February 7, 2018

By rickspeer

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Gene Therapy – the future of drug development

In a significant move for the future of drug development, regulators recently approved a new gene therapy for one of the world’s largest drug companies GSK.  Strimvelis was granted approval for the treatment of a rare disease called ADA-SCID, which leave babies without a fully functioning immune system and vulnerable to infections.

This is the first approval for a genetic therapy granted to a large multinational drug company and will mark the beginning of many more genetic medicines from the ‘Big Parma’ group over the next few years.

// Gene therapies aim to cure a disease by introducing healthy copies of the gene into the patient, with the key benefit that it only needs to be given once and can therefore provide a potentially permanent cure. There are hundreds of inherited disorders from cystic fibrosis to blindness that can benefit from this, as well as the potential to utilise gene therapy within Oncology and other disease areas. //

The name Strimvelis was developed for GSK by Origin.   Strimvelis is strong/confident sounding with a re-assuring tone, suggesting hope, adaptability, superior results and better disease management.

Gene therapy is an area that will no doubt receive much of the spotlight in the future, from development through to pricing/re-imbursement, the impact that it will have on Big Pharma has the potential to be revolutionary.




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