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. 𝐀𝐭𝐚𝐫𝐚 𝐁𝐢𝐨 𝐲𝐞𝐬𝐭𝐞𝐫𝐝𝐚𝐲 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 (𝐄𝐁𝐕+ 𝐏𝐓𝐋𝐃) 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 𝐧𝐨 𝐅𝐃𝐀-𝐚𝐩𝐩𝐫𝐨𝐯𝐞𝐝 𝐭𝐡𝐞𝐫𝐚𝐩𝐢𝐞𝐬 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. 𝐓𝐡𝐞 𝐏𝐃𝐔𝐅𝐀 𝐚𝐜𝐭𝐢𝐨𝐧 𝐝𝐚𝐭𝐞 is 16/11/24

PTC Therapeutics last week submitted a BLA for their gene therapy, Upstaza™ (eladocagene exuparvovec), targeting aromatic L-amino acid decarboxylase (AADC) deficiency. The FDA accepted this application and granted it Priority Review status. 𝐓𝐡𝐞 𝐭𝐚𝐫𝐠𝐞𝐭 𝐚𝐜𝐭𝐢𝐨𝐧 𝐝𝐚𝐭𝐞 𝐬𝐞𝐭 by 13/10/24.

𝐖𝐡𝐚𝐭 𝐁𝐋𝐀 𝐬𝐮𝐛𝐦𝐢𝐬𝐬𝐢𝐨𝐧𝐬 𝐚𝐫𝐞 𝐜𝐚𝐭𝐜𝐡𝐢𝐧𝐠 𝐲𝐨𝐮𝐫 𝐢𝐧𝐭𝐞𝐫𝐞𝐬𝐭?”

Source: Spencer Knight/LinkedIn