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Results For "rare-diseases"

115 News Found

India invites applications under PRIP scheme to drive pharma-medtech innovation
R&D | October 06, 2025

India invites applications under PRIP scheme to drive pharma-medtech innovation

Early stage projects of up to Rs. 9 crore eligible for up to Rs. 5 crore assistance and later stage projects of up to Rs. 285 crore eligible for up to Rs. 100 crore assistance


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


Merck to acquire US Biopharma firm SpringWorks Therapeutics for enterprise value of $3.4 billion
Biopharma | April 28, 2025

Merck to acquire US Biopharma firm SpringWorks Therapeutics for enterprise value of $3.4 billion

Planned acquisition will strengthen the presence of Merck Healthcare in the United States and expand reach of SpringWorks’ therapeutic innovations to more patients with rare tumors worldwide