Rituximab Therapy Correlates with Delayed Progression in Secondary Progressive Multiple Sclerosis
The study implies that B-cell depletion by rituximab therapy may be therapeutically beneficial in patients with secondary progressive multiple sclerosis.
Orphan Drug Designation Granted to Bietti's Crystalline Dystrophy Gene Therapy
The FDA has granted an orphan drug designation to ReflectionBio's AAV-based gene therapy, the RBIO-101 program (AAV.CYP4V2), for the treatment of Bietti's crystalline dystrophy (BCD).
FDA Approves Nivolumab for Small Cell Lung Cancer
The FDA has approved Bristol-Meyers Squibb Company’s nivolumab (Opdivo) for the treatment of metastatic small cell lung cancer (SCLC) that has progressed after platinum-based chemotherapy and at least 1 other line of therapy.
Gene Therapy in Patients with DMD Demonstrates Outstanding Preliminary Results
Positive preliminary results from the Sarepta Therapeutics, Inc phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin vector in individuals with Duchenne muscular dystrophy (DMD) indicate outstanding promise.
FDA Approves Pembrolizumab for PMBCL
The FDA has approved pembrolizumab for the treatment of refractory primary mediastinal large B-cell lymphoma (PMBCL) or who have relapsed after 2 or more previous lines of therapy.
Gilteritinib Proves Promising for the Treatment of FLT3mut + Acute Myeloid Leukemia
Planned analyses of phase 3 trial of gilteritinib as a maintenance therapy after allogeneic hematopoietic stem cell transplantation in patients with Fms-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) mutations in acute myeloid leukemia (AML).
Panel of Experts Discuss Gene Therapy Gaps in Hemophilia
Marijke van den Berg, MD, PhD, Alok Srivastava, MD, and Glenn Pierce, MD, PhD discuss gene therapy in hemophilia.
MPS IIIA Gene Therapy, ABO-102, Shows Efficacy in Trial Update
The phase 1/2 trial for ABO-102 (AAV-SGSH), clinical gene therapy for the treatment of Sanfilippo syndrome type A (MPS III A) shows efficacy in trial update.
FDA Grants Rare Pediatric Disease Designation to LGMD Type 2E
The FDA has granted Rare Pediatric Disease Designation to Myonexus Therapeutics for its MYO-101, which is an AAV-based gene therapy for the treatment of limb girdle muscular dystrophy (LGMD) type 2E.
FDA Accepts IND Application for BIVV003, a Gene Therapy for Sickle Cell Disease
The US Food and Drug Administration (FDA) has accepted the Bioverativ's Investigational New Drug (IND) application for BIVV003, a gene-edited cell therapy candidate for the treatment of people with sickle cell disease.
First Hemophilia A Patient in Phase 1/2 Study Dosed with Valoctocogene Roxaparvovec
The first patient has been dosed in aphase 1/2 study (BMN 270-203) evaluating the investigational gene therapy, valoctocogene roxaparvovec, in severe hemophilia A patients with pre-existing AAV5 antibodies.
Is New Treatment on the Horizon for Dystrophic Epidermolysis Bullosa?
CEO of Krystal Biotech discusses first topical gene therapy for dystrophic epidermolysis bullosa.
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