GENE THERAPY

More in GENE THERAPY

• Study gives more insight into understanding cancer risk in gene therapies

  The current study looked at samples from six sickle cell disease kids who were getting gene therapy as part of a large clinical trial at Boston Children's Hospital. The study gathered together an international team of scientists to examine the genetic changes in patients' stem cells before and after gene therapy and to compare the cells that were transformed with the therapy to those that were not.

  

• Study sheds more light on advances, challenges in gene therapy for rare diseases

  Juan Bueren, from Centro de Investigaciones Energeticas Medioambientalies y Tecnologicas (CIEMAT), and President of the European Society for Gene and Cell Therapy (ESGCT), and Alberto Auricchio, from Telethon Institute of Genetics and Medicine (TIGEM), and Vice-President of the European Society for Gene and Cell Therapy, coauthored the article titled "Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases."

  

• AstraZeneca to invest $245 mln in Cellectis to boost gene therapy prospects

  That includes an $80 million equity investment, which will be followed by another $140 million in early 2024, which will give AstraZeneca a stake of about 44 per cent in the French company.The Anglo-Swedish pharma company said it had exclusive rights to 25 genetic targets, of which up to 10 candidate products could be explored for development.

  

• US FDA approves BioMarin's gene therapy for hemophilia A

  Gene therapies use a deactivated virus to deliver the needed genes and the immune system would recognise and eliminate a second dose of the therapy.

  

• First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids

  The FDA OK'd the treatment only for children ages 4 and 5, based on study results showing the therapy helped produce a protein needed for muscle growth, which is missing in boys with the condition.

  

• Lauras Labs-IIT Kanpur to bring novel gene therapy assets to market

  IITK has a proven record of being a flag bearer for advancing research in India and this collaboration takes it to the next level, he added.

  

• IIT-K tops in innovation with Gene therapy, smart watch

  ‘Gene Therapy’ is a way to replace the faulty gene with a functional version of the gene to treat such disorders. This marks the first time that a gene therapy related technology has been developed and transferred from an academic institution to a company in India.

  

• UniQure to sell royalty interest in blood disorder therapy for up to $400 mln

  Australian drugmaker CSL Ltd acquired exclusive global rights to Hemgenix in June 2020 from uniQure, which would retain the rights to future milestones totaling up to $1.5 billion and maintain an interest in the therapy's royalties.

  

• FDA reviewers raise doubts over data on Sarepta's gene therapy

  Sarepta's mid-stage trial data showed it helped patients make a mini version of the dystrophin protein needed to keep muscle cells intact, and the company is also conducting a late-stage study, with data expected in September. The advisory committee's vote on Friday and an eventual decision by the FDA is seen as a test of the agency's accelerated approval pathway for gene therapies.

  

• Bluebird bio submits US marketing application for blood disorder gene therapy

  The announcement comes nearly a month after the company said it would miss its goal to submit the application by the end of March for regulatory approval of lovo-cel, its gene therapy. Bluebird said on Monday its application to the health regulator included a request for priority review, which if granted would reduce the review period to six months from the standard ten months.

  

• FDA's Marks hopes to align global regulators to boost gene therapy

  Health regulators including the FDA and the European Medicines Agency have different rules for cell and gene therapies that make it harder to get treatments on their markets at similar times.