Crispr Therapeutics’ allogeneic Car-T projects might still be disappointing investors, but at least its sickle cell and beta-thalassaemia asset exa-cel keeps delivering the goods. The latest data on the Vertex-partnered ex-vivo gene-edited therapy, presented at the EHA meeting over the weekend, continue to suggest that it could represent a functional cure.
The companies previously said they would file exa-cel with regulators in late 2022 and, assuming the therapy is endorsed by the FDA and payers, it could be bad news for Crispr and Vertex’s many rivals. The gene-edited sickle cell space looks particularly crowded, especially for a disorder that is relatively rare.
Like other ex-vivo projects, exa-cel, previously known as CTX001, has drawbacks, including the need for harsh pre-conditioning regimens. This leaves room for oral competitors, such as Global Blood’s GBT021601, incremental data on which also featured at EHA.
However, another oral player had a stumble: Fulcrum Therapeutics’ stock dropped 39% on Friday after its EHA update comprised just three of six patients treated with FTX-6058. The group said the other three patients were not evaluable because of non-adherence or protocol deviation.
Bluebird flies, for now
The march of exa-cel might also put a dampener on Bluebird Bio, whose beti-cel (Lentiglobin) looks to be heading for FDA approval in beta-thalassaemia following its unanimously positive adcom vote on Friday.
Exa-cel looks slightly better on efficacy than beti-cel; the FDA has also raised concerns about a risk of cancer with Bluebird’s project, an issue that Crispr and Vertex’s contender has so far avoided.
Bluebird is also behind in sickle cell disease. A filing of lovo-cel, as the project is known in this indication, is not expected until early 2023 – a timeline that the company has stuck to despite various clinical holds.
Climbing
The latest update on Crispr and Vertex's exa-cel comes from 44 patients in the Climb Thal-111 study in transfusion-dependent beta-thalassaemia, and 31 patients in the Climb SCD-121 study in sickle cell disease.
In beta-thalassaemia, 42 out of 44 patients are now transfusion free, with follow-up ranging from 1.2 to 37.2 months. This gives an efficacy of 95%, slightly ahead of the 89% seen with Bluebird’s beti-cel.
In sickle cell, all 31 patients treated with exa-cel are free of vaso-occlusive crises, with follow-up of 2.0-32.3 months. Patients also showed a mean increase in haemoglobin levels, boosted by an increase in foetal haemoglobin.
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June 13, 2022 at 07:00PM
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EHA 2022 – Crispr still looks bloody good - Vantage
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