Cystic fibrosis leader Vertex Pharmaceuticals is diving into a new of Duchene Muscular Dystrophy with the acquisition of Exonics Therapeutics and an expansion of its two-year-old collaboration with CRISPR Therapeutics. He said Vertex has a proven track record when it comes to developing important therapies and added that Exonics is excited to combine efforts to “potentially develop a safe and efficacious one-time treatment for severe neuromuscular diseases.”“These transactions are highly aligned with our strategy of investing in scientific innovation to create transformative medicines for people with serious diseases.
Gene editing approach: Various
Description: In vivo CRISPR/Cas9-based candidate in development for patients suffering from DM1, a rare genetic disease caused by a trinucleotide repeat expansion in the DMPK gene. CRISPR Therapeutics Announces Pricing of Public Offering of Common Shares. All rights reserved. In June 2019, CRISPR Therapeutics and Vertex expanded their collaboration and entered into an exclusive licensing agreement to discover and develop gene editing therapies for the treatment of Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1). It helps that CRISPR Therapeutics has built its pipeline to ward off some of the uncertainty facing CRISPR gene editing. CRISPR Therapeutics Announces the Build-Out of New Cell Therapy Manufacturing Facility But, what might investors expect in 2024?Stock Advisor launched in February of 2002.
We are continuing to build a toolbox of small molecule and nucleic acid technologies and capabilities that will allow us to drive scientific innovation to produce transformative medicines for a broad portfolio of diseases,” Leiden said in a statement.© 1985 - 2020 BioSpace.com.
The genetic disease causes a progressive loss of muscle strength attributable to a loss of a protein called dystrophin, which normally protects muscle fibers from breaking down. Founded in 1993 by brothers Tom and David Gardner, The Motley Fool helps millions of people attain financial freedom through our website, podcasts, books, newspaper column, radio show, and premium investing services.As the survey of possibilities makes clear, investors will have no shortage of updates and potential catalysts to watch in the next five years. CRISPR Therapeutics (CRSP) is developing its lead gene-editing candidate for thalassemia and sickle cell disease. Approximately 15,000 U.S. patients are affected with Duchenne, with a total of 300,000 patients worldwide. Vertex Pharmaceuticals is paying $420 million upfront to go after the muscular dystrophy market.
Here are possible scenarios for the distinct opportunities known to investors today:All things considered, CRISPR Therapeutics finds itself with an enviable balance sheet and pipeline as it approaches the end of 2019. That insulates the business and shareholders against a scenario in which Wall Street writes off the entire pipeline should the first drug development efforts stall. If some clinical applications fail, then other approaches could still work. Jun 25, 2020. Jun 29, 2020. Patients with congenital DM1 have severe generalized weakness at birth (hypotonia), often causing complications with breathing and early death.
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