotypic datasets, Genomics PLC aims to lead the genomic transformation of healthcare, revealing the human wiring diagram and setting standards for genomic data utilization.

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, Inc. is a biopharmaceutical company focused on developing disease-modifying therapies for rare genetic diseases with significant unmet medical needs. The company, based in Oakland, California, was incorporated in 2016 and is a subsidiary of Zogenix, Inc. Modis Therapeutics is advancing its lead therapy, MT1621, which aims to restore mitochondrial DNA replication fidelity. This therapeutic candidate is specifically designed to address thymidine kinase 2 deficiency (TK2d), an inherited mitochondrial disorder. By improving nucleotide balance, increasing mitochondrial DNA copy number, and enhancing cell function, Modis Therapeutics aims to provide meaningful treatment options that can prolong the lives of affected patients.

Sana Biotechnology, Inc. is a biotechnology company focused on developing engineered cells as therapies to treat a wide range of diseases. Established in 2018 and headquartered in Seattle, Washington, with additional offices in South San Francisco and Cambridge, the company utilizes advanced scientific techniques to reprogram cells or replace damaged cells and tissues. This innovative approach aims to create a new class of medicines that address unmet treatment needs across various therapeutic areas, including oncology, diabetes, autoimmune disorders, and central nervous system conditions. Sana's pipeline includes several product candidates designed to provide meaningful medical solutions and transform disease treatment methodologies.

Benchling, Inc. is a cloud-based informatics company that provides a comprehensive platform tailored for life sciences research and development. Its flagship offering allows scientists to design, share, and document experiments through an integrated interface, enhancing collaboration and efficiency. The platform includes Benchling Insights, which enables users to query and visualize structured data, and a Bioregistry system for managing biological entity registrations and inventory such as plasmids and cell strains. Benchling's solutions support a variety of applications, including antibodies, cell therapy, gene therapy, and vaccines, catering to the needs of scientists in biotech, pharmaceutical, academic, and government sectors. Founded in 2012 and headquartered in San Francisco, Benchling aims to streamline the research process from discovery to bioprocessing, facilitating critical R&D decisions with its unified software suite.

Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, specializing in the development of precision genetic medicines utilizing its innovative base editing technology. Founded in 2017, the company aims to provide lifelong cures for patients suffering from serious diseases by targeting specific bases in the genome without inducing double-stranded breaks in DNA. Beam Therapeutics is actively developing therapies for a range of conditions, including sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, pediatric acute myeloid leukemia, alpha-1 antitrypsin deficiency, glycogen storage disorder type 1A, as well as ocular and central nervous system disorders. The company’s diverse pipeline includes several programs such as BEAM-101, BEAM-201, BEAM-301, BEAM-302, and ESCAPE, reflecting its commitment to advancing gene correction, gene modification, gene activation, gene silencing, and multiplex editing technologies.

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.

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

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.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

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 biopharmaceutical company dedicated to discovering and developing therapies for neurodegenerative diseases such as Alzheimer's, Parkinson's disease, and amyotrophic lateral sclerosis (ALS). The company is advancing several therapeutic candidates, including LRRK2 inhibitors like DNL201 and DNL151 for Parkinson's disease, and DNL747, a RIPK1 inhibitor targeting Alzheimer's and ALS. Denali's innovative approaches also include enzyme replacement therapy for MPS II and antibody transport vehicle programs aimed at addressing alpha-synuclein and Tau pathology. Established in 2013 and headquartered in South San Francisco, California, Denali collaborates with notable organizations including Takeda, Genentech, and the Michael J. Fox Foundation to leverage research and development in the field of neurodegeneration. The company aims to translate scientific insights into effective treatments, contributing to the ongoing effort to combat these debilitating conditions.

Caribou Biosciences, Inc. is a biotechnology company based in Berkeley, California, that specializes in cellular engineering and genome editing solutions using CRISPR technology. Incorporated in 2011, the company has developed a CRISPR-Cas gene editing platform that allows precise modifications to DNA, which is applicable across various fields including human and animal therapeutics, agricultural biotechnology, and industrial biotech. Caribou's innovative approach aims to create transformative therapies, particularly in the development of allogeneic CAR-T and CAR-NK cell therapies, targeting serious diseases. The company focuses on advancing its pipeline of next-generation genome-edited cell therapies while also contributing to basic and applied biological research.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

Regenxbio Inc. is a clinical-stage biotechnology company focused on developing gene therapy products to address genetic disorders and enable cells to produce therapeutic proteins and antibodies. The company leverages its proprietary NAV Technology Platform, which utilizes adeno-associated virus vectors for gene delivery. Regenxbio's lead product candidate, RGX-314, is under investigation in a Phase I/IIa clinical trial for wet age-related macular degeneration. Other notable product candidates include RGX-121 and RGX-111, which are in clinical trials for mucopolysaccharidosis type II and I, respectively, as well as RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia. Additionally, Regenxbio licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and collaborates 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 biopharmaceutical company dedicated to discovering and developing therapies for neurodegenerative diseases such as Alzheimer's, Parkinson's disease, and amyotrophic lateral sclerosis (ALS). The company is advancing several therapeutic candidates, including LRRK2 inhibitors like DNL201 and DNL151 for Parkinson's disease, and DNL747, a RIPK1 inhibitor targeting Alzheimer's and ALS. Denali's innovative approaches also include enzyme replacement therapy for MPS II and antibody transport vehicle programs aimed at addressing alpha-synuclein and Tau pathology. Established in 2013 and headquartered in South San Francisco, California, Denali collaborates with notable organizations including Takeda, Genentech, and the Michael J. Fox Foundation to leverage research and development in the field of neurodegeneration. The company aims to translate scientific insights into effective treatments, contributing to the ongoing effort to combat these debilitating conditions.

Precision BioSciences, Inc. is a biotechnology company focused on genome editing, headquartered in Durham, North Carolina. The company utilizes its proprietary ARCUS technology to develop therapeutic products aimed at treating human diseases and improving food and agricultural solutions. Precision BioSciences operates through two segments: Therapeutic and Food. The Therapeutic segment is particularly dedicated to advancing allogeneic CAR T immunotherapies for cancer treatment, including candidates like PBCAR0191 and PBCAR20A, which target specific tumor antigens. The company is also involved in in vivo gene correction and is exploring treatments for conditions such as acute lymphoblastic leukemia and chronic lymphocytic leukemia. Additionally, the Food segment develops innovative nutrition products. Precision BioSciences has formed strategic collaborations, including agreements with Shire Plc and Gilead Sciences, to enhance its research and development capabilities. Founded in 2006, the company aims to harness its next-generation gene editing technology to address pressing health challenges.

Dimension Therapeutics, Inc. is a gene therapy company dedicated to developing innovative treatments for rare and metabolic diseases linked to liver function. Founded in 2013 and based in Cambridge, Massachusetts, the company has established a robust pipeline of gene therapy candidates. Its lead products include DTX101 for hemophilia B and DTX201 for hemophilia A, alongside other programs targeting conditions such as ornithine transcarbamylase deficiency, glycogen storage disease type Ia, phenylketonuria, Wilson disease, and citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, leveraging its extensive patent portfolio related to NAV vector technology. This partnership enhances Dimension's ability to advance its gene therapy initiatives, particularly in the realm of adeno-associated virus therapeutics. In 2017, Dimension Therapeutics became a subsidiary of Ultragenyx Pharmaceutical Inc.

Caribou Biosciences, Inc. is a biotechnology company based in Berkeley, California, that specializes in cellular engineering and genome editing solutions using CRISPR technology. Incorporated in 2011, the company has developed a CRISPR-Cas gene editing platform that allows precise modifications to DNA, which is applicable across various fields including human and animal therapeutics, agricultural biotechnology, and industrial biotech. Caribou's innovative approach aims to create transformative therapies, particularly in the development of allogeneic CAR-T and CAR-NK cell therapies, targeting serious diseases. The company focuses on advancing its pipeline of next-generation genome-edited cell therapies while also contributing to basic and applied biological research.

Regenxbio Inc. is a clinical-stage biotechnology company focused on developing gene therapy products to address genetic disorders and enable cells to produce therapeutic proteins and antibodies. The company leverages its proprietary NAV Technology Platform, which utilizes adeno-associated virus vectors for gene delivery. Regenxbio's lead product candidate, RGX-314, is under investigation in a Phase I/IIa clinical trial for wet age-related macular degeneration. Other notable product candidates include RGX-121 and RGX-111, which are in clinical trials for mucopolysaccharidosis type II and I, respectively, as well as RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia. Additionally, Regenxbio licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and collaborates 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 dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

Dimension Therapeutics, Inc. is a gene therapy company dedicated to developing innovative treatments for rare and metabolic diseases linked to liver function. Founded in 2013 and based in Cambridge, Massachusetts, the company has established a robust pipeline of gene therapy candidates. Its lead products include DTX101 for hemophilia B and DTX201 for hemophilia A, alongside other programs targeting conditions such as ornithine transcarbamylase deficiency, glycogen storage disease type Ia, phenylketonuria, Wilson disease, and citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, leveraging its extensive patent portfolio related to NAV vector technology. This partnership enhances Dimension's ability to advance its gene therapy initiatives, particularly in the realm of adeno-associated virus therapeutics. In 2017, Dimension Therapeutics became a subsidiary of Ultragenyx Pharmaceutical Inc.

Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.