Bill OBrien
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.
SpecialtiesGenomics, Rare Diseases, Next-Generation Sequencing, Crowdfunding
Dr Gary Olson, PhD
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.
Provid Pharmaceuticals
President & CEOMr Kathleen Otto
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.
BioBAT
Executive DirectorDr Felicia Pagliuca
Type 1 diabetes (T1D), formerly known as juvenile diabetes, is a chronic, life-threatening disease that affects millions of people worldwide. In the United States, 30,000 new cases are estimated every year with half of those cases diagnosed in young children. Type 1 diabetes is an autoimmune disease in which the patient’s immune system goes awry and attacks and destroys the pancreatic beta cells. Beta cells are responsible for regulating blood sugar (glucose) levels by producing precise amounts of the essential hormone insulin.
The discovery of injectable insulin in the 1920s changed T1D from a uniformly fatal disease with a life expectancy of months to one that could be carefully managed for decades through multiple daily blood glucose measurements and insulin injections. However, insulin injections are not a cure and patients face a lifetime of difficult disease management and serious complications including kidney failure, blindness and nerve damage. Despite nearly a century passing since the discovery of insulin, insulin injection remains the only treatment available to patients.
Semma Therapeutics was founded to develop transformative therapies for patients who currently depend on insulin injections. Recent work in the laboratory of Professor Douglas Melton led to the discovery of a method to generate billions of functional, insulin-producing beta cells in the laboratory. These cells develop in islet-like clusters grown from stem cells. Initial preclinical work in animal models of diabetes has shown that transplantation of these cells are sufficient to control blood glucose levels. This breakthrough technology has been exclusively licensed to Semma Therapeutics for the development of a cell-based therapy for diabetes.
Ongoing research at Semma Therapeutics is focused on combining these proprietary cells with a state-of-the-art cell delivery and immune protection strategy that can protect these cells from the patient’s immune system and allow the beta cells to function as they do in non-diabetic individuals. Implantation of the beta cell-filled device has the potential to provide a true replacement for the missing beta cells in a diabetic patient and would not require patient immunosuppression. Semma Therapeutics is working to bring this new therapeutic option to the clinic and improve the lives of patients with diabetes.
Semma Therapeutics
Director of Technology and Corporate DevelopmentAsit Panja
AlfaGene is an innovative biotechnology company that uses its own novel proprietary cutting edge adult stem cell technology to create the only stem cell and non-transformed primary epithelial cell derived model of each segment of the entire gastrointestinal (GI) tract.
Gastrointestinal diseases affect millions of people, young and old all across the globe and cause heavy economical consequences for nations around the world. In the United States alone, GI diseases consume over $22 billion health care dollars annually with treatments that are usually not curative but just symptom relieving. GI stem and epithelial cells are the main cellular component affected in the malignant transformation, inflammation, immune responses (e.g. autoimmune response), and disease of the GI tract, including:
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colorectal cancer (CRC)
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ulcerative colitis (UC)
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Crohn's Disease (CD)
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irritable bowel syndrome (IBS)
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short bowel syndrome
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gastroesophageal reflux disease (GERD) and Barrett’s esophagitis.
AlfaGene Bioscience Inc
Chief Scientific OfficerMr Lynne Parker
Trillium Medical Center is a South Florida based medical facility specializing in Internal Medicine. The center has facilities to diagnose and treat Internal Medical problems. By providing our patients with state of the art Preventative, Acute, and Chronic treatment options we are able to help patients lose weight safely, maintain healthy diet regimens and life styles, and ensure their health conditions are monitored on a regular basis.
By extending our capabilities to provide 24 hour/365 Day access to patient's health records, each patient can gain access to their important and vital medical information no matter where they travel or which physician they visit, even if it is not at our center.
We also offer a variety of "In House" tertiary services including an in house Med Spa complete with Esthetic services including Botox, Juvederm, Microdermabrasion and other wellness treatments like Weight Management and Nutrition training.
Trillium Medical LLC
Managing PartnerMr Joseph Patanella
HackensackUMC Mountainside is unique among New Jersey hospitals. While many others are seeking new owners because their futures remain uncertain, one of the nation’s most prestigious hospitals, HackensackUMC and LHP Hospital Group, Inc. one of the nation’s most innovative capital and healthcare management firms are choosing to affiliate with HackensackUMC Mountainside because of its strength and stability.