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Spencer Knight: CGT approvals are ARRIVING, Here’s 3 BLAs to keep your eye on
May 22, 2024, 11:55

Spencer Knight: CGT approvals are ARRIVING, Here’s 3 BLAs to keep your eye on

Spencer Knight, Principal Business Consultant of Cell and Gene Therapy, shared on LinkedIn:

“CGT approvals are ARRIVING,
Here’s 3 BLAs to keep your eye on

1.ย ๐€๐ญ๐š๐ซ๐š ๐๐ข๐จย ๐ฒ๐ž๐ฌ๐ญ๐ž๐ซ๐๐š๐ฒ submitted a Biological Licence Application (BLA) to the FDA for tabelecleucel, proposed as a monotherapy for treating Epstein-Barr virus positive post-transplant lymphoproliferative disease (๐„๐๐•+ ๐๐“๐‹๐ƒ) in patients aged 2+ and have undergone at least one prior therapy.

For solid organ transplant patients, prior therapy includes chemotherapy unless deemed inappropriate. Notably, there are currently ๐ง๐จ ๐…๐ƒ๐€-๐š๐ฉ๐ฉ๐ซ๐จ๐ฏ๐ž๐ ๐ญ๐ก๐ž๐ซ๐š๐ฉ๐ข๐ž๐ฌ for this condition.

Autolus Therapeutics‘ submitted a BLA for obe-cel as a treatment for relapsed/refractory B-cell ALL. The submission was supported by data from the phase 1b/2 FELIX trial, results included a high complete response rate and minimal residual disease negativity. ๐“๐ก๐ž ๐๐ƒ๐”๐…๐€ ๐š๐œ๐ญ๐ข๐จ๐ง ๐๐š๐ญ๐ž is 16/11/24

PTC Therapeuticsย last week submitted a BLA for their gene therapy,ย Upstazaโ„ขย (eladocagene exuparvovec), targeting aromatic L-amino acid decarboxylase (AADC) deficiency. The FDA accepted this application and granted it Priority Review status. ๐“๐ก๐ž ๐ญ๐š๐ซ๐ ๐ž๐ญ ๐š๐œ๐ญ๐ข๐จ๐ง ๐๐š๐ญ๐ž ๐ฌ๐ž๐ญ by 13/10/24.

๐–๐ก๐š๐ญ ๐๐‹๐€ ๐ฌ๐ฎ๐›๐ฆ๐ข๐ฌ๐ฌ๐ข๐จ๐ง๐ฌ ๐š๐ซ๐ž ๐œ๐š๐ญ๐œ๐ก๐ข๐ง๐  ๐ฒ๐จ๐ฎ๐ซ ๐ข๐ง๐ญ๐ž๐ซ๐ž๐ฌ๐ญ?”

Spencer Knight: CGT approvals are ARRIVING, Here's 3 BLAs to keep your eye on

Source: Spencer Knight/LinkedIn