ResearchFocus on the pharmaceutical and biotech industry
- The Success of the Orphan Drug Act
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals discusses the success of the Orphan Drug Act (ODA).
- Corbus Pharmaceuticals Update
Yuval Cohen, PhD, CEO of Corbus Pharmaceuticals discusses the appointment of John K. Jenkins, MD to its Board of Directors and the upcoming data from open-label extensions (OLEs) of its systemic sclerosis (SSc) and dermatomyositis (DM) Phase 2 studies which will be presented at the Annual European Congress of Rheumatology.
- Baebies: A New Born Screening Company
Richard West, Founder and CEO of Baebies, a newborn testing diagnostic company, provides an overview of his platform.
- Cure Pharmaceuticals: A Drug Delivery and Development Company
Jessica Rousset, Chief Operating Officer of CURE Pharmaceutical, discusses her company. CURE is an integrated drug delivery and development company and has proprietary drug dosage forms and delivery systems.
- FDA and EMA Grants Orphan Drug Designation for ENPP1 Deficiency Treatment
The U.S. Food and Drug Administration (FDA) and European Medicines Agency’s (EMA) Committee for Orphan Medical Products (COMP) granted an orphan drug designation to Inozyme Pharma for their product INZ-701 for the treatment of ENPP1 deficiency, a serious, life-threatening calcification disorder.
- Daniel de Boer Discusses His Company’s Focus on Usher Syndrome
Daniel de Boer, Founder and CEO of ProQR, discusses his company’s clinical program focusing on Usher syndrome is a rare genetic disease that is the leading cause of combined deafness and blindness.
- Scioto Biosciences Focus on Microbiome Therapeutics
Dr. Joe Trebley, Co-founder and CEO of Scioto Biosciences, discusses his company’s focus on developing a novel probiotic delivery platform that provides enhanced function and persistent probiotic benefits to patients.
- Human Gene Therapy for Rare Diseases: Draft Guidance for Industry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.