Rare Disease Focused Therapeutics M&A Soars & More — This Week in RARE Daily

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, May 3-9, 2024:

Rare Disease Focused Therapeutics M&A Soars in April
Two sizable acquisitions from Vertex Pharmaceuticals and ONO Pharmaceutical marked an otherwise slow April for rare disease focused therapeutics financings and deals, with total potential M&A deal values year-to-date up more than 300 percent compared to the same period in 2023, according to data from Dealforma and Global Genes.
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Regeneron’s Experimental Gene Therapy Restores Hearing to Normal Level in Deaf Child
Regeneron’s experimental gene therapy DB-OTO improved hearing to normal levels for two children born with profound genetic deafness due to variants of the otoferlin gene. The single intracochlear injection of DB-OTO the children received is the same approach used for cochlear implants, which is amenable for use in young infants.
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Researchers Develop Global Approach for Using AI to Identify Kids with Rare Diseases
Diagnosing children with rare, genetic diseases often involves misdiagnoses and prolonged diagnostic odysseys but researchers at the University of Wisconsin have developed a digital phenotyping algorithm using artificial intelligence that they say can identify kids likely to have a rare genetic disease.
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Pfizer Pauses Enrollment in DMD Gene Therapy Study 
Pfizer reported that a participant in its phase 2 DAYLIGHT study of its experimental gene therapy for the treatment of Duchenne muscular dystrophy died suddenly and that it is pausing enrollment in the crossover portion of its CIFFREO study.
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Marinus Cuts Staff by 20%

Marinus Pharmaceuticals announced it is laying off 20 percent of its staff as part of cost-cutting measures as it prioritizes efforts to continue commercialization of its drug Ztalmy to treat developmental and epileptic encephalopathies.
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