In 2023, biotech experts working on gene and cell therapy and similar cutting-edge treatments will be bombarded by a range of opportunities.
Some therapies are expected to get FDA approval this year. Roctavian, the improved bone marrow transplant omidubicel, and SRP-9001, which might be the first gene treatment for the uncommon condition Duchenne’s muscular dystrophy, are some of the possibilities.
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“There is a lot of pressure on the FDA from patient advocacy groups to approve the therapy as soon as possible since DMD is not reversible and SRP-9001 can only prevent further deterioration. This will be a very closely watched regulatory decision, especially given that results from the phase 3 confirmatory trial are due to drop only a few months after,” informs Fady Riad, CEO of Centurion Life Sciences, a consulting firm.
Exa-cel, a gene-edited cell treatment for beta-thalassemia and sickle cell disease, will also be of interest. Early in 2023, the therapy’s creators, Vertex and CRISPR Therapeutics, hope to finish the application for approval.
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The high costs associated with the therapies may surface as a challenge in the process.
Still, gene therapies will be necessary for 2023 and the next few years.