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The Thalassemia Channel on VJHemOnc is an independent medical education platform, supported with funding from Agios (Gold). Supporters have no influence on the production of content. The levels of sponsorship listed are reflective of the amount of funding given.
ASH 2024 | Selecting the best candidates with thalassemia for gene therapy and genome editing
Maria Domenica Cappellini, MD, University of Milan, Milan, Italy, comments on selecting the best candidates with thalassemia for gene therapy or genomic editing, emphasizing that a clear profile of the ideal patient is needed due to the high cost and complexity of the treatment. Prof. Cappellini highlights that an algorithm is not yet established to optimize this selection but suggests that young patients with a favorable clinical status and no comorbidities may be the most suitable candidates at the present moment. This interview took place at the 66th ASH Annual Meeting and Exposition, held in San Diego, CA.
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Transcript (AI-generated)
This is an important question. Gene therapy and genome editing are now ready, they have been approved both. Gene therapy is approved in the USA by the FDA and in Europe it is in a standby phase the gene therapy, whereas the genomic testing is approved. The cost is very high and of course dealing with the authorities we should be able to define the characteristics of, you want to say, the profile of the better patient to start with...
This is an important question. Gene therapy and genome editing are now ready, they have been approved both. Gene therapy is approved in the USA by the FDA and in Europe it is in a standby phase the gene therapy, whereas the genomic testing is approved. The cost is very high and of course dealing with the authorities we should be able to define the characteristics of, you want to say, the profile of the better patient to start with. We are not yet at the stage of having an algorithm or at least an algorithm was suggested a couple of years ago for gene therapy but now it needs to be revisited according to the further results obtained and that’s something the thalassemia community is keeping to do for the coming weeks or months.
From my point of view I think that those kinds of treatments which are curative, because one shot cures the disease if it works, should be preferentially used for young people, young patients, because they still are free of comorbidities, they are in a good clinical status and of course they may benefit for a long time. That’s my point of view. But the community is working on that and I hope that soon a kind of recommendation or guidance for selecting the patient will be available.
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Disclosures
Membership on an entity’s Board of Directors or advisory committees: Novo Nordisk, Vertex Pharmaceuticals, Pfizer, Silence, Sanofi-Genzyme, Pharmacosmos, Bristol Myers Squibb (Celgene), Vifor, Agios Pharmaceuticals, Inc.
