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 Inc is a biotechnology company committed to delivering genetic therapies to address diseases by deploying gene editing technology, Prime Editing. The Prime Editing technology is a next-generation technology that can search and replace to restore normal genetic function in the genome and can treat a wide spectrum of diseases with high unmet medical needs and efficient and broad gene editing technology.
Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for various diseases, particularly those affecting muscle and the central nervous system (CNS). Founded in 2019 and based in Waltham, Massachusetts, the company specializes in creating rationally designed adeno-associated virus (AAV) vectors. By integrating synthetic and systems biology with high-throughput screening and advanced resolution techniques, Affinia Therapeutics aims to address significant unmet medical needs. The company’s innovative approach allows for the development of novel gene therapies with enhanced properties, targeting the treatment of devastating diseases and improving the lives of affected individuals.
Genomics PLC is focused on advancing the use of genomic data to transform healthcare. The company develops algorithms and software solutions that facilitate the analysis of large genomic and phenotypic datasets, aimed at understanding human biology and improving disease diagnosis and treatment. Its offerings serve a diverse range of clients, including governments, healthcare providers, pharmaceutical companies, and research laboratories. By providing tools that enhance the accuracy of genetic analysis, Genomics PLC helps de-risk the drug development process and enables clinicians to better identify individuals at risk for specific diseases. Founded in 2014 and based in Oxford, United Kingdom, the company aims to set standards in genomic data application, ultimately benefiting organizations and patients alike through innovative healthcare solutions.
Ensoma is expanding the reach of the curative power of genomic medicine by pioneering a next-generation in vivo approach using its Engenious™ vectors. Ensoma’s vectors are designed to deliver a diverse range of gene modification technologies without the need for stem cell collection or prior myeloablative conditioning (e.g., chemotherapy). As a result, Ensoma’s therapies can be delivered as a single injection in a diverse range of settings, including outpatient and settings where access to sophisticated healthcare systems may be limited.
Atalanta Therapeutics is a biotechnology company focused on developing innovative treatment options for neurodegenerative diseases through its proprietary RNA interference platform. The company aims to create therapeutics that target various conditions, including Huntington's disease by specifically targeting the HTT gene. By addressing the underlying causes of these diseases, Atalanta Therapeutics seeks to provide clinicians with effective strategies to halt disease progression and improve patient outcomes.
Verve Therapeutics is a biotechnology company focused on developing innovative therapies aimed at editing the adult human genome to provide lifelong protection against coronary artery disease, a leading global cause of mortality. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company employs gene-editing technology to create treatments that shift the management of cardiovascular diseases from chronic care to potentially curative interventions. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are recognized for their roles in regulating blood lipids, specifically low-density lipoprotein cholesterol. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance delivery technologies for its cardiovascular treatments.
Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for various diseases, particularly those affecting muscle and the central nervous system (CNS). Founded in 2019 and based in Waltham, Massachusetts, the company specializes in creating rationally designed adeno-associated virus (AAV) vectors. By integrating synthetic and systems biology with high-throughput screening and advanced resolution techniques, Affinia Therapeutics aims to address significant unmet medical needs. The company’s innovative approach allows for the development of novel gene therapies with enhanced properties, targeting the treatment of devastating diseases and improving the lives of affected individuals.
Adagene (Suzhou) Limited is a clinical-stage biotechnology company based in Suzhou, China, focused on antibody discovery and engineering. The company specializes in developing immuno-oncology antibodies that target novel epitopes, utilizing its proprietary Smart Antibody Technology. This technology enhances the success rates of antibody development, significantly shortens the time required to bring products to market, and lowers associated costs. Adagene's offerings extend beyond therapeutic applications, as its technology is also applicable in diagnostics and research, aiming to advance the field of immunotherapy and improve patient outcomes. Founded in 2011, Adagene continues to innovate in the biopharmaceutical sector.
Prime Medicine Inc is a biotechnology company committed to delivering genetic therapies to address diseases by deploying gene editing technology, Prime Editing. The Prime Editing technology is a next-generation technology that can search and replace to restore normal genetic function in the genome and can treat a wide spectrum of diseases with high unmet medical needs and efficient and broad gene editing technology.
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.
LunaPBC, Inc. operates a community-owned platform focused on health and DNA research. The company offers LunaDNA, a database that facilitates the sharing of health and genetic data for medical research. This platform supports health research initiatives by both non-profit and for-profit organizations, contributing to medical discoveries. LunaPBC operates as a Public Benefit Corporation, emphasizing community welfare by allowing individuals to share their health data, which in turn drives research and innovation. Additionally, members of the platform have the opportunity to share in the earnings derived from medical advancements, promoting a sense of shared ownership and benefit within the community. Founded in 2017 and based in San Diego, California, LunaPBC aims to advance genomic and medical research while prioritizing the interests of its contributors.
Verve Therapeutics is a biotechnology company focused on developing innovative therapies aimed at editing the adult human genome to provide lifelong protection against coronary artery disease, a leading global cause of mortality. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company employs gene-editing technology to create treatments that shift the management of cardiovascular diseases from chronic care to potentially curative interventions. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are recognized for their roles in regulating blood lipids, specifically low-density lipoprotein cholesterol. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance delivery technologies for its cardiovascular treatments.
Embark offers dog DNA testing that ends preventable diseases in dogs. It was founded by top experts in genomics, computational biology, and software design to sell an industry-leading dog DNA test. They use a clinical-grade CLIA-certified SNP chip to provide the most accurate and comprehensive results (200,000+ genetic markers) available on the market. Their data contributes to proprietary genetics research that will help dog owners optimize nutrition, exercise, allergies, and prevent a variety of heritable conditions. Their technological advantage, intellectual capital, and research-focus enable them to push canine health toward our shared vision of preventative veterinary care.
Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing precision genetic medicines using its innovative base editing technology. This technology allows for targeted alterations of single bases in the genome without creating double-stranded breaks in DNA, representing a potential advancement in genetic therapies. The company's pipeline includes programs aimed at treating serious diseases such as sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, and pediatric acute myeloid leukemia, as well as conditions like alpha-1 antitrypsin deficiency and certain central nervous system disorders. Beam Therapeutics is committed to providing life-long cures through its various approaches, which include gene correction, gene modification, gene activation, gene silencing, and multiplex editing. Founded in 2017, Beam continues to build collaborations to enhance its research and development efforts.
Singular Genomics is a biotechnology company based in La Jolla, California, founded in 2016. It specializes in developing advanced gene sequencing technologies utilizing fluorescing molecular tags and novel next-generation sequencing and multiomics approaches. The company aims to empower researchers and clinicians through its innovative products, which include the G4 Sequencing Platform for next-generation sequencing and the PX Integrated Solution for single-cell analysis, spatial analysis, and proteomics. Singular Genomics is strategically located within a prominent biotech hub, adjacent to esteemed institutions such as the Salk Institute and UC San Diego, fostering collaboration and innovation in the rapidly growing genomics field. Its product offerings cater to a wide range of applications, including basic biology, oncology, and immunology, positioning the company as a key player in the life sciences sector.
Genomics PLC is focused on advancing the use of genomic data to transform healthcare. The company develops algorithms and software solutions that facilitate the analysis of large genomic and phenotypic datasets, aimed at understanding human biology and improving disease diagnosis and treatment. Its offerings serve a diverse range of clients, including governments, healthcare providers, pharmaceutical companies, and research laboratories. By providing tools that enhance the accuracy of genetic analysis, Genomics PLC helps de-risk the drug development process and enables clinicians to better identify individuals at risk for specific diseases. Founded in 2014 and based in Oxford, United Kingdom, the company aims to set standards in genomic data application, ultimately benefiting organizations and patients alike through innovative healthcare solutions.
Avidity Biosciences is a biopharmaceutical company specializing in the development of oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disorder. The company's development pipeline also includes programs focused on treating various muscle diseases, such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Additionally, Avidity is exploring therapeutic applications for immune and other cell types, leveraging its proprietary AOC platform to reach previously undruggable tissues and effectively target the genetic underpinnings of these conditions. Founded in 2012, Avidity Biosciences is headquartered in La Jolla, California.
Nebula Genomics, founded in 2016 and based in San Francisco, California, is a company focused on human genome sequencing and health data. It aims to create a comprehensive and trusted marketplace for genomic and health data, facilitating access for consumers, researchers, and the medical community. Utilizing blockchain technology, Nebula Genomics empowers individuals to control their genomic data while receiving compensation for its use. The marketplace aggregates extensive genetic information, which researchers can analyze to enhance drug development and streamline clinical trials, ultimately contributing to personalized medicine. The company's innovative DNA sequencing technology allows for the examination of all genes, regulatory regions, the Y chromosome, and mitochondrial DNA, enabling clients to explore genetic variants comprehensively. Nebula Genomics was co-founded by Harvard genomics pioneer George Church and has received backing from notable investors.
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.
Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing precision genetic medicines using its innovative base editing technology. This technology allows for targeted alterations of single bases in the genome without creating double-stranded breaks in DNA, representing a potential advancement in genetic therapies. The company's pipeline includes programs aimed at treating serious diseases such as sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, and pediatric acute myeloid leukemia, as well as conditions like alpha-1 antitrypsin deficiency and certain central nervous system disorders. Beam Therapeutics is committed to providing life-long cures through its various approaches, which include gene correction, gene modification, gene activation, gene silencing, and multiplex editing. Founded in 2017, Beam continues to build collaborations to enhance its research and development efforts.
Biopalette is a operator of a platform intended to offer gene-editing techniques. Bio Palette plans to develop our business in three fields, classified based on the type of cells targeted for genome editing.
Nebula Genomics, founded in 2016 and based in San Francisco, California, is a company focused on human genome sequencing and health data. It aims to create a comprehensive and trusted marketplace for genomic and health data, facilitating access for consumers, researchers, and the medical community. Utilizing blockchain technology, Nebula Genomics empowers individuals to control their genomic data while receiving compensation for its use. The marketplace aggregates extensive genetic information, which researchers can analyze to enhance drug development and streamline clinical trials, ultimately contributing to personalized medicine. The company's innovative DNA sequencing technology allows for the examination of all genes, regulatory regions, the Y chromosome, and mitochondrial DNA, enabling clients to explore genetic variants comprehensively. Nebula Genomics was co-founded by Harvard genomics pioneer George Church and has received backing from notable investors.
Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. Operating in the United Kingdom, European Union, and the United States, Orchard employs an autologous ex vivo gene therapy approach to transform patients’ hematopoietic stem cells into gene-modified products for treatment through a single administration. Its portfolio includes Strimvelis, the first approved gene therapy for adenosine deaminase-severe combined immunodeficiency (ADA-SCID), along with several products in clinical development targeting metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has a robust preclinical pipeline addressing various mucopolysaccharidosis types. Orchard collaborates with leading institutions in gene therapy, enhancing its research capabilities and clinical programs. Founded in 2015, Orchard Therapeutics continues to demonstrate a commitment to transforming the lives of patients through its advanced therapies.
PlasmaGen Biosciences Pvt. Ltd. is an Indian bio-pharmaceutical company based in Bengaluru, specializing in the manufacture of plasma-derived products and therapies. Established in 2010, the company develops a range of plasma protein therapies, including PlasmaGlob and AlbuMax, which are Normal Human Immunoglobulin formulations for intravenous use. Their product portfolio also includes PlasmaRho-D I.V. for Rho (D) immunization, PlasmaRAB for rabies post-exposure prophylaxis, and PlasmaHep for various hepatitis B-related indications. Additionally, PlasmaGen offers VerBumin for conditions such as hypovolemic shock and plasma exchange dialysis. The company serves a diverse clientele, including clinicians, medical practitioners, corporate hospitals, and government institutions, positioning itself as a key provider of plasma products across various medical fields, including hematology, oncology, and immunology.
Biopalette is a operator of a platform intended to offer gene-editing techniques. Bio Palette plans to develop our business in three fields, classified based on the type of cells targeted for genome editing.
Biopalette is a operator of a platform intended to offer gene-editing techniques. Bio Palette plans to develop our business in three fields, classified based on the type of cells targeted for genome editing.
Core Diagnostics Private Limited is a clinical laboratory based in Gurugram, India, established in 2012. The company specializes in next-generation diagnostics aimed at disease stratification and therapy selection. By employing advanced testing techniques, Core Diagnostics seeks to provide comprehensive diagnostic solutions and become a leading provider of high-end diagnostic services in the region.
Caribou Biosciences, Inc. is a biotechnology company based in Berkeley, California, specializing in cellular engineering and genome analysis through CRISPR-Cas9 technology. Founded in 2011, the company provides a gene editing platform that allows for targeted modifications in the genome. Its applications span various sectors, including human and animal therapeutics, agricultural biotechnology, and industrial biotechnology. Caribou's solutions facilitate the development of new disease models, enhance traits in plants, and support functional genomic screens. The company aims to advance both basic and applied biological research, leveraging its transformative capabilities to impact therapeutic development and bioproduction.
Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. Operating in the United Kingdom, European Union, and the United States, Orchard employs an autologous ex vivo gene therapy approach to transform patients’ hematopoietic stem cells into gene-modified products for treatment through a single administration. Its portfolio includes Strimvelis, the first approved gene therapy for adenosine deaminase-severe combined immunodeficiency (ADA-SCID), along with several products in clinical development targeting metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has a robust preclinical pipeline addressing various mucopolysaccharidosis types. Orchard collaborates with leading institutions in gene therapy, enhancing its research capabilities and clinical programs. Founded in 2015, Orchard Therapeutics continues to demonstrate a commitment to transforming the lives of patients through its advanced therapies.
Adagene (Suzhou) Limited is a clinical-stage biotechnology company based in Suzhou, China, focused on antibody discovery and engineering. The company specializes in developing immuno-oncology antibodies that target novel epitopes, utilizing its proprietary Smart Antibody Technology. This technology enhances the success rates of antibody development, significantly shortens the time required to bring products to market, and lowers associated costs. Adagene's offerings extend beyond therapeutic applications, as its technology is also applicable in diagnostics and research, aiming to advance the field of immunotherapy and improve patient outcomes. Founded in 2011, Adagene continues to innovate in the biopharmaceutical sector.
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 Inc a biotechnology company focused on developing precision therapies for genetically defined diseases. It designs rational precision therapies that treat the underlying cause of disease and improve the lives of patients. Its program CGT9486, is a selective tyrosine kinase inhibitor designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. In the vast majority of cases, KIT D816V is responsible for driving Systemic Mastocytosis (SM), a serious disease caused by the unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancermore
REGENXBIO Inc. is a clinical-stage biotechnology company focused on developing gene therapy product candidates that utilize its proprietary NAV Technology Platform, which is based on adeno-associated virus (AAV) gene delivery. This platform aims to address genetic defects and enable cells to produce therapeutic proteins or antibodies to combat various diseases. The company's lead candidate, RGX-314, is currently undergoing Phase I/IIa clinical trials for wet age-related macular degeneration. Other notable product candidates include RGX-121 for mucopolysaccharidosis type II, RGX-111 for mucopolysaccharidosis type I, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia, all in various stages of clinical trials. Additionally, REGENXBIO licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and has partnered 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.
Precision BioSciences, Inc. is a biotechnology company that specializes in genome editing, utilizing its proprietary ARCUS platform to develop therapeutic and food products. The Therapeutic segment focuses on creating allogeneic CAR T immunotherapies aimed at treating various cancers, including acute lymphoblastic leukemia and multiple myeloma, with several candidates currently in clinical trials. Notable product candidates include PBCAR0191, targeting CD19, and PBCAR269A, targeting BCMA. Additionally, the company is engaged in in vivo gene correction and has collaborations for developing treatments for chronic Hepatitis B. The Food segment aims to innovate in food and nutrition products. Founded in 2006 and headquartered in Durham, North Carolina, Precision BioSciences is committed to translating advanced genome editing technology into impactful medical and agricultural solutions.
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, specializing in cellular engineering and genome analysis through CRISPR-Cas9 technology. Founded in 2011, the company provides a gene editing platform that allows for targeted modifications in the genome. Its applications span various sectors, including human and animal therapeutics, agricultural biotechnology, and industrial biotechnology. Caribou's solutions facilitate the development of new disease models, enhance traits in plants, and support functional genomic screens. The company aims to advance both basic and applied biological research, leveraging its transformative capabilities to impact therapeutic development and bioproduction.
REGENXBIO Inc. is a clinical-stage biotechnology company focused on developing gene therapy product candidates that utilize its proprietary NAV Technology Platform, which is based on adeno-associated virus (AAV) gene delivery. This platform aims to address genetic defects and enable cells to produce therapeutic proteins or antibodies to combat various diseases. The company's lead candidate, RGX-314, is currently undergoing Phase I/IIa clinical trials for wet age-related macular degeneration. Other notable product candidates include RGX-121 for mucopolysaccharidosis type II, RGX-111 for mucopolysaccharidosis type I, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia, all in various stages of clinical trials. Additionally, REGENXBIO licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and has partnered 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.
Adagene (Suzhou) Limited is a clinical-stage biotechnology company based in Suzhou, China, focused on antibody discovery and engineering. The company specializes in developing immuno-oncology antibodies that target novel epitopes, utilizing its proprietary Smart Antibody Technology. This technology enhances the success rates of antibody development, significantly shortens the time required to bring products to market, and lowers associated costs. Adagene's offerings extend beyond therapeutic applications, as its technology is also applicable in diagnostics and research, aiming to advance the field of immunotherapy and improve patient outcomes. Founded in 2011, Adagene continues to innovate in the biopharmaceutical sector.
Cogent Biosciences Inc a biotechnology company focused on developing precision therapies for genetically defined diseases. It designs rational precision therapies that treat the underlying cause of disease and improve the lives of patients. Its program CGT9486, is a selective tyrosine kinase inhibitor designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. In the vast majority of cases, KIT D816V is responsible for driving Systemic Mastocytosis (SM), a serious disease caused by the unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancermore
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.
Ultragenyx Pharmaceutical is a biopharmaceutical company based in the United States that focuses on developing therapeutics for rare and ultra-rare diseases, particularly serious genetic conditions. The company identifies, acquires, and commercializes novel treatments, leveraging existing scientific knowledge to address unmet medical needs in this challenging area. Its portfolio includes several medicines such as Crysvita, which treats X-linked hypophosphatemia in both adults and pediatric patients, Dojolvi, and Mepsevii, indicated for Mucopolysaccharidosis VII. Ultragenyx aims to create impactful therapeutics by employing effective development strategies tailored to the complexities of rare diseases.
Ultragenyx Pharmaceutical is a biopharmaceutical company based in the United States that focuses on developing therapeutics for rare and ultra-rare diseases, particularly serious genetic conditions. The company identifies, acquires, and commercializes novel treatments, leveraging existing scientific knowledge to address unmet medical needs in this challenging area. Its portfolio includes several medicines such as Crysvita, which treats X-linked hypophosphatemia in both adults and pediatric patients, Dojolvi, and Mepsevii, indicated for Mucopolysaccharidosis VII. Ultragenyx aims to create impactful therapeutics by employing effective development strategies tailored to the complexities of rare diseases.
FoldRx Pharmaceuticals, Inc. is a biotechnology company focused on discovering and developing disease-modifying drug therapies for conditions related to protein misfolding and amyloidosis. The company’s pipeline includes advanced clinical programs targeting genetic neurological and cardiovascular disorders, specifically transthyretin-associated amyloidoses that affect both polyneuropathy and cardiomyopathy. Additionally, FoldRx is engaged in early-stage discovery efforts aimed at addressing diseases such as Parkinson's and cystic fibrosis. Established in 2003 and headquartered in Cambridge, Massachusetts, the company aims to provide effective treatments for the accumulation of misfolded proteins caused by genetic mutations and deficiencies in cellular quality control mechanisms.
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.
FoldRx Pharmaceuticals, Inc. is a biotechnology company focused on discovering and developing disease-modifying drug therapies for conditions related to protein misfolding and amyloidosis. The company’s pipeline includes advanced clinical programs targeting genetic neurological and cardiovascular disorders, specifically transthyretin-associated amyloidoses that affect both polyneuropathy and cardiomyopathy. Additionally, FoldRx is engaged in early-stage discovery efforts aimed at addressing diseases such as Parkinson's and cystic fibrosis. Established in 2003 and headquartered in Cambridge, Massachusetts, the company aims to provide effective treatments for the accumulation of misfolded proteins caused by genetic mutations and deficiencies in cellular quality control mechanisms.