Clinical TrialsInformation and insight on the latest clinical trials
- Human Gene Therapy for Rare Diseases: Draft Guidance for Industry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).
- 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.
- APOLLO Phase 3 Clinical Study Results for Hereditary Transthyretin-Mediated Amyloidosis
Alnylam Pharmaceuticals announced the publication of the pivotal study results from the APOLLO Phase 3 trial of patisiran in The New England Journal of Medicine (NEJM).
- FDA Grants Fast Track Designation to CUTX-101 for Classic Menkes Disease
The U.S. Food and Drug Administration (FDA) granted Fast Track Designation to Cyprium Therapeutics’ Copper Histidinate, (CUTX-101), for patients diagnosed with classic Menkes disease who have not demonstrated significant clinical progression.
- 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).
- ELX-02: Emerging Therapy for Cystic Fibrosis
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals provides an overview of his company’s leading drug candidate, ELX-02 for cystic fibrosis.
- Alexion Submits BLA For Priority Review And Approval Of ALXN1210
Alexion Pharmaceuticals has submitted a Biologics License Application (BLA) to the YOU.S. Food and Drug Administration (FDA) for approval of ALXN1210 for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare, potentially deadly disease of the blood.T
- First Participant Dosed in Phase 2 Study of Vosoritide for Children with Achondroplasia
BioMarin Pharmaceutical dosed the first participant in their global phase 2 study for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in infants and young children with achondroplasia.
- FDA Grants Orphan Drug Designation to New Treatment for Rett Syndrome
The U.S. Food and Drug Administration has granted Orphan Drug Designation to AMO Pharma for it’s investigational therapy called AMO-04 for the treatment of Rett syndrome, a rare childhood neurodevelopmental disorder.