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Rare Genomics Institute empowers patient communities to accelerate research by helping fund and create personalized research projects based on individuals with rare diseases. Their first initiative is to sequence the genomes of these patients. To do this, they provide three innovative services. First, a micro-funding website: an online fundraising platform and social network that helps patient communities raise funds. Second, a researcher network: access to top genomics researchers who are willing to sequence and analyze these patients. Third, a clinical network: connecting patients with clinicians and genetic counselors who will help interpret and translate the individualized research findings.

Genomics, Rare Diseases, Next-Generation Sequencing, Crowdfunding

Provid Pharmaceuticals Inc. was founded to discover novel therapeutics for autoimmune diseases using peptide mimetics technology.   Provid’s strategy has resulted in the discovery of PV-267, a small molecule drug candidate for the treatment of multiple sclerosis (MS) and leads for rheumatoid arthritis and other autoimmune diseases that operate by blocking disease-associated MHC class II molecules.

Provid’s MS drug candidate, PV-267 uniquely inhibits the MHC Class II molecule HLA-DR2b, the most important gene target in the autoimmune disease process in MS.  MS is genetically linked with HLA-DR2b (DRB1*1501).  DR2b is carried by a majority of MS patients (ca. 700,000 worldwide) and binds and presents antigenic myelin fragments to autoreactive T cells, triggering the autoimmune response that damages myelin insulation on nerves, leading to MS disease symptoms and disability.  Provid designed PV-267 to bind tightly and selectively to DR2b, blocking antigen binding.  PV-267 has been shown to disrupt the autoimmune process in MS animal models and in human cells from DR2+ patients.  Because PV-267 does not compromise other aspects of the immune system, its potential for superior safety is a major advantage over current disease-modifying drugs.

Recent data have shown that PV-267 has high oral bioavailability in a proprietary formulation being developed in collaboration with Enteris Biopharma.  Oral efficacy, while not essential to test the concept of the DR2 inhibitory mechanism, is an important factor commercially and for the MS patient.

Early Clinical Trial Concept.  Because of its DR2b antagonist mechanism, it is feasible to assess immunological efficacy in DR2b+ individuals in early clinical trials, greatly reducing the clinical development risk and clearing the path for a targeted phase II efficacy trial.  These studies are being planned by our Clinical Advisory Board under the leadership of Prof. Larry Steinman at Stanford. 

Development Plan. Provid will pursue preclinical and early clinical studies of our DR2b inhibitors and is seeking partnerships with pharma company partners.

1) Developement of patented injectable resorbable biomaterials which harden in situ, allowing minimally invasive surgery and are quickly resorbed by the cellular activity and replaced by natural bone. 2) Design and development of combinaison devices utilizing synthetic bone substitute materials and therapeutic agents as well as autologous tissue which will address such clinical challenges as prevention of osteoporotic fractures, prevention of infection and enhanced bone ingrowth

1) Bone defect filling via MIS techniques for younger patients; 2) "Super-Fast" resorption and remodeling to boost bone reconstruction for non union management; 3) Local treatment or prevention of osteoporotic patient ; 4) management of bone Infection by our innovative calcium phosphate cement combined with antibiotic (exposed trauma, military application...)

1) Orthopaedic: Trauma and Reconstruction; 2) Spine Surgery; 3)Dentistry

1) Patented technology which provides unique soluctions to address key clinical needs; 2) Proprietary pipeline of innovative bone tissue engineering solutions; 3) Fully integrated organization and facilities which include R&D, manufacturing, regulatory, sales and marketing; 4) Strong relationships with leading global orthopedic companies; 5) years of very successful sales and clinical experience in 23 countries of the world

Formally known as The State University of New York Health Science Center at Brooklyn—but better known to our patients and Brooklyn neighbors as SUNY Downstate Medical Center—we are older than the Brooklyn Bridge. 

We trace our roots back to 1860, when a school of medicine was founded at the Long Island College Hospital. The new college’s faculty revolutionized medical education in this country by bringing the teaching of medicine to the hospital bedside, thus rejecting the idea that physicians should be trained exclusively in university lecture halls. 

Today, SUNY Downstate is one of the nation’s leading urban medical centers. SUNY Downstate comprises a College of Medicine, College of Health Related Professions, College of Nursing, School of Graduate Studies, School of Public Health, and University Hospital of Brooklyn.