A novel second-generation approach to achieve a safe, durable, and stable transgene expression to overcome the challenges of factor VIII replacement therapies
RNA, cell & gene therapies, artificial intelligence, CRISPR and oncology-related deals dominate the landscape
Casimir further differentiates Emmes’ industry-leading rare disease research capabilities
Nulibry is approved by the U.S. Food and Drug Administration (USFDA) to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening paediatric genetic disorder
It is a Phase 3 ready anti-myostatin adnectin for Spinal Muscular Atrophy (SMA).
With ValGenesis VLMS, change control driven validation activities are reduced from weeks to mere hours, and the company gains faster access to detailed metrics that help reduce costs and cycle time
New subsidiaries to be established in Malaysia, Taiwan, Singapore, and Hong Kong
Sanofi unveiled a new bold and unifying corporate brand that supports the modernization and transformation the company launched in December 2019
The FDA grants ODD status to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US
This surpasses the number of approvals supported in 2020
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