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 employs simulation-guided techniques to identify safer gene factors for cellular rejuvenation, aiming to develop a drug family that treats multiple age-related diseases. Its high-throughput aging biomarker enables accurate cell simulations, with the goal of making full health in old age accessible via prescription.

RareCyte, Inc. is a life sciences company based in Seattle, Washington, founded in 2009. The company specializes in developing advanced platforms for liquid and tissue biopsy analysis, focusing on the detection and characterization of rare cells, particularly circulating tumor cells (CTCs) and circulating endothelial cells (CECs) in blood samples. RareCyte's AccuCyte system provides an open platform that facilitates the unbiased discovery and isolation of these rare cells, supporting various applications in cancer research, cardiovascular disease studies, and diagnostic development. The company offers a range of products, including sample preparation systems, staining kits, automated fluorescence microscopy instruments, and slide imaging systems. By enabling precision medicine approaches, RareCyte aims to provide new tools for medical professionals and researchers to address complex questions in the fields of cancer and other therapeutic areas.

Founded in 2014, Genomics develops algorithms and software solutions focused on cancer, microbes, and rare diseases. Its solutions empower governments, healthcare providers, pharmaceutical companies, clinicians, and researchers to improve diagnoses, treatment options, and drug development processes through accurate genomic data analysis.

T-Therapeutics is a biopharmaceutical company focused on developing engineered soluble biologics for oncological and immunological applications. The company utilizes a proprietary T cell receptor (TCR) discovery platform based on a highly humanized mouse model, which allows access to TCRs for human antigens that are typically unavailable from human samples. This innovative approach enables the binding of specific peptide-MHC (pMHC) targets on cells, facilitating the recruitment of T cells to combat cancer or modulate immune responses. Through its advanced capabilities in mouse genome engineering, single-cell genomics, machine learning, and structural biology, T-Therapeutics aims to enhance the quality of life for patients suffering from chronic and infectious diseases. The company's culture emphasizes creativity and collaboration, fostering an environment conducive to groundbreaking research and development in the biopharmaceutical field.

Adela specializes in developing innovative technologies for early detection of high-morbidity conditions like cancer through routine blood tests. Its proprietary platform efficiently captures and analyzes the methylome, focusing on highly informative regions to enhance diagnostic accuracy.

Nvelop Therapeutics develops programmable delivery systems for in vivo gene editing and therapeutic cargo delivery. It combines epigenetic editing technologies with next-generation delivery methods to address unmet medical needs. The company focuses on genetic medicines designed to target cells and tissues within the body, aiming to treat severe genetic diseases by delivering gene-editing payloads to therapeutically relevant sites. Early efforts are aligned with enabling editing in cells and tissues that have been difficult to reach, with the objective of expanding the range of actionable genetic therapies. The company seeks to advance in vivo approaches that can improve precision and safety in delivering therapeutic cargo to patients.

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.

Immuneel Therapeutics specializes in developing innovative cell and gene therapies for cancer treatment. The company focuses on CAR-T cell therapies, which are created by extracting blood cells from patients and genetically engineering healthy immune cells to specifically target malignant cells. This approach allows cancer patients to benefit from advanced cell-based immunotherapies that have the potential to transform their treatment outcomes.

AviadoBio focuses on developing transformative gene therapies for devastating neurodegenerative disorders such as frontotemporal dementia and amyotrophic lateral sclerosis. The company's platform combines proprietary technologies with a deep understanding of the brain to deliver effective treatments.

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.

RareCyte, Inc. is a life sciences company based in Seattle, Washington, founded in 2009. The company specializes in developing advanced platforms for liquid and tissue biopsy analysis, focusing on the detection and characterization of rare cells, particularly circulating tumor cells (CTCs) and circulating endothelial cells (CECs) in blood samples. RareCyte's AccuCyte system provides an open platform that facilitates the unbiased discovery and isolation of these rare cells, supporting various applications in cancer research, cardiovascular disease studies, and diagnostic development. The company offers a range of products, including sample preparation systems, staining kits, automated fluorescence microscopy instruments, and slide imaging systems. By enabling precision medicine approaches, RareCyte aims to provide new tools for medical professionals and researchers to address complex questions in the fields of cancer and other therapeutic areas.

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.

Adela specializes in developing innovative technologies for early detection of high-morbidity conditions like cancer through routine blood tests. Its proprietary platform efficiently captures and analyzes the methylome, focusing on highly informative regions to enhance diagnostic accuracy.

Affinia Therapeutics develops adeno-associated virus (AAV) gene therapies for muscular and central nervous system diseases. The company uses an AAV vector design platform built on synthetic and systems biology, high-throughput screening, and tissue- and single-cell-resolution analyses to create vectors with improved properties. It leverages sequencing, artificial intelligence, and structural modeling to enable rational design of therapies. Headquartered in Waltham, Massachusetts, Affinia Therapeutics was founded in 2019 and targets diseases with significant unmet needs in muscle and CNS.

Founded in 2014, Genomics develops algorithms and software solutions focused on cancer, microbes, and rare diseases. Its solutions empower governments, healthcare providers, pharmaceutical companies, clinicians, and researchers to improve diagnoses, treatment options, and drug development processes through accurate genomic data analysis.

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.

J-Pharma specializes in developing novel pharmaceuticals based on a human-genomic approach. Its intellectual property focuses on cell membrane transporters linked to various diseases, enabling the creation of targeted drugs and diagnostic methods. The company aims to improve global health and enhance quality of life for aging populations by generating new drug agents.

Founded in 2018, Sana Biotechnology focuses on creating and delivering engineered cells as medicine for patients. The company aims to reprogram cells in the body or replace damaged ones to treat a broad range of diseases, with a pipeline including SC291, SC262, SC255, UP421, and other candidates across therapeutic areas like oncology, diabetes, autoimmune disorders, and CNS.

Verve Therapeutics is a biotechnology company developing genetic medicines for cardiovascular disease, aiming to transform treatment from chronic management to single-course gene editing therapies. It leverages human genetics analysis and gene-editing technology, with expertise in cardiovascular medicine, human genetics, gene editing, delivery technologies, drug development, and commercialization.

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 develops adeno-associated virus (AAV) gene therapies for muscular and central nervous system diseases. The company uses an AAV vector design platform built on synthetic and systems biology, high-throughput screening, and tissue- and single-cell-resolution analyses to create vectors with improved properties. It leverages sequencing, artificial intelligence, and structural modeling to enable rational design of therapies. Headquartered in Waltham, Massachusetts, Affinia Therapeutics was founded in 2019 and targets diseases with significant unmet needs in muscle and CNS.

AviadoBio focuses on developing transformative gene therapies for devastating neurodegenerative disorders such as frontotemporal dementia and amyotrophic lateral sclerosis. The company's platform combines proprietary technologies with a deep understanding of the brain to deliver effective treatments.

Immuneel Therapeutics specializes in developing innovative cell and gene therapies for cancer treatment. The company focuses on CAR-T cell therapies, which are created by extracting blood cells from patients and genetically engineering healthy immune cells to specifically target malignant cells. This approach allows cancer patients to benefit from advanced cell-based immunotherapies that have the potential to transform their treatment outcomes.

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.

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.

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.

Verve Therapeutics is a biotechnology company developing genetic medicines for cardiovascular disease, aiming to transform treatment from chronic management to single-course gene editing therapies. It leverages human genetics analysis and gene-editing technology, with expertise in cardiovascular medicine, human genetics, gene editing, delivery technologies, drug development, and commercialization.

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 is a biotechnology company developing precision genetic medicines using its proprietary base editing technology. Its focus includes therapies for hematological and genetic diseases, with ongoing programs targeting sickle cell disease, alpha-1 antitrypsin deficiency, and other serious conditions.

Founded in 2018, Sana Biotechnology focuses on creating and delivering engineered cells as medicine for patients. The company aims to reprogram cells in the body or replace damaged ones to treat a broad range of diseases, with a pipeline including SC291, SC262, SC255, UP421, and other candidates across therapeutic areas like oncology, diabetes, autoimmune disorders, and CNS.

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.

Founded in 2014, Genomics develops algorithms and software solutions focused on cancer, microbes, and rare diseases. Its solutions empower governments, healthcare providers, pharmaceutical companies, clinicians, and researchers to improve diagnoses, treatment options, and drug development processes through accurate genomic data analysis.

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

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.

Founded in 2018, Sana Biotechnology focuses on creating and delivering engineered cells as medicine for patients. The company aims to reprogram cells in the body or replace damaged ones to treat a broad range of diseases, with a pipeline including SC291, SC262, SC255, UP421, and other candidates across therapeutic areas like oncology, diabetes, autoimmune disorders, and CNS.

Beam Therapeutics is a biotechnology company developing precision genetic medicines using its proprietary base editing technology. Its focus includes therapies for hematological and genetic diseases, with ongoing programs targeting sickle cell disease, alpha-1 antitrypsin deficiency, and other serious conditions.

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.

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.

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

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

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 develops cellular engineering solutions based on CRISPR technologies, offering a gene editing platform with applications in human therapeutics, disease modeling, genomics research, and agricultural biotechnology.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

Proteostasis Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule therapies that regulate the body's protein homeostasis network to treat cystic fibrosis and other genetic or degenerative diseases. It develops Proteostasis Regulators intended to restore proteostasis balance and address conditions such as cystic fibrosis, emphysema, diabetes, and neurodegenerative disorders. In CF, its lead candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. The company collaborates with the Cystic Fibrosis Foundation to research, develop, and commercialize CF-related products, and has licensing relationships with Genentech. It was founded in 2006, formerly known as Proteoguard, and was renamed Proteostasis Therapeutics in 2007, based in Boston. It was acquired by Yumanity Therapeutics in 2020 in a reverse merger.

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 is a clinical-stage biotechnology company developing gene therapy product candidates using its proprietary NAV Technology Platform. This platform delivers genes to cells to address genetic defects or produce therapeutic proteins. REGENXBIO's pipeline includes RGX-314 for wet age-related macular degeneration, RGX-121 and RGX-111 for mucopolysaccharidoses, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II disease, and RGX-501 for homozygous familial hypercholesterolemia. The company also licenses its platform to other biopharmaceutical companies.

Precision BioSciences, founded in 2006, is a biotechnology company headquartered in Durham, North Carolina. It specializes in genome editing, developing therapeutic products using its proprietary ARCUS technology. The company focuses on creating allogeneic CAR T immunotherapies to treat cancers like acute lymphoblastic leukemia and non-hodgkin lymphoma, as well as genetic disorders.

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 develops cellular engineering solutions based on CRISPR technologies, offering a gene editing platform with applications in human therapeutics, disease modeling, genomics research, and agricultural biotechnology.

REGENXBIO is a clinical-stage biotechnology company developing gene therapy product candidates using its proprietary NAV Technology Platform. This platform delivers genes to cells to address genetic defects or produce therapeutic proteins. REGENXBIO's pipeline includes RGX-314 for wet age-related macular degeneration, RGX-121 and RGX-111 for mucopolysaccharidoses, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II disease, and RGX-501 for homozygous familial hypercholesterolemia. The company also licenses its platform to other biopharmaceutical companies.

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.

Blueprint Medicines is a Cambridge, Massachusetts-based biopharmaceutical company that develops targeted, small-molecule therapies to treat genomically defined cancers, rare diseases, and related conditions. The company focuses on precision medicines that address molecular drivers and resistance mechanisms, advancing a pipeline that includes avapritinib for systemic mastocytosis and certain gastrointestinal stromal tumors, BLU-263, a KIT inhibitor for indolent systemic mastocytosis and other mast cell disorders, fisogatinib for advanced hepatocellular carcinoma, and pralsetinib, a RET inhibitor for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. It is also pursuing BLU-782, an ALK2 inhibitor in early clinical trials for fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with partner companies such as Clementia Pharmaceuticals, CStone Pharmaceuticals, Genentech, and Hoffmann-La Roche. Founded in 2008, the company operates in the United States and Europe and combines its Insights-to-Validation platform with a proprietary chemical library to discover and develop new therapeutic compounds and combination therapies in oncology, hematology, and related areas.

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

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

Blueprint Medicines is a Cambridge, Massachusetts-based biopharmaceutical company that develops targeted, small-molecule therapies to treat genomically defined cancers, rare diseases, and related conditions. The company focuses on precision medicines that address molecular drivers and resistance mechanisms, advancing a pipeline that includes avapritinib for systemic mastocytosis and certain gastrointestinal stromal tumors, BLU-263, a KIT inhibitor for indolent systemic mastocytosis and other mast cell disorders, fisogatinib for advanced hepatocellular carcinoma, and pralsetinib, a RET inhibitor for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. It is also pursuing BLU-782, an ALK2 inhibitor in early clinical trials for fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with partner companies such as Clementia Pharmaceuticals, CStone Pharmaceuticals, Genentech, and Hoffmann-La Roche. Founded in 2008, the company operates in the United States and Europe and combines its Insights-to-Validation platform with a proprietary chemical library to discover and develop new therapeutic compounds and combination therapies in oncology, hematology, and related areas.

FoldRx Pharmaceuticals, Inc. is engaged in the discovery and development of disease-modifying drug therapies targeting diseases caused by protein misfolding and amyloidosis. Based in Cambridge, Massachusetts, the company focuses on addressing conditions related to the accumulation of misfolded proteins, which can result from genetic mutations or defects in cellular quality control mechanisms. Its pipeline includes advanced clinical programs aimed at treating transthyretin-associated amyloidoses, specifically those affecting the nervous and cardiovascular systems. Additionally, FoldRx is exploring therapeutic options for Parkinson's disease and cystic fibrosis. Founded in 2003, the company aims to provide healthcare professionals with effective treatments for these complex conditions.

Proteostasis Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule therapies that regulate the body's protein homeostasis network to treat cystic fibrosis and other genetic or degenerative diseases. It develops Proteostasis Regulators intended to restore proteostasis balance and address conditions such as cystic fibrosis, emphysema, diabetes, and neurodegenerative disorders. In CF, its lead candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. The company collaborates with the Cystic Fibrosis Foundation to research, develop, and commercialize CF-related products, and has licensing relationships with Genentech. It was founded in 2006, formerly known as Proteoguard, and was renamed Proteostasis Therapeutics in 2007, based in Boston. It was acquired by Yumanity Therapeutics in 2020 in a reverse merger.

Bikam Pharmaceuticals is a drug discovery company based in Cambridge, Massachusetts, established in 2007. The company specializes in the development of novel therapeutics aimed at treating degenerative diseases, particularly retinal disorders. It focuses on creating small molecule, non-retinoid pharmacological chaperones designed to address issues related to misfolded proteins in the retina, which are implicated in serious conditions such as retinitis pigmentosa and dry age-related macular degeneration. Bikam Pharmaceuticals has developed an orally active pharmacological chaperone that effectively binds to misfolded rod opsin, a protein associated with retinitis pigmentosa, and facilitates its proper trafficking to the surface of rod cells, thereby aiming to mitigate the effects of this rare and incurable genetic disease that leads to blindness.

FoldRx Pharmaceuticals, Inc. is engaged in the discovery and development of disease-modifying drug therapies targeting diseases caused by protein misfolding and amyloidosis. Based in Cambridge, Massachusetts, the company focuses on addressing conditions related to the accumulation of misfolded proteins, which can result from genetic mutations or defects in cellular quality control mechanisms. Its pipeline includes advanced clinical programs aimed at treating transthyretin-associated amyloidoses, specifically those affecting the nervous and cardiovascular systems. Additionally, FoldRx is exploring therapeutic options for Parkinson's disease and cystic fibrosis. Founded in 2003, the company aims to provide healthcare professionals with effective treatments for these complex conditions.