SOHO 2024 | Gene therapy as the standard of care for patients with SCD who lack a matched sibling donor

So currently I think if you are lacking… if you fit the criteria to be eligible for a curative therapy and if you are lacking a matched sibling donor as a source for bone marrow transplant, my opinion is that gene therapy, now having FDA and EMA approved products, should be the standard of care. Why is that? Again, if there is a matched-sibling donor transplant, the outcomes are excellent and it’s a real cure because you replace the diseased marrow for healthy marrow. Historically, in registries in Europe and in the US, the outcomes with alternative donor transplants have been pretty poor.

Recently, since the FDA has approved two gene therapy solutions and gene addition, let’s say products, lovo-cel and then let’s say CRISPR product, exa-cel. We have access to an approved product which has shown to be safe in the short-term. So I think although some alternative donor transplant clinical trials show encouraging results, we are not there yet to consider them as a standard of care. So missing a matched-sibling donor, the second line therapy should be gene therapy because it has shown to be safe in the short-term. And maybe in the near future when alternative donor transplants show to be safe and effective as well, my opinion may change. But at this stage, after matched-sibling, gene therapy.