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 specifically for diverse and underserved populations that have historically been underrepresented in genetics research. Galatea Bio operates a biobank that serves as a biosample and bioinformatics platform, utilizing advanced algorithms and innovative analyses to facilitate genomic discoveries. Through its world-class clinical network, the company aims to identify patients at risk for severe multi-inflammatory syndromes or infections, ultimately working to develop new tests, treatments, and cures that enhance healthcare outcomes for all.

Rhygaze is a biotechnology company focused on developing innovative gene therapies aimed at restoring vision in individuals affected by blindness-related diseases. The company’s therapy utilizes advanced technology to deliver a light-sensor gene directly to cone cells that have lost their light sensitivity. By repairing these cells' ability to detect light, Rhygaze seeks to enable effective vision restoration for patients. Through its specialized approach, the company aims to make significant contributions to the healthcare industry and improve the quality of life for those facing vision loss.

Shift Bioscience is a biotechnology company focused on combating the diseases associated with aging through innovative cellular reprogramming techniques. The company has developed a high-throughput, high-accuracy aging biomarker, which is integrated into AI-driven simulations to identify safer gene factors for rejuvenation. By leveraging deep learning technology, Shift tests extensive gene combinations to discover potential drug candidates that can reverse aging and age-related diseases. The company's goal is to create a single family of drugs capable of addressing multiple age-related conditions, ultimately allowing for improved health and longevity through simple prescriptions.

Genomics PLC is a company based in Oxford, United Kingdom, specializing in algorithms and software solutions that focus on cancer, microbes, and rare diseases. Founded in 2014, it aims to revolutionize healthcare through advanced analyses of extensive genomic and phenotypic datasets. By leveraging its expertise, the company provides healthcare providers and governments with tools to enhance patient diagnoses and treatment options. Additionally, Genomics assists pharmaceutical and biotechnology firms in mitigating risks during the drug development process by providing insights into genomic data. Its analytical platform facilitates the investigation of genomic sequences, helping to identify individuals at risk of various diseases. Through these innovations, Genomics seeks to set standards in the use of genomic data, ultimately aiming to improve healthcare outcomes and advance human biology understanding.

Nvelop Therapeutics is a biotechnology company dedicated to developing advanced genetic medicines aimed at treating severe genetic diseases. The company specializes in creating innovative, programmable delivery systems that facilitate efficient in vivo gene editing and the delivery of therapeutic cargo. By integrating epigenetic editing technologies with next-generation delivery methods, Nvelop Therapeutics addresses significant unmet medical needs. Its focus is on delivering a range of therapeutic payloads, initially prioritizing gene editing within therapeutically relevant cells and tissues, thereby enhancing the potential for effective treatment options for patients suffering from serious genetic 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 a biotechnology company focused on developing transformative gene therapies for neurodegenerative disorders, specifically targeting diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The company leverages innovative research from King’s College London and the UK Dementia Research Institute to create its therapeutic platform. By integrating a deep understanding of brain biology with proprietary gene therapies, AviadoBio aims to effectively address the challenges of delivering treatments to the appropriate areas within the nervous system. The ultimate goal is to enhance the therapeutic potential of gene therapy, potentially halting or even reversing the progression of these debilitating conditions, thereby improving the quality of life for affected individuals.

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 specifically for diverse and underserved populations that have historically been underrepresented in genetics research. Galatea Bio operates a biobank that serves as a biosample and bioinformatics platform, utilizing advanced algorithms and innovative analyses to facilitate genomic discoveries. Through its world-class clinical network, the company aims to identify patients at risk for severe multi-inflammatory syndromes or infections, ultimately working to develop new tests, treatments, and cures that enhance healthcare outcomes for all.

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 innovative Prime Editing technology. This next-generation gene editing method allows for precise "search and replace" modifications in the genome, aimed at restoring normal genetic function. The company targets a wide range of diseases with significant unmet medical needs, leveraging the efficiency and versatility of its gene editing capabilities to address the underlying causes of these conditions. Through its commitment to advancing gene editing, Prime Medicine seeks to transform the landscape of genetic medicine.

Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for the treatment of severe diseases affecting the muscle and central nervous system (CNS). Established in 2019 and based in Waltham, Massachusetts, the company specializes in creating adeno-associated virus (AAV) vectors and utilizes a platform that integrates synthetic and systems biology with high-throughput screening techniques. This innovative approach allows Affinia Therapeutics to design novel gene therapies with enhanced properties tailored to address significant unmet medical needs. The company aims to provide healthcare professionals with advanced therapeutic options for patients suffering from debilitating conditions.

Genomics PLC is a company based in Oxford, United Kingdom, specializing in algorithms and software solutions that focus on cancer, microbes, and rare diseases. Founded in 2014, it aims to revolutionize healthcare through advanced analyses of extensive genomic and phenotypic datasets. By leveraging its expertise, the company provides healthcare providers and governments with tools to enhance patient diagnoses and treatment options. Additionally, Genomics assists pharmaceutical and biotechnology firms in mitigating risks during the drug development process by providing insights into genomic data. Its analytical platform facilitates the investigation of genomic sequences, helping to identify individuals at risk of various diseases. Through these innovations, Genomics seeks to set standards in the use of genomic data, ultimately aiming to improve healthcare outcomes and advance human biology understanding.

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.

Verve Therapeutics is a genetic medicines company based in Cambridge, Massachusetts, focused on innovative therapies for cardiovascular disease. Founded in 2018, the company aims to transform the management of cardiovascular conditions from chronic treatments to single-course gene editing medicines. Verve Therapeutics employs advanced gene-editing technology and human genetic analysis to develop targeted therapies that reduce the risk of coronary artery diseases. Its initial programs focus on the PCSK9 and ANGPTL3 genes, which are crucial for lowering harmful blood lipids like low-density lipoprotein cholesterol. The company collaborates with strategic partners, including Beam Therapeutics for delivery technologies and Verily for gene editing delivery vehicles, enhancing its capabilities in the biotechnology and healthcare sectors.

NFlection Therapeutics, Inc. is a biotechnology company dedicated to discovering and developing targeted therapies for rare disorders, particularly focusing on neurofibromatosis type 1 and related conditions. The company specializes in addressing diseases driven by the aberrant activation of the Ras/Raf/MEK/ERK pathway, including cutaneous neurofibromas and other conditions such as immunosuppressant-mediated squamous cell carcinoma and congenital birthmarks. NFlection Therapeutics is known for its innovative approach to drug formulation, offering treatments in a cosmetically elegant gel that can be applied topically, allowing for localized suppression of the Ras pathway while minimizing systemic side effects associated with oral medications. Established in 2014, the company is headquartered in Wayne, Pennsylvania.

Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for the treatment of severe diseases affecting the muscle and central nervous system (CNS). Established in 2019 and based in Waltham, Massachusetts, the company specializes in creating adeno-associated virus (AAV) vectors and utilizes a platform that integrates synthetic and systems biology with high-throughput screening techniques. This innovative approach allows Affinia Therapeutics to design novel gene therapies with enhanced properties tailored to address significant unmet medical needs. The company aims to provide healthcare professionals with advanced therapeutic options for patients suffering from debilitating conditions.

AviadoBio is a biotechnology company focused on developing transformative gene therapies for neurodegenerative disorders, specifically targeting diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The company leverages innovative research from King’s College London and the UK Dementia Research Institute to create its therapeutic platform. By integrating a deep understanding of brain biology with proprietary gene therapies, AviadoBio aims to effectively address the challenges of delivering treatments to the appropriate areas within the nervous system. The ultimate goal is to enhance the therapeutic potential of gene therapy, potentially halting or even reversing the progression of these debilitating conditions, thereby improving the quality of life for affected individuals.

Prime Medicine is a biotechnology company focused on developing genetic therapies using its innovative Prime Editing technology. This next-generation gene editing method allows for precise "search and replace" modifications in the genome, aimed at restoring normal genetic function. The company targets a wide range of diseases with significant unmet medical needs, leveraging the efficiency and versatility of its gene editing capabilities to address the underlying causes of these conditions. Through its commitment to advancing gene editing, Prime Medicine seeks to transform the landscape of genetic medicine.

LunaPBC, Inc. is a Public Benefit Corporation based in San Diego, California, focused on developing a community-owned platform for health and DNA research. The company's primary offering is LunaDNA, a genomic and medical research knowledge database that enables individuals to share their health and DNA data for research purposes. This platform facilitates collaborations with pharmaceutical companies, insurance providers, and healthcare IT organizations to drive advancements in medical discoveries. By allowing members to contribute their health data, LunaPBC not only supports vital health research projects but also ensures that participants can share in the financial benefits arising from medical breakthroughs, fostering a sense of community ownership and engagement in the research process.

LunaDNA is a community-owned genomic and medical research platform founded by the creators of a leading DNA sequencing company. It facilitates the sharing of health and DNA data, allowing individuals to contribute their personal information for medical research while maintaining ownership of their data. By incentivizing participation, LunaDNA addresses the challenge of data silos that have emerged as personal DNA sequencing has gained popularity. Participants are rewarded with shares in the database, granting them a stake in the profits generated from medical research. These profits are returned to the community as dividends when researchers pay for access to the data. LunaDNA empowers individuals to support health research while ensuring that their contributions benefit the broader community and drive medical discoveries.

Verve Therapeutics is a genetic medicines company based in Cambridge, Massachusetts, focused on innovative therapies for cardiovascular disease. Founded in 2018, the company aims to transform the management of cardiovascular conditions from chronic treatments to single-course gene editing medicines. Verve Therapeutics employs advanced gene-editing technology and human genetic analysis to develop targeted therapies that reduce the risk of coronary artery diseases. Its initial programs focus on the PCSK9 and ANGPTL3 genes, which are crucial for lowering harmful blood lipids like low-density lipoprotein cholesterol. The company collaborates with strategic partners, including Beam Therapeutics for delivery technologies and Verily for gene editing delivery vehicles, enhancing its capabilities in the biotechnology and healthcare sectors.

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.

Singular Genomics is a life science technology company that develops innovative sequencing platforms. Its core product, the Singular Sequencing Engine, powers two integrated solutions: G4 for next-generation sequencing (NGS) markets, offering high accuracy and speed; and PX, which combines single-cell analysis, spatial analysis, genomics, and proteomics in one versatile multi-omics instrument. These products aim to empower researchers and clinicians across diverse fields such as basic biology, oncology, immunology, and more.

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

Genomics PLC is a company based in Oxford, United Kingdom, specializing in algorithms and software solutions that focus on cancer, microbes, and rare diseases. Founded in 2014, it aims to revolutionize healthcare through advanced analyses of extensive genomic and phenotypic datasets. By leveraging its expertise, the company provides healthcare providers and governments with tools to enhance patient diagnoses and treatment options. Additionally, Genomics assists pharmaceutical and biotechnology firms in mitigating risks during the drug development process by providing insights into genomic data. Its analytical platform facilitates the investigation of genomic sequences, helping to identify individuals at risk of various diseases. Through these innovations, Genomics seeks to set standards in the use of genomic data, ultimately aiming to improve healthcare outcomes and advance human biology understanding.

Avidity Biosciences, Inc. is a biopharmaceutical company based in La Jolla, California, specializing in the development of oligonucleotide-based therapies, particularly through its innovative Antibody Oligonucleotide Conjugates (AOC) platform. This technology aims to address the limitations of traditional oligonucleotide therapies by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide treatments. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disease. In addition to this, the company is advancing multiple programs focused on muscle-related conditions, including muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Furthermore, Avidity is expanding its research to include immune and other cell types, positioning itself at the forefront of biopharmaceutical innovation for serious diseases.

Nebula Genomics is a biotechnology company founded in 2016 and based in San Francisco, California. It specializes in human genome sequencing and the creation of a marketplace for genomic and health data aimed at consumers, researchers, and the medical community. The company's mission is to facilitate genomic sequencing and enhance personalized medicine by aggregating a substantial repository of genetic information. Utilizing blockchain technology, Nebula Genomics ensures that consumers retain control over their data and receive compensation for its use. The marketplace enables researchers to analyze rich genetic datasets, which can accelerate drug development and improve the efficiency of clinical trials. Founded by prominent genomics researcher George Church and his associates, Nebula Genomics offers advanced DNA sequencing technology that allows clients to explore all genes and genetic variants in the genome, thereby providing comprehensive insights into individual health.

Modis Therapeutics, Inc. is a biopharmaceutical company focused on developing disease-modifying therapies for rare genetic diseases characterized by high unmet medical needs. Incorporated in 2016 and based in Oakland, California, the company is known for its lead therapy, MT1621, which aims to restore mitochondrial DNA replication fidelity. This therapy specifically targets thymidine kinase 2 deficiency (TK2d), an inherited mitochondrial disorder. By improving nucleotide balance and increasing mitochondrial DNA copy number, Modis Therapeutics' approach seeks to enhance cellular function and prolong the lives of affected patients. As of September 2019, Modis Therapeutics operates as a subsidiary of Zogenix, Inc.

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.

Nebula Genomics is a biotechnology company founded in 2016 and based in San Francisco, California. It specializes in human genome sequencing and the creation of a marketplace for genomic and health data aimed at consumers, researchers, and the medical community. The company's mission is to facilitate genomic sequencing and enhance personalized medicine by aggregating a substantial repository of genetic information. Utilizing blockchain technology, Nebula Genomics ensures that consumers retain control over their data and receive compensation for its use. The marketplace enables researchers to analyze rich genetic datasets, which can accelerate drug development and improve the efficiency of clinical trials. Founded by prominent genomics researcher George Church and his associates, Nebula Genomics offers advanced DNA sequencing technology that allows clients to explore all genes and genetic variants in the genome, thereby providing comprehensive insights into individual health.

Bio Palette is a company focused on advancing gene-editing techniques and developing therapies through innovative technology. The firm operates a platform that combines genome editing with microbiome engineering, allowing for the cultivation of bacteria tailored through genetic modifications. This approach enables researchers to explore and test the potential of gene editing in various applications. Bio Palette categorizes its business development into three fields based on the types of cells targeted for genome editing, positioning itself at the forefront of biotechnology and therapeutic innovation.

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.

Bio Palette is a company focused on advancing gene-editing techniques and developing therapies through innovative technology. The firm operates a platform that combines genome editing with microbiome engineering, allowing for the cultivation of bacteria tailored through genetic modifications. This approach enables researchers to explore and test the potential of gene editing in various applications. Bio Palette categorizes its business development into three fields based on the types of cells targeted for genome editing, positioning itself at the forefront of biotechnology and therapeutic innovation.

Bio Palette is a company focused on advancing gene-editing techniques and developing therapies through innovative technology. The firm operates a platform that combines genome editing with microbiome engineering, allowing for the cultivation of bacteria tailored through genetic modifications. This approach enables researchers to explore and test the potential of gene editing in various applications. Bio Palette categorizes its business development into three fields based on the types of cells targeted for genome editing, positioning itself at the forefront of biotechnology and therapeutic innovation.

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 aimed at treating cystic fibrosis (CF) and other genetic and degenerative disorders. The company specializes in discovering small molecule therapeutics that regulate the Proteostasis Network, which is essential for maintaining the balance of proteins in the body. Its lead product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis Therapeutics employs a theratyping approach to tailor treatments based on individual responses, regardless of specific CFTR mutations. The company has established collaboration agreements with organizations such as the Cystic Fibrosis Foundation to advance research and commercialization efforts for CF and other pulmonary diseases. Founded in 2006 and headquartered in Boston, Massachusetts, Proteostasis Therapeutics was previously known as Proteoguard, Inc. before rebranding in 2007.

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).

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 based in Cambridge, Massachusetts, specializing in the development of innovative treatments for rare and metabolic diseases associated with the liver. Founded in 2013, the company has a robust pipeline of gene therapy programs, including DTX101 and DTX201 for hemophilia B and A, respectively. Additionally, its portfolio encompasses DTX301 for ornithine transcarbamylase deficiency, DTX401 for glycogen storage disease type Ia, DTX501 for phenylketonuria, DTX701 for Wilson disease, and DTX601 for citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, gaining access to a significant portfolio of patents related to adeno-associated virus (AAV) therapeutics. This collaboration supports the company's aim to advance its gene therapy platform and enhance its product development capabilities in the treatment of rare diseases. Dimension Therapeutics operates as a subsidiary of Ultragenyx Pharmaceutical Inc. since November 2017.

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.

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 based in Cambridge, Massachusetts, specializing in the development of innovative treatments for rare and metabolic diseases associated with the liver. Founded in 2013, the company has a robust pipeline of gene therapy programs, including DTX101 and DTX201 for hemophilia B and A, respectively. Additionally, its portfolio encompasses DTX301 for ornithine transcarbamylase deficiency, DTX401 for glycogen storage disease type Ia, DTX501 for phenylketonuria, DTX701 for Wilson disease, and DTX601 for citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, gaining access to a significant portfolio of patents related to adeno-associated virus (AAV) therapeutics. This collaboration supports the company's aim to advance its gene therapy platform and enhance its product development capabilities in the treatment of rare diseases. Dimension Therapeutics operates as a subsidiary of Ultragenyx Pharmaceutical Inc. since November 2017.

Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics aimed at treating cystic fibrosis (CF) and other genetic and degenerative disorders. The company specializes in discovering small molecule therapeutics that regulate the Proteostasis Network, which is essential for maintaining the balance of proteins in the body. Its lead product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis Therapeutics employs a theratyping approach to tailor treatments based on individual responses, regardless of specific CFTR mutations. The company has established collaboration agreements with organizations such as the Cystic Fibrosis Foundation to advance research and commercialization efforts for CF and other pulmonary diseases. Founded in 2006 and headquartered in Boston, Massachusetts, Proteostasis Therapeutics was previously known as Proteoguard, Inc. before rebranding in 2007.