Clinical TrialsInformation and insight on the latest clinical trials
- Breakthrough Designation for Idiopathic Pulmonary Fibrosis Therapy
The U.S. Food and Drug Administration (FDA) granted PRM-151, a novel investigational anti-fibrotic immunomodulator, Breakthrough Therapy designation for Idiopathic Pulmonary Fibrosis (IPF).
- KEYNOTE-695 Study of TAVO + Keytruda in Refractory Metastatic Melanoma
Dan O’Connor, President, Director and Chief Executive Officer of OncoSec discusses the KEYNOTE-695 Study of TAVO + Keytruda in Refractory Metastatic Melanoma.
- The Role of IL-12 in Anti-PD-1 Therapies with TAVO
Dan O’Connor, President, Director and Chief Executive Officer of OncoSec discusses his company and it’s focus on developing cytokine-based intratumoral immunotherapies to stimulate the body’s immune system to target and attack cancer.
- Biallelic RPE65 Mutation-Associated Retinal Dystrophy
Ron Philip, Chief Commercial Officer of Spark Therapeutics, discusses biallelic RPE65 mutation-associated retinal dystrophy, a rare retinal disorder.
- Primary Hyperoxaluria Type 1: Overview and an Emerging Treatment
Pushkal Garg, MD, Chief Medical Officer at Alnylam discusses Primary Hyperoxaluria Type 1 and his company’s investigational RNAi therapy, lumasiran.
- Clinical Trial for X-linked Severe Combined Immunodeficiency
Manuel Litchman, MD, President, Chief Executive Officer at Mustang Bio discusses his company’s clinical program focused on X-linked severe combined immunodeficiency (SCID).
- Rare Disease Day: Spotlight on Rare Disorders and the Need for Clinical Trials
February 28 is the 12th Annual Rare Disease Day, the theme of which this year is “bridging health and social care”. This day brings together the rare disease community and highlights the need for better coordination of medical, social, and support services to lessen the burden that rare diseases have on the daily lives of patients, their families, and caregivers.
- FDA Accepts Golodirsen NDA for Duchenne Muscular Dystrophy Patients Amenable to Skipping Exon 53
The Food and Drug Administration’s (FDA’s) Division of Neurology has accepted its New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053) and provided a regulatory action date of August 19, 2019.
- Acute Hepatic Porphyria: Overview and Emerging Therapies
Pushkal Garg, MD, Chief Medical Officer at Alnylam discusses Acute Hepatic Porphyria (AHPs) and givosiran, an investigational, subcutaneously administered RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of AHP.
- Management of Hematologic Diseases With Cellular and Immunotherapies
Mohamad Mohty, MD, discusses the management of hematologic diseases with cellular and immune therapies such as Chimeric antigen receptor (CAR) T cells, which have recently emerged as a novel therapeutic approach, yielding complete responses even in heavily pretreated patients with leukemia, lymphoma, and myeloma.