Operator of a biotechnology company intended for pioneering the development of genetic medicines for treating intractable diseases. The company specializes in developing genetic drugs by simultaneously engineering (Adeno-associated viruses) AAV capsids that bind to the human Transferrin Receptor 1 (TfR1) to enable intravenous delivery of therapeutics that use receptor-mediated transcytosis across the blood brain barrier allowing broad penetration of the central nervous system, and payloads to overcome limitations in cellular access, gene expression, pre-existing immunity and manufacturability, thereby enabling patients to treat debilitating diseases with limited options.
PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the delivery of nucleic acid therapeutics, particularly antisense oligonucleotides. Established in 2018, PepGen aims to enhance the clinical efficacy of these therapeutics through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative technology employs engineered peptides designed for optimized tissue penetration, cellular uptake, and nuclear delivery, enabling effective transport of oligonucleotides to various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. By leveraging its drug delivery capabilities, PepGen seeks to transform the treatment landscape for severe neuromuscular and neurological diseases.
Jaguar Gene Therapy is a clinical-stage biotechnology company focused on advancing gene therapy solutions for patients with severe genetic diseases. The company aims to address significant unmet medical needs by developing therapies that target nonworking or absent genes, introducing healthy copies of these genes to restore their function. By accelerating the process of bringing innovative gene therapies from research to clinical application, Jaguar Gene Therapy seeks to improve patient outcomes and enhance the overall standard of care for those affected by genetic disorders.
Jaguar Gene Therapy is a clinical-stage biotechnology company focused on advancing gene therapy solutions for patients with severe genetic diseases. The company aims to address significant unmet medical needs by developing therapies that target nonworking or absent genes, introducing healthy copies of these genes to restore their function. By accelerating the process of bringing innovative gene therapies from research to clinical application, Jaguar Gene Therapy seeks to improve patient outcomes and enhance the overall standard of care for those affected by genetic disorders.
Encodia, Inc. engages in proteomics research and creates scalable and parallelized approaches to protein analysis. The company was founded in 2015 and is based in San Diego, California.
Cystetic Medicines
Funding Round in 2020
Cystetic Medicines develops a treatment that potentially could help people with cystic fibrosis (CF), regardless of genetic mutation.
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.
Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, dedicated to leveraging the body's natural biology to repair or reverse damage from various degenerative diseases. The company employs a unique Progenitor Cell Activation approach, utilizing combinations of small molecules to stimulate progenitor cells to regenerate functional tissue. Its lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at addressing the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is developing therapies for a variety of degenerative conditions, including multiple sclerosis and ailments affecting muscle, skin, gastrointestinal tract, and bone health. The company has also established a collaboration agreement with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics is focused on disease modification through innovative therapeutic strategies.
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.
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.
Concert Genetics, Inc. is a healthcare information technology company based in Franklin, Tennessee, that specializes in simplifying and unifying genetic testing processes for various stakeholders, including hospitals, health systems, patients, and clinicians. The company offers software and analytical tools designed to streamline genetic test selection, ordering, payment, and management operations. Its services include claim-to-test matching, medical policy support, utilization management, and business intelligence reports, catering to health plans, hospitals, laboratories, and regulatory agencies. By providing a comprehensive platform, Concert Genetics helps physicians access the necessary information to efficiently order genetic tests for diagnosing over 2,000 diseases, thereby enhancing clinical outcomes. The company also offers the Concert Genetics Benefit Program, which facilitates access to clinically appropriate genetic testing while improving payment integrity and reducing administrative burdens. Founded in 2010 and originally known as NextGxDx Inc., Concert Genetics rebranded in 2017 and has established a strategic partnership with Trapelo Health.
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.
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.
Ribon Therapeutics, Inc. is a biotechnology company based in Lexington, Massachusetts, founded in 2015. The company is focused on developing innovative cancer therapies that target monoPARP proteins, which are essential regulators of cancer cell survival. Ribon's scientific founders have made significant contributions to the understanding of PARPs, or poly(ADP-ribose) polymerases, and their roles in cancer. Leveraging this expertise, Ribon is establishing a proprietary drug discovery platform aimed at exploring the molecular actions and biological functions of monoPARPs. This platform supports the creation of a pipeline of therapeutics that selectively target these proteins to potentially treat cancer and other diseases. Ribon Therapeutics is supported by notable life science investors, enhancing its capability to advance its research and development efforts.
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.
Audentes Therapeutics is a clinical-stage biotechnology company dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. The company is advancing several therapies, including AT132, currently in Phase I/II clinical trials for X-linked myotubular myopathy, and AT342, also in Phase I/II studies for Crigler-Najjar syndrome. Additionally, Audentes is exploring AT845 in preclinical studies for Pompe disease and AT307 for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. The company is also working on vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Collaborations include a partnership with Genethon for XLMTM treatments and an agreement with the University of Pennsylvania for Crigler-Najjar syndrome products. Founded in 2012 and headquartered in San Francisco, California, Audentes Therapeutics operates as a subsidiary of Astellas US Holding, Inc.
Based in Dallas, Texas, AveXis is a clinic-ready, synthetic biology platform company. AveXis has, at its core, a desire to establish unique industry and research alliances which will bring innovative treatments to people with unmet medical needs. Our work in spinal muscular atrophy (SMA), a rare/orphan disease, is our first focus.
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.
Based in Dallas, Texas, AveXis is a clinic-ready, synthetic biology platform company. AveXis has, at its core, a desire to establish unique industry and research alliances which will bring innovative treatments to people with unmet medical needs. Our work in spinal muscular atrophy (SMA), a rare/orphan disease, is our first focus.
Audentes Therapeutics is a clinical-stage biotechnology company dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. The company is advancing several therapies, including AT132, currently in Phase I/II clinical trials for X-linked myotubular myopathy, and AT342, also in Phase I/II studies for Crigler-Najjar syndrome. Additionally, Audentes is exploring AT845 in preclinical studies for Pompe disease and AT307 for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. The company is also working on vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Collaborations include a partnership with Genethon for XLMTM treatments and an agreement with the University of Pennsylvania for Crigler-Najjar syndrome products. Founded in 2012 and headquartered in San Francisco, California, Audentes Therapeutics operates as a subsidiary of Astellas US Holding, Inc.
Spark Therapeutics, Inc. is dedicated to developing gene therapy products aimed at treating debilitating genetic diseases. The company is known for LUXTURNA, a treatment for patients with biallelic RPE65 mutation-associated retinal dystrophy, and has an extensive pipeline that includes candidates like SPK-8011 and SPK-8016 for hemophilia, SPK-7001 for choroideremia, and SPK-9001 for hemophilia B. Additionally, Spark is exploring liver-directed gene therapies such as SPK-3006 for Pompe disease and has programs targeting neurodegenerative diseases, including Huntington's disease and a form of Batten disease. Spark Therapeutics also engages in collaborations, notably with Pfizer for the SPK-FIX program for hemophilia B, and has a licensing agreement with Novartis for the commercialization of LUXTURNA outside the United States. Established in 2013 and based in Philadelphia, Spark Therapeutics operates as a subsidiary of Roche Holding AG and leverages a proprietary manufacturing platform to support its clinical studies across various therapeutic areas.
Dicerna Pharmaceuticals is a biopharmaceutical company dedicated to the discovery and development of innovative treatments primarily for liver-related diseases, including rare inherited conditions, chronic liver diseases, and viral infections. Utilizing its proprietary GalXC RNA interference technology, Dicerna focuses on silencing disease-causing genes to address various health issues. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and belcesiran for genetic liver disease, among others. The company's extensive research efforts also encompass treatments for cardiovascular diseases and neurodegeneration. Dicerna has established strategic collaborations with prominent pharmaceutical companies to enhance its development capabilities. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna is at the forefront of advancing RNA-based therapeutics.
Pacific Biosciences designs, develops, and manufactures advanced sequencing systems that address complex genetic challenges. Utilizing single molecule real-time (SMRT) sequencing technology, it enables the real-time detection of biological processes at the single-molecule level. The company offers products such as the PacBio RS II and Sequel Systems, which are capable of conducting, monitoring, and analyzing biochemical reactions in real time. In addition to sequencing systems, Pacific Biosciences provides consumables like SMRT cells and various reagent kits essential for template preparation, binding, and sequencing. Its diverse customer base includes research institutions, commercial laboratories, genome centers, and various sectors such as pharmaceuticals and agriculture. The company markets its products through a direct sales force in North America and Europe, along with distribution partners in Asia, the Middle East, and Latin America. Founded in 2000, Pacific Biosciences is headquartered in Menlo Park, California.
WaferGen Bio-systems, Inc. is an emerging genomic analysis company in the early stage of commercialization. The company offers the transformative SmartChip Real-Time PCR System—a next-generation Real-Time PCR System for profiling and validation of gene expression patterns (biomarkers) on a single platform. The SmartChip System provides a range of high-throughput capabilities including microRNA and mRNA gene expression profiling and single nucleotide polymorphism (SNP) genotyping.
Pacific Biosciences designs, develops, and manufactures advanced sequencing systems that address complex genetic challenges. Utilizing single molecule real-time (SMRT) sequencing technology, it enables the real-time detection of biological processes at the single-molecule level. The company offers products such as the PacBio RS II and Sequel Systems, which are capable of conducting, monitoring, and analyzing biochemical reactions in real time. In addition to sequencing systems, Pacific Biosciences provides consumables like SMRT cells and various reagent kits essential for template preparation, binding, and sequencing. Its diverse customer base includes research institutions, commercial laboratories, genome centers, and various sectors such as pharmaceuticals and agriculture. The company markets its products through a direct sales force in North America and Europe, along with distribution partners in Asia, the Middle East, and Latin America. Founded in 2000, Pacific Biosciences is headquartered in Menlo Park, California.
ZymoGenetics is focused on the discovery, development, and early manufacture of therapeutic proteins. ZymoGenetics is a fully-integrated research and development organization with a broad range of skills and technologies, including scientific computing, molecular and cellular biology, protein chemistry, antibody generation and engineering, clinical development and operations, regulatory affairs, quality, drug formulation, process development and protein manufacturing.
Third Wave Technologies, Inc. is involved in the development and marketing of molecular diagnostic products that cater to a range of DNA and RNA analysis applications. The company provides innovative solutions for physicians and researchers focused on disease analysis and treatment. Its proprietary Invader chemistry is utilized across several segments, including women's health, infectious diseases, genetics, pharmacogenetics, and oncology. Third Wave offers in vitro diagnostic devices and analyte-specific reagents that enable laboratories to perform various assays, such as hepatitis C virus genotyping and testing for inherited disorders and genetic mutations. Additionally, the company develops products for research and agricultural biotechnology markets. Its clientele comprises clinical laboratories, pharmaceutical and biotechnology companies, academic institutions, and healthcare providers. Third Wave employs direct sales strategies and collaborative partnerships to market its products in the United States and Europe. Founded in 1993, Third Wave Technologies is headquartered in Madison, Wisconsin, and is currently a subsidiary of Hologic Inc.