VERTEX PHARMACEUTICALS

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• Alkeus says its eye therapy halts progression of genetic disease

  The inherited condition, known as Stargardt disease, has no available treatments and is a leading genetic cause of blindness in children and young adults, with an estimated 30,000 people affected in the United States and more than 150,000 worldwide.

  

• US FDA approvals bounce back in 2023, sparking hopes of a biotech recovery

  The agency approved several high-profile therapies such as Eli Lilly's obesity drug Zepbound and Eisai and Biogen's Alzheimer's treatment Leqembi. It also approved five gene therapies in addition to the 55 novel drugs, including a sickle cell disease treatment from Vertex Pharmaceuticals and CRISPR Therapeutics using the latter's innovative gene editing technology.

  

• US approves two gene therapies for sickle cell disease

  The U.S. Food and Drug Administration (FDA) on Friday approved two gene therapies for sickle cell disease, including one that is the first treatment in the United States based on the Nobel Prize-winning CRISPR gene editing technology. Casgevy, developed by partners Vertex Pharmaceuticals and CRISPR Therapeutics, and bluebird bio's Lyfgenia were approved for people aged 12 years and older.

  

• UK authorises gene therapy for blood disorders in world first

  Casgevy is the first medicine to be licensed that uses the gene-editing tool CRISPR, which won its inventors the Nobel Prize in 2020, Britain's Medicines and Healthcare products Regulatory Agency (MHRA) said.

  

• Vertex/CRISPR's gene editing therapy cost effective at $1.9 million

  The two companies are hoping to get approval for the world's first therapy based on Nobel prize-winning CRISPR technology, for two types of blood disorders - sickle cell disease (SCD) and transfusion-dependent beta thalassemia.

  

• Novel ex vivo engineered cell therapies to be developed by Vertex Pharma and Arbor Biotechnologies

  Under this new partnership, Vertex will receive rights to use Arbor’s technology to research and develop ex vivo engineered cell therapies towards Vertex’s goal of generating fully differentiated, insulin-producing hypoimmune islet cells for the treatment of type 1 diabetes, for next-generation approaches in sickle cell disease and beta thalassemia

  

• Vertex cystic fibrosis triple combo therapy succeeds in important trials

  The company had previously tested another experimental drug, VX-659, with the two-drug combination approved under the brand name Symdeko.

  

• Vertex cystic fibrosis drug gets expanded approval for use in infants

  The drug is currently approved in the United States to treat patients aged 2 and older whose CFTR gene has certain mutations.