| Name | Title | Contact Details |
|---|
Avidity Biosciences, Inc.`s mission is to profoundly improve people`s lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity`s proprietary AOCs are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics. Avidity`s advancing and expanding pipeline has three programs in clinical development. AOC 1001 is designed to treat people with myotonic dystrophy type 1 (DM1) and is currently in Phase 1/2 development with the ongoing MARINA™ and MARINA-OLE™ trials. AOC 1020 is designed to treat people living with facioscapulohumeral muscular dystrophy (FSHD) and is currently in Phase 1/2 development with the FORTITUDE™ trial. AOC 1044 is designed for people with Duchenne muscular dystrophy (DMD) mutations amenable to exon 44 skipping and is currently in Phase 1/2 development with the EXPLORE44™ trial. AOC 1044 is the first of multiple AOCs the company is developing for DMD. Avidity is also broadening the reach of AOCs beyond muscle tissues through both internal discovery efforts and key partnerships as the company continues to deliver on the RNA revolution. Avidity is headquartered in San Diego, CA.
The trillions of microbes that call us home – the human microbiome – perform critical functions that preserve our health including helping develop our immune system, manufacturing nutrients such as vitamins, and harvesting energy from the diet. Alterations of the human microbiome are increasingly recognized as a key factor in autoimmune, metabolic, infectious and many other diseases. For millions of years, humans have co-evolved with this community of microbes, communicating in an ancient language that holds critical clues about how autoimmune and infectious diseases develop. This language is based on a system of signals that our resident microbes use to successfully colonize us, such as byproducts of bacterial metabolism and factors that bacteria use to adapt to human niches. Recent discoveries have shown that most of these signals do not lead to disease, rather, they promote our long-term survival. Vedanta Biosciences is developing an innovative class of therapies that modulate pathways of interaction between the human microbiome and the host immune system. Vedanta Biosciences was co-founded by PureTech and a group of world renowned experts in immunology and microbiology.
Sera Prognostics is a leading proteomic and bioinformatics company dedicated to improving the lives of women and babies through precision biomarker-based tests designed to enhance pregnancy care. Sera’s vision is to deliver pivotal information in early pregnancy to physicians, to help them to improve the health of their patients and reduce costs of healthcare delivery. Sera’s PreTRM® Test reports to a physician the individualized risk of a pregnant woman to deliver prematurely, enabling earlier proactive interventions in patients with higher risk. Rigorous clinical validation of PreTRM® Test performance (accuracy of predicting premature delivery) was reported in the American Journal of Obstetrics & Gynecology in 2016 in a U.S. cohort of 5,501 patients across 11 centers. Preterm birth is defined as any birth before 37 weeks gestation and is a leading cause of illness and death in newborns. The 2020 March of Dimes Report Card shows that of nearly 4 million babies born annually in the U.S., more than one in ten is born prematurely. Prematurity is associated with a significantly increased risk of major long-term medical complications, including learning disabilities, cerebral palsy, chronic respiratory illness, intellectual disability, seizures, vision and hearing loss, and can generate significant cost throughout the lives of affected children. The annual U.S. health care costs to manage complications of prematurity were estimated at $31.5B for 2015. Sera is also developing a robust pipeline of innovative tests focused on other complications of pregnancy. Using its advanced proprietary mass spectrometry and bioinformatics platform technologies, Sera detects biologically important protein expression differences to build high performing predictions of risk for adverse pregnancy outcomes (including preterm birth, preeclampsia, gestational diabetes, growth restriction, and others).
AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC`s lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC`s product pipeline includes six named ophthalmology development programs across five targets (X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration and blue cone monochromacy), two non-ophthalmology programs (alpha-1 antitrypsin deficiency and adrenoleukodystrophy) and AGTC is continuing to develop early research studies in additional indications. The company is also exploring genetic defects in cells in the inner ear that lead to deafness and expects to advance several product candidates into development within the next few years. AGTC employs a highly targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need, clinical feasibility and commercial potential. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as, expertise in the formulation, manufacture and physical delivery of gene therapy products
CHUL Research Center is a Quebec, QC-based company in the Healthcare, Pharmaceuticals, and Biotech sector.