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Results For "rare-disease"

212 News Found

Moderna opens manufacturing and R&D facility in UK
News | September 27, 2025

Moderna opens manufacturing and R&D facility in UK

The Moderna Innovation and Technology Centre in Harwell, Oxfordshire is now fully operational and Moderna is licensed to supply British-made COVID-19 vaccines to the UK population


Datavant partners with Indegene to power clinical trial recruitment
Digitisation | September 21, 2025

Datavant partners with Indegene to power clinical trial recruitment

Collaboration matches more eligible patients, including those with rare and complex diseases, to appropriate clinical trials


FDA approves Sanofi’s BTK inhibitor for immune thrombocytopenia
Drug Approval | September 02, 2025

FDA approves Sanofi’s BTK inhibitor for immune thrombocytopenia

ITP is a disease of complex immune dysregulation leading to low platelet counts, bleeding, and reduced quality of life


Sarepta advances siRNA collaboration and sale of arrowhead equity investment
News | August 19, 2025

Sarepta advances siRNA collaboration and sale of arrowhead equity investment

Sarepta has also entered into an agreement with Arrowhead to transfer 2,660,989 shares of Arrowhead common stock in satisfaction of $50 million of the company’s previously announced $100 million milestone payment obligation


ImCheck’s ICT01 receives FDA ODD for treatment of acute myeloid leukemia
Clinical Trials | July 21, 2025

ImCheck’s ICT01 receives FDA ODD for treatment of acute myeloid leukemia

Clinical data showing unprecedented remission rates in newly diagnosed AML patients support advancing ICT01 into pivotal trials


Capricor receives FDA’s CRL for Deramiocel BLA for duchenne muscular dystrophy
News | July 15, 2025

Capricor receives FDA’s CRL for Deramiocel BLA for duchenne muscular dystrophy

Capricor’s BLA for Deramiocel received Priority Review in March 2025


Sanofi acquires US biopharma firm Blueprint Medicines for $9.1 billion
News | June 02, 2025

Sanofi acquires US biopharma firm Blueprint Medicines for $9.1 billion

Adds fast-growing and only approved medicine for advanced and indolent systemic mastocytosis to the Sanofi portfolio