FDA Panel Considers Gene Therapy for Hereditary Eye Disease
Title: FDA Panel Considers Gene Therapy for Hereditary Eye DiseaseCategory: Health NewsCreated: 10/10/2017 12:00:00 AMLast Editorial Review: 10/11/2017 12:00:00 AM
(Reuters) - Sarepta Therapeutics' shares jumped 60 percent on Tuesday after promising results from a gene therapy study positioned the specialty drugmaker as a potential leader in the market for Duchenne muscular dystrophy (DMD) treatments.
Sarepta Therapeutics on Tuesday released promising data from a small, early-stage trial of its experimental gene therapy treatment for Duchenne muscular dystrophy, showing that the drug significantly boosted an important muscle protein in three boys with the disease. Shares of Sarepta (Nasdaq: SRPT) soared Tuesday morning on the news, propelling its market cap from $6.9 billion to more than $11 billion briefly, before dropping back to $10.6 billion as of 11:15 a.m. The stock increase of 54 percent…
(Reuters) - Sarepta Therapeutics Inc shares jumped 60 percent on Tuesday after the drugmaker reported promising results from an early-stage study testing its gene therapy in patients with Duchenne muscular dystrophy (DMD).
(Reuters) - Sarepta Therapeutics Inc reported promising results on Tuesday from an early-stage study testing its gene therapy in patients with Duchenne muscular dystrophy (DMD), driving the company's shares up 60 percent.
An experimental gene therapy appeared to dramatically increase the production of a muscle-making protein in three young boys with Duchenne muscular dystrophy, a deadly and irreversible disease, according to results being presented by the drugmaker Sarepta Therapeutics at an investor event.
Two lead inventors of three of the four gene therapy treatments that Nationwide Children's Hospital has licensed to spinoffs have been hired away since fall and work full-time at big pharmaceutical companies. Children's counts those as wins.
Upregulation of microRNA-3129 suppresses epithelial ovarian cancer through CD44, Published online: 19 June 2018; doi:10.1038/s41417-018-0026-1Upregulation of microRNA-3129 suppresses epithelial ovarian cancer through CD44
Advances in cell and gene therapy are setting off a biotech manufacturing building boom. This South San Francisco-based company is one of the latest to open a new facility.
Enhanced expressions of FHL2 and iASPP predict poor prognosis in acute myeloid leukemia, Published online: 18 June 2018; doi:10.1038/s41417-018-0027-0Enhanced expressions of FHL2 and iASPP predict poor prognosis in acute myeloid leukemia
In conclusion, gene therapy methods for bone regeneration are systematically becoming more efficient and show proof-of-concept in clinically relevant animal models. Yet, on the pathway to clinical use, more investigation is needed to determine the safety aspects of the various techniques in terms of biodistribution, toxicity, and tumorigenicity.