Arrowhead and Sarepta link for rare genetic disease treatments
Pharmaceutical Technology
NOVEMBER 27, 2024
Arrowhead has entered a worldwide licensing and partnership agreement with Sarepta Therapeutics for rare genetic disease treatments.
Pharmaceutical Technology
NOVEMBER 27, 2024
Arrowhead has entered a worldwide licensing and partnership agreement with Sarepta Therapeutics for rare genetic disease treatments.
XTalks
DECEMBER 4, 2024
These efforts aim to fast-track commercialization and out-licensing agreements in one of the world’s most promising pharmaceutical markets. CAMP4’s RNA-based therapies focus on genetic diseases like urea cycle disorders, while Rapport’s small molecules aim to address epilepsy, pain and bipolar disorder.
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Astellas chief strategy officer Naoki Okamura said: “Gene therapy is the cornerstone of Astellas’ Primary Focus, Genetic Regulation; our goal is to bring new transformative treatment options to patients living with serious genetic diseases and limited treatment options.
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In 2018, the company also received approval for Orilissa (elagolix), which it licenses to AbbVie, for the treatment of endometriosis. It also received the FDAs rare pediatric disease priority review voucher along with the approval, which will help expedite the agencys assessment of another application. billion in revenue by 2024.
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With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.
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