Krista Rossi

The study implies that B-cell depletion by rituximab therapy may be therapeutically beneficial in patients with secondary progressive multiple sclerosis.

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).

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.

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.

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.

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).

Marijke van den Berg, MD, PhD, Alok Srivastava, MD, and Glenn Pierce, MD, PhD discuss gene therapy in hemophilia.

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.

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.

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.

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.

CEO of Krystal Biotech discusses first topical gene therapy for dystrophic epidermolysis bullosa.