Big Boost in Rare Disease Financings & More — This Week in RARE Daily

March 14, 2024

This Week in RARE Daily is a new 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, March 8-14, 2024:

February Roars with $3.2 Billion Raised by Public Rare Disease Therapeutics Developers
The rare disease industry started with a bang in 2024 when rare disease drug developers raised $3.2 billion, coupled with recent IPOs, licensing agreements, and acquisitions.

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As Spending on Cell and Gene Therapies Grows, Challenges Remain
Despite a a 38 percent increase last year in cell and gene therapies spending, the future is uncertain for these therapies and the sustainability of the cell and gene therapy sector.

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Researchers Take Aim at DMD Therapies to Make Case for Timely Completion of Confirmatory Trials
While advances have been made in genetically targeted therapies for Duchenne muscular dystrophy in the last decade, delayed confirmatory trials and high cost of the therapies have hindered access to care for DMD patients.

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AstraZeneca to Acquire Amolyt Pharma
AstraZeneca is increasing its pipeline of therapeutic peptides targeting rare endocrine diseases, and expects the transaction to close by the end of the third quarter of 2024.

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Bluebird Bio Enters First Outcomes-Based Agreement with Medicaid for SCD Gene Therapy
Bluebird Bio’s agreement with Michigan’s Medicaid program will expand cell-based gene therapy to treat sickle cell disease. Approximately 50 percent of individuals living with sickle cell disease in the United States are insured by Medicaid.

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