Feed Category: Gene Therapy

Updated data from studies of beti-cel in 63 patients with transfusion-dependent beta-thalassemia showed sustained transfusion independence and improvements in iron management through up to 9… [Read More]

In this piece, we will take a look at the 12 best genomics stocks to buy now. If you want to skip our introduction to… [Read More]

Vertex Pharmaceuticals (NASDAQ: VRTX) has built a billion-dollar cystic fibrosis (CF) treatment empire, but investors have been looking for something more. When Vertex announced the… [Read More]

Allogene Therapeutics, Inc.Comprehensive Review of All Patients in the Phase 1 ALPHA/ALPHA2 Trials Demonstrate that Adding Investigational ALLO-647 to Standard Lymphodepletion Can Yield Durable Responses… [Read More]

Oral presentation to include data on 47 patients through five years of follow-up (median 35.5 months, range 0.3-61 months)Endpoints of sVOE-CR and VOE-CR achieved in… [Read More]

Key InsightsThe considerable ownership by private equity firms in Solid Biosciences indicates that they collectively have a greater say in management and business strategyA total… [Read More]

Vertex, CRISPR Therapeutics Get FDA Approval for First CRISPR Gene-Editing Therapy in USIn this article: Oops!Something went wrong.Please try again later.Oops!Something went wrong.Please try again… [Read More]

The FDA approved gene-editing therapy Casgevy to treat sickle cell disease. Developed by Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP), the one-time treatment harnesses gene… [Read More]

The US Food and Drug Administration approved two therapies for the treatment of sickle cell disease. One of the treatments, Casgevy, uses CRISPR gene-editing. The… [Read More]

Outcomes-based contract offerings available to both commercial payers and MedicaidLYFGENIA will be available through bluebird’s established national network of Qualified Treatment Centers beginning in Q1… [Read More]

The Food and Drug Administration on Friday approved two gene therapies to treat patients with sickle cell disease, a serious genetic blood disorder most common… [Read More]

– First-ever approval of a CRISPR-based gene-editing therapy in the U.S. –– Approximately 16,000 patients 12 years of age and older with severe sickle cell… [Read More]