Improved Gene Editing System Makes Gene-Sized Edits & More — This Week in RARE Daily

June 13, 2024

This Week in RARE Daily is a feature from Global Genes where you can get a quick rundown of the top 5 headlines in the rare disease space from our editorial staff. Here are top stories from this past week, June 7 – 13, 2024:

Improved Gene Editing System Makes Gene-Sized Edits

Scientists at the Broad Institute of MIT and Harvard have improved a gene-editing technology that they say is now capable of inserting or substituting entire genes in the genome in human cells, opening the way for therapeutic applications that can address diseases caused by varying genetic mutations with a single therapy.

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Rare Disease Therapeutics M&A Maintains Momentum in May

Rare disease focused M&A transactions maintained their momentum in May as total potential deal values year-to-date were 192 percent above the same period in 2023, according to data compiled by Dealforma and Global Genes.

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Pfizer Gene Therapy for Ambulatory Boys with DMD Fails Late-Stage Study

Pfizer reported that the CIFFREO late-stage trial of its experimental mini-dystrophin gene therapy did not meet its primary endpoint of improvement in motor function among boys 4 to 7 years of age with Duchenne muscular dystrophy compared to placebo.

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Blood Test Could Prevent Death of Children with Hereditary Heart Condition

Researchers in England have developed a blood test they say could identify children with the potentially fatal heart condition hypertrophic cardiomyopathy.

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Cranbury Pharmaceuticals Receives FDA Approves Cranbury’s Generic Version of Emflaza for DMD

The U.S. Food and Drug Administration approved Tris Pharma subsidiary Cranbury Pharmaceuticals’ Abbreviated New Drug Application for the first generic version of Emflaza oral suspension  for the treatment of Duchenne muscular dystrophy.

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