BioPharmaFocus on the pharmaceutical and biotech industry
- Human Gene Therapy for Rare Diseases: Draft Guidance for Industry
Statement from FDA Commissioner Scott Gottlieb, MD on agency’s efforts to advance development of gene therapies
- Scioto Biosciences: Focused on Reducing Necrotizing Enterocolitis
Jim Schulz, Co-founder and Chief Financial Officer of Scioto Biosciences, discusses his company’s focus on developing therapies devoted to having a impact on the delivery of microbiome therapeutics with the goal of reducing the incidence of necrotizing enterocolitis (NEC).
- Primary Biliary Cholangitis: Overview, Diagnosis, and Treatment Options
Gail Cawkwell, MD, PhD, Senior Vice President, Medical Affairs at Intercept, provides an overview of primary biliary cholangitis (PBC), a rare liver disease that is caused by an autoimmune reaction.
- ArQule: Focused on Targeted, Biomarker-Defined Patient Population
Brian Schwartz, Chief Medical Officer of ArQule, discusses his company and it’s clinical-stage pipeline consistlng of five drug candidates, all of which are in targeted, biomarker-defined patient populations.
- Baebies: A New Born Screening Company
Richard West, Founder and CEO of Baebies, a newborn testing diagnostic company.
- Synspira: Focusing on Rare Pulmonary Diseases
Dr. Shenda Baker is Chief Executive Officer of Synspira, a clinical stage biotech company developing a new class of glycopolymer-based therapeutics that have the potential to address a major unmet need associated with pulmonary disorders-
- Imara: A Clinical Stage Biotech Focused on Sickle Cell Disease
James McArthur, PhD, Founder of Imara and President, R&D of Cydan discusses the development of IMR-687 for the treatment or rare hemoglobinopathies.
- Alnylam’s Pending Approval of Patisiran
Barry Greene, President of Alnylam, discusses his company and it’s pending approval of Patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of hereditary ATTR amyloidosis (hATTR amyloidosis).
- The Biotech Industry in Ireland
Rory Mullen, Vice President, New Business Bio-Pharma and Food, discusses Ireland’s Life Sciences sector, which has grown from very humble beginnings in the 1960s to reach global significance.
- Corbus Pharma Provides an Update on Their Clinical Trial Programs
Yuval Cohen, PhD, CEO of Corbus Pharmaceuticals provides and update on his company’s clinical trial programs.