Castle Creek Biosciences, Inc. is a gene therapy company based in Exton, Pennsylvania, that specializes in developing personalized gene therapies for rare and serious skin and connective tissue disorders. Founded in 2015, the company utilizes a fibroblast technology platform that extracts fibroblast cells from a patient's own skin. This approach allows for the creation of localized therapies tailored to the unique biology of each individual. By addressing the underlying causes of these conditions, Castle Creek aims to improve the lives of patients facing high unmet medical needs in underserved areas.
SalioGen Therapeutics advances in curative genetic therapies using its Exact DNA Integration Technology (EDIT) platform, a mammal-derived genome engineering technology. It is focused on providing durable, safe, and affordable non-viral gene therapies to more patients with inherited diseases. Looking ahead, SalioGen will also explore the EDIT platform’s capabilities to streamline cell therapy, cell engineering, and biologics manufacturing to help an even broader population of patients in need.
DNA Script SAS, founded in 2014 and based in Paris, France, specializes in synthetic biology tools and innovative DNA synthesis technology. The company has developed a groundbreaking enzymatic technology that powers the SYNTAX, the world's first benchtop DNA printer. This device allows laboratories to produce synthetic oligonucleotides on-site, significantly enhancing workflow control and expediting access to results. By employing an efficient biochemical process for nucleic acid synthesis, DNA Script enables researchers to rapidly generate essential genomic materials, such as oligonucleotides, which are crucial for genomics and molecular biology applications. Through its advancements, DNA Script aims to facilitate breakthroughs in life sciences and human health.
Generate Biomedicines, Inc. specializes in developing a generative biology platform aimed at inventing novel therapeutic proteins, including antibodies, peptides, enzymes, and other related compounds. Utilizing advanced machine learning techniques, the platform analyzes the genetic code that determines protein function, allowing for the creation of new protein sequences optimized for therapeutic applications. This innovative approach enables the company to generate proteins that interact specifically and effectively with targeted therapeutic needs, facilitating the development of new drugs across various protein modalities. Founded in 2018 and located in Cambridge, Massachusetts, Generate Biomedicines was previously known as Generate Biologics, Inc. until its name change in March 2020.
DNA Script SAS, founded in 2014 and based in Paris, France, specializes in synthetic biology tools and innovative DNA synthesis technology. The company has developed a groundbreaking enzymatic technology that powers the SYNTAX, the world's first benchtop DNA printer. This device allows laboratories to produce synthetic oligonucleotides on-site, significantly enhancing workflow control and expediting access to results. By employing an efficient biochemical process for nucleic acid synthesis, DNA Script enables researchers to rapidly generate essential genomic materials, such as oligonucleotides, which are crucial for genomics and molecular biology applications. Through its advancements, DNA Script aims to facilitate breakthroughs in life sciences and human health.
Skyhawk Therapeutics is focused on the discovery and development of small molecule therapeutics aimed at correcting RNA expression. The company utilizes proprietary technology to design small molecules that specifically target binding pocket regions on RNA, ensuring both sequence and structural specificity during critical phases of RNA splicing. This innovative approach enables the development of therapies for various conditions, including cancer, neurological disorders, and rare diseases, particularly those associated with RNA mis-splicing known as exon skipping. Founded in 2015 and headquartered in Waltham, Massachusetts, Skyhawk Therapeutics was previously known as Rare Genetix before rebranding in 2018.
Deep Genomics Inc. is an artificial intelligence therapeutics company that focuses on developing individualized genetic medicines. Founded in 2014 and based in Toronto, Canada, the company utilizes advanced AI systems to enhance various stages of drug discovery and development, including target identification, lead optimization, toxicity evaluation, and trial design. Deep Genomics specializes in creating oligonucleotide therapies that address the genetic causes of diseases at the RNA and DNA levels. Its innovative AI-powered platform analyzes RNA biology to uncover new therapeutic targets and mechanisms that traditional methods cannot access, enabling the development of treatments for rare metabolic, ophthalmologic, and neurodegenerative disorders. The company recruits top talent in genomics, drug development, and AI to drive its mission of revolutionizing drug discovery.
Element Biosciences, Inc. develops genetic analysis tools for the research and diagnostic markets. The company provides a disruptive DNA sequencing technology and a sequencing platform, such as surface chemistry, sequencing chemistry, detection, and data analysis. The company was incorporated in 2017 and is based in San Diego, California.
23andMe, Inc. is a consumer genetics company that provides DNA analysis services through home-based saliva collection kits, enabling users to explore their ancestry, inherited traits, and health insights. Founded in 2006 and headquartered in Sunnyvale, California, the company offers various services, including Health + Ancestry and Ancestry + Traits, which provide insights into a person's genetic background and over 30 trait reports. 23andMe aims to help individuals understand the implications of their genetic information while also catering to researchers by offering categorized and searchable data. The company was co-founded by Linda Avey and Anne Wojcicki and has positioned itself as a significant player in the growing biotechnology and healthcare sectors.
Inscripta, Inc. is a gene editing technology company focused on developing advanced CRISPR enzymes, specifically MADzymes, for precision gene editing. Founded in 2015 and headquartered in Boulder, Colorado, with additional offices in Pleasanton and San Diego, Inscripta aims to revolutionize genome engineering through its innovative benchtop platform. This platform integrates an instrument, consumables, software, and assays to facilitate fully automated and scalable editing of single cells, enabling researchers to conduct massively parallel experiments with unprecedented efficiency. By offering tools that simplify the editing process and making its MAD7 CRISPR nuclease available for research, Inscripta seeks to empower scientists in overcoming existing technical and licensing barriers, fostering a new era of biological discovery.
Omega Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, that focuses on developing genomic medicines aimed at curing diseases. Founded in 2016, the company utilizes its proprietary epigenomic programming platform to create Omega Epigenomic Controllers. These innovative controllers are designed to selectively modulate the activity of the human genome, allowing for precise tuning of gene expression to achieve therapeutic effects. By targeting specific cell types, Omega Therapeutics aims to transform human medicine through the direct control of genomic functions, offering potential solutions for a variety of diseases.
Gemini Therapeutics, Inc. is a precision medicine company established in 2015 and based in Cambridge, Massachusetts. The company specializes in developing therapies for patients with dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini's approach focuses on addressing genetically-defined conditions by creating therapeutic candidates that target specific molecular abnormalities prevalent in patients with significant clinical needs. Their diverse pipeline features a range of innovative treatment modalities, including monoclonal antibodies, recombinant proteins, and gene therapies. The company has secured funding from prominent life science investors and collaborates with academic institutions globally to advance its mission of delivering potentially first-in-class therapeutics.
Vera Therapeutics is engaged in the development of innovative gene editing solutions aimed at curing genetic diseases, particularly sickle cell disease and cystic fibrosis. The company utilizes a proprietary triplex gene editing platform to create a pipeline of therapeutic products targeting high-penetrance disease genes. Founded in 2016 and based in South San Francisco, California, Vera Therapeutics is dedicated to alleviating and potentially curing genetic disorders in affected patients.
Taysha Gene Therapies is a company focused on developing adeno-associated virus (AAV) based gene therapies aimed at treating monogenic diseases of the central nervous system. Founded in 2019 and based in Dallas, Texas, the company is advancing several therapeutic candidates, including TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies collaborates with The University of Texas Southwestern Medical Center to enhance the development and commercialization of its innovative treatments, with a mission to eradicate severe and life-threatening CNS diseases through curative gene therapies. The company's expertise in drug development and its partnership with a leading gene therapy program enable it to effectively progress its pipeline of potential therapies.
Castle Creek Biosciences, Inc. is a gene therapy company based in Exton, Pennsylvania, that specializes in developing personalized gene therapies for rare and serious skin and connective tissue disorders. Founded in 2015, the company utilizes a fibroblast technology platform that extracts fibroblast cells from a patient's own skin. This approach allows for the creation of localized therapies tailored to the unique biology of each individual. By addressing the underlying causes of these conditions, Castle Creek aims to improve the lives of patients facing high unmet medical needs in underserved areas.
Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing gene therapies aimed at treating both rare and prevalent diseases. Established in 2016, the company focuses on conditions affecting the liver, retina, skeletal muscle, central nervous system, and oncology. Generation Bio employs an innovative non-viral platform that includes a proprietary high-capacity DNA construct known as closed-ended DNA (ceDNA), a cell-targeted lipid nanoparticle delivery system (ctLNP), and a scalable, capsid-free manufacturing process. This approach allows for the targeted delivery of genetic materials, including large and multiple genes across various tissues, and enables redosable treatments tailored to individual patient needs. By enhancing access to therapies for rare diseases and addressing more common conditions, Generation Bio aims to provide durable and sustainable treatment options for patients throughout their lives.
Element Biosciences, Inc. develops genetic analysis tools for the research and diagnostic markets. The company provides a disruptive DNA sequencing technology and a sequencing platform, such as surface chemistry, sequencing chemistry, detection, and data analysis. The company was incorporated in 2017 and is based in San Diego, California.
BioNTech is a biotechnology company focused on developing and commercializing immunotherapies for cancer and infectious diseases. Founded in 2008 and headquartered in Mainz, Germany, the company has pioneered innovative therapies through advanced computational discovery and drug development platforms. BioNTech's oncology pipeline includes various neoantigen-targeted therapies, such as NEO-PV-01 for advanced non-small cell lung cancer and multiple FixVac product candidates for different cancers, including melanoma, prostate cancer, and HPV-positive head and neck cancers. The company also explores mRNA vaccine candidates for infectious diseases and maintains collaborations with prominent pharmaceutical firms. BioNTech Cell & Gene Therapies GmbH, a subsidiary, specializes in developing T cell immunotherapies for cancer treatment.
10X Genomics is a life science technology company that specializes in developing and selling instruments, consumables, and software for analyzing biological systems. Headquartered in Pleasanton, California, the company offers a range of products, including chromium instruments, microfluidic chips, reagents, and other consumables. Its advanced single-cell solutions allow researchers to measure gene expression, immune profiling, epigenetics, and cellular heterogeneity, providing insights into the physical organization of DNA and gene activity on a cell-by-cell basis. Additionally, the Visium spatial gene expression solution enables the measurement of spatial gene expression patterns across tissue samples. 10X Genomics serves a diverse clientele, including academic institutions, government agencies, and biopharmaceutical companies, and has a presence in North America, Europe, the Middle East, Africa, China, and the Asia Pacific. The company was founded in 2012 and rebranded from 10X Technologies, Inc. in November 2014.
Castle Creek Biosciences, Inc. is a gene therapy company based in Exton, Pennsylvania, that specializes in developing personalized gene therapies for rare and serious skin and connective tissue disorders. Founded in 2015, the company utilizes a fibroblast technology platform that extracts fibroblast cells from a patient's own skin. This approach allows for the creation of localized therapies tailored to the unique biology of each individual. By addressing the underlying causes of these conditions, Castle Creek aims to improve the lives of patients facing high unmet medical needs in underserved areas.
Allogene Therapeutics is a clinical-stage biotechnology company focused on immuno-oncology, particularly the development of genetically engineered allogeneic T cell therapies for cancer treatment. The company is advancing a pipeline of allogeneic T cell product candidates that leverage validated gene editing and proprietary cell manufacturing technologies. Key offerings include UCART19, which targets relapsed or refractory acute lymphoblastic leukemia, alongside several preclinical allogeneic CAR T therapies aimed at various malignancies and autoimmune diseases. By using T cells derived from healthy donors, Allogene Therapeutics aims to provide off-the-shelf treatment options that enhance patient eligibility and scalability. The company seeks to redefine CAR T therapy through its innovative approaches, addressing conditions like lymphoma, leukemia, and solid tumors.
Allogene Therapeutics is a clinical-stage biotechnology company focused on immuno-oncology, particularly the development of genetically engineered allogeneic T cell therapies for cancer treatment. The company is advancing a pipeline of allogeneic T cell product candidates that leverage validated gene editing and proprietary cell manufacturing technologies. Key offerings include UCART19, which targets relapsed or refractory acute lymphoblastic leukemia, alongside several preclinical allogeneic CAR T therapies aimed at various malignancies and autoimmune diseases. By using T cells derived from healthy donors, Allogene Therapeutics aims to provide off-the-shelf treatment options that enhance patient eligibility and scalability. The company seeks to redefine CAR T therapy through its innovative approaches, addressing conditions like lymphoma, leukemia, and solid tumors.
10X Genomics is a life science technology company that specializes in developing and selling instruments, consumables, and software for analyzing biological systems. Headquartered in Pleasanton, California, the company offers a range of products, including chromium instruments, microfluidic chips, reagents, and other consumables. Its advanced single-cell solutions allow researchers to measure gene expression, immune profiling, epigenetics, and cellular heterogeneity, providing insights into the physical organization of DNA and gene activity on a cell-by-cell basis. Additionally, the Visium spatial gene expression solution enables the measurement of spatial gene expression patterns across tissue samples. 10X Genomics serves a diverse clientele, including academic institutions, government agencies, and biopharmaceutical companies, and has a presence in North America, Europe, the Middle East, Africa, China, and the Asia Pacific. The company was founded in 2012 and rebranded from 10X Technologies, Inc. in November 2014.
Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing gene therapies aimed at treating both rare and prevalent diseases. Established in 2016, the company focuses on conditions affecting the liver, retina, skeletal muscle, central nervous system, and oncology. Generation Bio employs an innovative non-viral platform that includes a proprietary high-capacity DNA construct known as closed-ended DNA (ceDNA), a cell-targeted lipid nanoparticle delivery system (ctLNP), and a scalable, capsid-free manufacturing process. This approach allows for the targeted delivery of genetic materials, including large and multiple genes across various tissues, and enables redosable treatments tailored to individual patient needs. By enhancing access to therapies for rare diseases and addressing more common conditions, Generation Bio aims to provide durable and sustainable treatment options for patients throughout their lives.
BioNTech is a biotechnology company focused on developing and commercializing immunotherapies for cancer and infectious diseases. Founded in 2008 and headquartered in Mainz, Germany, the company has pioneered innovative therapies through advanced computational discovery and drug development platforms. BioNTech's oncology pipeline includes various neoantigen-targeted therapies, such as NEO-PV-01 for advanced non-small cell lung cancer and multiple FixVac product candidates for different cancers, including melanoma, prostate cancer, and HPV-positive head and neck cancers. The company also explores mRNA vaccine candidates for infectious diseases and maintains collaborations with prominent pharmaceutical firms. BioNTech Cell & Gene Therapies GmbH, a subsidiary, specializes in developing T cell immunotherapies for cancer treatment.
Translate Bio is a clinical-stage biotechnology company based in Lexington, Massachusetts, that focuses on developing messenger RNA (mRNA) therapeutics aimed at treating diseases linked to protein or gene dysfunction. Since its founding in 2011, the company has been working on innovative treatments, including MRT5005, which is currently undergoing Phase I/II clinical trials for cystic fibrosis, and MRT5201, intended for the treatment of ornithine transcarbamylase deficiency. Translate Bio aims to create transformative medicines that address unmet medical needs in rare diseases.
Codiak is a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics, a new class of medicines with the potential to transform the treatment of a wide spectrum of diseases with high unmet medical need. By leveraging the biology of exosomes as natural intercellular transfer mechanisms, Codiak has developed its proprietary engEx Platform to expand upon the innate properties of exosomes to design, engineer and manufacture novel exosome therapeutic candidates. Codiak has utilized its engEx Platform to generate a deep pipeline of engineered exosomes aimed at treating a broad range of disease areas, spanning oncology, neuro-oncology, neurology, neuromuscular disease and infectious disease.
23andMe, Inc. is a consumer genetics company that provides DNA analysis services through home-based saliva collection kits, enabling users to explore their ancestry, inherited traits, and health insights. Founded in 2006 and headquartered in Sunnyvale, California, the company offers various services, including Health + Ancestry and Ancestry + Traits, which provide insights into a person's genetic background and over 30 trait reports. 23andMe aims to help individuals understand the implications of their genetic information while also catering to researchers by offering categorized and searchable data. The company was co-founded by Linda Avey and Anne Wojcicki and has positioned itself as a significant player in the growing biotechnology and healthcare sectors.
Homology Medicines, Inc. is a genetic medicines company based in Bedford, Massachusetts, focused on improving the lives of patients with rare genetic diseases. The company employs a proprietary platform that utilizes human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to deliver genetic therapies in vivo, either through gene therapy or nuclease-free gene editing. This innovative approach allows for targeted treatment of various disease-relevant tissues, including the liver and central nervous system, through a single injection. Homology's lead product candidate, HMI-102, is currently in a Phase 1/2 clinical trial for treating phenylketonuria (PKU) in adults, while HMI-103 targets pediatric PKU patients, and HMI-202 is being developed for metachromatic leukodystrophy. Founded in 2015, Homology Medicines aims to address significant unmet medical needs by targeting the underlying causes of genetic disorders.
Translate Bio is a clinical-stage biotechnology company based in Lexington, Massachusetts, that focuses on developing messenger RNA (mRNA) therapeutics aimed at treating diseases linked to protein or gene dysfunction. Since its founding in 2011, the company has been working on innovative treatments, including MRT5005, which is currently undergoing Phase I/II clinical trials for cystic fibrosis, and MRT5201, intended for the treatment of ornithine transcarbamylase deficiency. Translate Bio aims to create transformative medicines that address unmet medical needs in rare diseases.
Castle Creek Biosciences, Inc. is a gene therapy company based in Exton, Pennsylvania, that specializes in developing personalized gene therapies for rare and serious skin and connective tissue disorders. Founded in 2015, the company utilizes a fibroblast technology platform that extracts fibroblast cells from a patient's own skin. This approach allows for the creation of localized therapies tailored to the unique biology of each individual. By addressing the underlying causes of these conditions, Castle Creek aims to improve the lives of patients facing high unmet medical needs in underserved areas.
10X Genomics is a life science technology company that specializes in developing and selling instruments, consumables, and software for analyzing biological systems. Headquartered in Pleasanton, California, the company offers a range of products, including chromium instruments, microfluidic chips, reagents, and other consumables. Its advanced single-cell solutions allow researchers to measure gene expression, immune profiling, epigenetics, and cellular heterogeneity, providing insights into the physical organization of DNA and gene activity on a cell-by-cell basis. Additionally, the Visium spatial gene expression solution enables the measurement of spatial gene expression patterns across tissue samples. 10X Genomics serves a diverse clientele, including academic institutions, government agencies, and biopharmaceutical companies, and has a presence in North America, Europe, the Middle East, Africa, China, and the Asia Pacific. The company was founded in 2012 and rebranded from 10X Technologies, Inc. in November 2014.
Codiak is a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics, a new class of medicines with the potential to transform the treatment of a wide spectrum of diseases with high unmet medical need. By leveraging the biology of exosomes as natural intercellular transfer mechanisms, Codiak has developed its proprietary engEx Platform to expand upon the innate properties of exosomes to design, engineer and manufacture novel exosome therapeutic candidates. Codiak has utilized its engEx Platform to generate a deep pipeline of engineered exosomes aimed at treating a broad range of disease areas, spanning oncology, neuro-oncology, neurology, neuromuscular disease and infectious disease.
Twist Bioscience Corporation, a synthetic biology company, manufactures and sells synthetic DNA-based products. The company’s DNA synthesis platform enables the manufacturing of synthetic DNA by writing DNA on a silicon chip. It offers synthetic DNA-based products, including synthetic genes, tools for sample preparation, antibody libraries for drug discovery and development, and DNA as a digital data storage medium. The company has a collaboration agreement with ImmunoPrecise Antibodies Ltd. Twist Bioscience Corporation was founded in 2013 and is headquartered in South San Francisco, California.
Twist Bioscience Corporation, a synthetic biology company, manufactures and sells synthetic DNA-based products. The company’s DNA synthesis platform enables the manufacturing of synthetic DNA by writing DNA on a silicon chip. It offers synthetic DNA-based products, including synthetic genes, tools for sample preparation, antibody libraries for drug discovery and development, and DNA as a digital data storage medium. The company has a collaboration agreement with ImmunoPrecise Antibodies Ltd. Twist Bioscience Corporation was founded in 2013 and is headquartered in South San Francisco, California.
Syros Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead candidates, SY-1425, a selective retinoic acid receptor alpha agonist currently undergoing Phase II trials for specific patients with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor in Phase I trials for select advanced solid tumors. Syros employs a proprietary platform that integrates assay technologies, bioinformatics, and biological insights to map gene regulatory circuits and modulate gene expression, positioning itself as a leader in gene control research. Additionally, Syros has established collaborations, including a target discovery agreement with Incyte Corporation focused on myeloproliferative neoplasms, and a licensing agreement with TMRC Co. Ltd. for tamibarotene's development and commercialization. Founded in 2011, Syros Pharmaceuticals aims to address significant unmet medical needs in oncology and beyond.
Codiak is a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics, a new class of medicines with the potential to transform the treatment of a wide spectrum of diseases with high unmet medical need. By leveraging the biology of exosomes as natural intercellular transfer mechanisms, Codiak has developed its proprietary engEx Platform to expand upon the innate properties of exosomes to design, engineer and manufacture novel exosome therapeutic candidates. Codiak has utilized its engEx Platform to generate a deep pipeline of engineered exosomes aimed at treating a broad range of disease areas, spanning oncology, neuro-oncology, neurology, neuromuscular disease and infectious disease.
MiRagen is a biopharmaceutical company that discovers and develops innovative microRNA-targeting therapies to improve human health, specifically in disease areas of high unmet medical need. The company leverages in-house expertise in miRNA biology, oligonucleotide chemistry, and drug development to evaluate and advance best-in-class technologies and high-potential drug candidates for its own pipeline or in conjunction with strategic partners. The company was founded in 2007 and headquartered in Boulder, Colorado.
23andMe, Inc. is a consumer genetics company that provides DNA analysis services through home-based saliva collection kits, enabling users to explore their ancestry, inherited traits, and health insights. Founded in 2006 and headquartered in Sunnyvale, California, the company offers various services, including Health + Ancestry and Ancestry + Traits, which provide insights into a person's genetic background and over 30 trait reports. 23andMe aims to help individuals understand the implications of their genetic information while also catering to researchers by offering categorized and searchable data. The company was co-founded by Linda Avey and Anne Wojcicki and has positioned itself as a significant player in the growing biotechnology and healthcare sectors.
Intellia Therapeutics is a genome editing company based in Cambridge, Massachusetts, focused on developing curative gene-editing treatments using the CRISPR/Cas9 technology. The company aims to address severe and life-threatening genetic diseases through both in vivo and ex vivo therapeutic approaches. Its pipeline includes programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Additionally, Intellia is developing engineered cell therapies for oncological and autoimmune diseases. The company has established collaborations with various entities, including leading pharmaceutical firms and research institutions, to enhance its research and development efforts. Founded in 2014, Intellia Therapeutics holds a comprehensive intellectual property platform for the therapeutic application of CRISPR technology, positioning it at the forefront of gene editing innovation.
WaVe Life Sciences Ltd. is a clinical stage genetic medicine company focused on developing novel stereopure oligonucleotides through its proprietary PRISM platform. The company aims to address genetic defects by either reducing the expression of harmful proteins or converting dysfunctional mutant proteins into functional ones. It primarily concentrates on neurological disorders affecting the central and neuromuscular systems. WaVe Life Sciences has established research and collaboration agreements with major pharmaceutical companies to advance its oligonucleotide therapeutics. Founded in 2012, the company is headquartered in Singapore and maintains research facilities in Boston and Japan. The organization was established by leading scientists in the field of chemistry and life sciences, underscoring its commitment to innovative approaches in nucleic acid therapeutics.
Ovid Therapeutics Inc. is a biopharmaceutical company based in New York, focused on developing innovative therapies for patients with neurological disorders, particularly rare diseases. Founded in 2014, the company is advancing a promising pipeline of drug candidates aimed at treating conditions such as angelman syndrome and fragile X syndrome. Its lead candidate, OV101, is in Phase III clinical trials for angelman syndrome and has completed Phase II trials for fragile X syndrome. Additionally, Ovid is developing OV935, currently in Phase II trials for cyclin-dependent kinase-like 5 deficiency disorder and Dravet syndrome. The company is also researching OV329 for infantile spasms and OV881 as a microRNA gene therapy for angelman syndrome. Ovid Therapeutics has established collaborations and licensing agreements with various pharmaceutical and research organizations to enhance its development efforts. The company's commitment to addressing unmet medical needs is underpinned by strong intellectual property and a focus on safe, effective treatments for patients facing challenging neurological conditions.
Editas Medicine is a clinical-stage company focused on developing innovative genome editing therapeutics to address serious diseases at the genetic level. Established in 2013 and headquartered in Cambridge, Massachusetts, with an additional site in Boulder, Colorado, the company leverages its proprietary CRISPR/Cas9 technology to create precise and corrective molecular modifications. This approach utilizes a protein-RNA complex, which includes the Cas9 enzyme and a guide RNA, to target and repair specific DNA sequences. Editas Medicine aims to translate its advances in genome editing into transformative human therapies, supported by a robust portfolio of patents and intellectual property that underpins its foundational technologies.
GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, specializing in the discovery and development of gene therapy treatments for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. The company utilizes two primary technology platforms: mitochondrial targeting sequences and optogenetics, aiming to preserve or restore vision in patients affected by severe retinal diseases. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III clinical trials for treating Leber hereditary optic neuropathy associated with ND4 gene mutations, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. Additionally, GenSight is advancing various preclinical programs targeting both ophthalmic and neurodegenerative conditions, reflecting its commitment to innovative solutions for patients with significant vision impairment or blindness. GenSight Biologics was founded in 2012.
Twist Bioscience Corporation, a synthetic biology company, manufactures and sells synthetic DNA-based products. The company’s DNA synthesis platform enables the manufacturing of synthetic DNA by writing DNA on a silicon chip. It offers synthetic DNA-based products, including synthetic genes, tools for sample preparation, antibody libraries for drug discovery and development, and DNA as a digital data storage medium. The company has a collaboration agreement with ImmunoPrecise Antibodies Ltd. Twist Bioscience Corporation was founded in 2013 and is headquartered in South San Francisco, California.
Private Equity Round in 2012
Humanigen, Inc. is a clinical-stage biopharmaceutical company based in Burlingame, California, that specializes in the development of proprietary monoclonal antibodies for immunotherapy and oncology. The company's lead product candidate, Lenzilumab, targets granulocyte-macrophage colony-stimulating factor and is currently undergoing Phase Ib/II clinical trials for chronic myelomonocytic leukemia and juvenile myelomonocytic leukemia. Additionally, Humanigen is developing Ifabotuzumab, which targets the EphA3 receptor and has completed the Phase I dose escalation in multiple hematologic malignancies, as well as HGEN005, an anti-EMR1 monoclonal antibody in pre-clinical stages for eosinophilic diseases. The company collaborates with Kite Pharma to conduct a multi-center Phase Ib/II study of Lenzilumab in patients with relapsed or refractory diffuse large B-cell lymphoma and has partnered with the Department of Defense to further develop Lenzilumab for potential emergency use in COVID-19. Founded in 2000, Humanigen was formerly known as KaloBios Pharmaceuticals and rebranded in August 2017.