Best in Biotech 3 Oct 2024 Six biotechs driving progress in Duchenne muscular dystrophy In this article, we take a look at six clinical-stage biotechs that are fostering R&D in the Duchenne muscular dystrophy therapeutic space. October 3, 2024 - 8 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
In Depth 3 May 2024Can emerging new treatments lead the way to cure Duchenne muscular dystrophy? Delve into the new treatment approaches to Duchenne muscular dystrophy, a challenging disease for those suffering from it, and research. May 3, 2024 Share WhatsApp Twitter Linkedin Email
Best in Biotech 16 Apr 2024 Seven biotech companies climbing their way up in Colorado We take a look at seven biotechs in Colorado that have secured funding in recent years, to make headway with their pipeline candidates. April 16, 2024 - 8 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
In Depth 4 Apr 2024 Seven diseases CRISPR technology could cure Here are some of the diseases that scientists aim to tackle using CRISPR technology, testing its possibilities and limits as a medical tool. April 4, 2024 - 11 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
News and Trends 22 Aug 2022 Santhera and ReveraGen announce first patient with Becker muscular dystrophy dosed in FDA-funded pilot study The first patient has been dosed in a phase 2 pilot study looking at a treatment for muscular dystrophy. Santhera Pharmaceuticals and ReveraGen BioPharma made the announcement that vamorolone is to be assessed in patients with Becker muscular dystrophy (BMD). The study is being funded by the U.S. Food and Drug Administration (FDA). Eric Hoffman, […] August 22, 2022 - 3 minutesmins - By Liza Laws Share WhatsApp Twitter Linkedin Email
News and Trends 29 Jul 2022 Sarepta looks for quick approval for Duchenne muscular dystrophy gene therapy treatment U.S. company Sarepta Therapeutics, Inc. said it intends to submit a Biologics License Application (BLA) seeking accelerated approval for SRP-9001 (delandistrogene moxeparvovec) to treat ambulant individuals with Duchenne muscular dystrophy. SRP-9001 is an investigational gene therapy for Duchenne being developed in partnership with Roche. “We are delighted to confirm that based on the feedback we […] July 29, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 7 Jun 2019 CRISPR Therapeutics and Vertex Seal Deal Worth up to €890M The US giant Vertex Pharmaceuticals is expanding an existing collaboration with the Swiss company CRISPR Therapeutics, paying up to €890M ($1B) to develop treatments for genetic diseases causing muscle weakness using the gene editing tool CRISPR/Cas9. The deal includes €155M ($175M) paid upfront, and potential future payments based on achieving developmental milestones. In exchange, Vertex […] June 7, 2019 - 2 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 28 Jun 2018 Muscular Dystrophy Drug Fails in Phase 2 Trial The stock of Summit Therapeutics has plunged by 80% after announcing that its drug candidate for Duchenne muscular dystrophy failed to meet the primary and secondary endpoints of a Phase II clinical trial. Duchenne muscular dystrophy is a genetic disease that causes progressive muscle deterioration in children born with the disease. The British-American company Summit […] June 28, 2018 - 2 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
News and Trends 27 Feb 2018 This Small Molecule Protects the Muscles of Patients With Duchenne Muscular Dystrophy Summit Therapeutics’ treatment for the devastating disease, Duchenne muscular dystrophy, reduces inflammation, which could protect muscle fibers from damage. Summit Therapeutics focuses on the development of treatments that can help patients suffering from diseases that currently lack options. The biotech has reported Phase II results for its drug for the genetic disease, Duchenne muscular dystrophy […] February 27, 2018 - 3 minutesmins - By Alex Dale Share WhatsApp Twitter Linkedin Email
News and Trends 5 Jul 2017 Cutting Through the BS in Biopharma: How To Spot Hype Faced with the oft-quoted figure that 90% of clinical trials end in failure, how can patients, investors, and readers be spared more disappointment? After discussing failures in biotech at Refresh earlier in June, I headed to Copenhagen last week for the European Conference for Science Journalists to cajole a panel into discussing how some of these stories feature […] July 5, 2017 - 4 minutesmins - By Evelyn Warner Share WhatsApp Twitter Linkedin Email
News and Trends 28 Jun 2017 Swiss Scientists Have Designed New Proteins to Treat Muscular Dystrophy Researchers at the University of Basel have designed two proteins that can reverse congenital muscular dystrophy and increase the lifespan of mice. Congenital muscular dystrophy (CMD) englobes several genetic diseases that cause muscle weakness from birth. As patients age, the disease becomes more severe and affects breathing, often leading to death before adulthood. Unfortunately, there […] June 28, 2017 - 2 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
News and Trends 22 Jun 2017 French Nonprofit Partners with Big American Name to Advance a Gene Therapy for Muscular Dystrophy Noble Généthon has teamed up with ignominious Sarepta to develop a gene therapy for the Duchenne variety of the muscle wasting disease. Dedicated to rare diseases since 1990 and more recently to gene therapy, Généthon is one of very few not-for-profit companies in European biotech. Though it has not yet brought a drug to market, it is […] June 22, 2017 - 3 minutesmins - By Evelyn Warner Share WhatsApp Twitter Linkedin Email