Plus   Neg

Pfizer's Gene Therapy Candidate For Duchenne Muscular Dystrophy Receives Fast Track designation

Pfizer Inc. (PFE) said the company's investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the FDA. The designation was granted based on data from the phase 1b study that indicated that the intravenous administration of PF-06939926 was well-tolerated during the infusion period and dystrophin expression levels were sustained over a 12-month period.

PF-06939926 is an investigational, recombinant adeno-associated virus serotype 9 capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The company started the phase 1b multi-center, open-label, non-randomized, ascending dose study of a single intravenous infusion of PF-06939926 in 2018.

"We are working to advance our planned Phase 3 program as quickly as possible," said Brenda Cooperstone, Chief Development Officer, Rare Disease, Pfizer Global Product Development.

For comments and feedback contact: editorial@rttnews.com

Business News

Editors Pick
Amazon said Wednesday that free one-hour grocery pickup is now available to prime members at all U.S. Whole Foods Market locations. Previously, the prime members could pick up Whole Foods orders only at select stores. J.C. Penney said it is near to exiting Chapter 11 ahead of the busy December 2020 holiday season. The beleaguered retailer filed a draft asset purchase agreement towards finalization of the acquisition by Brookfield Asset Management, Inc. and Simon Property Group, Inc. They are expected to buy substantially all of J.C. Penney's retail and operating assets in a combination of cash and debt. Kohl's is launching a new specialty athleisure brand, FLX, in March 2021. The new private label with offerings for both men and women will be available in select stores and online at Kohls.com. Athleisure, a style of clothing worn as athletic apparel but also suitable for casual, everyday wear, is in high demand at present with majority of people across the world are homebound amid coronavirus.
Follow RTT