https://www.isctglobal.org/telegrafthub/blogs/lauren-reville/2024...

https://www.isctglobal.org/telegrafthub/blogs/lauren-reville/2024/02/13/from-the-editor-guiding-potency-assurance-in-cell.

This new 2023 draft guideline for potency assurance provides a very useful and timely framework. Where the earlier guidance published in 2011 mainly focussed on potency tests, the 2023 document describes a broader approach, that can be flexible as needed, but remains stringent on core measures, such as definition of acceptable ranges for potency measures and weighing the relevance of data output from different measures. Who’d have “thunk” using FDA and flexibility in the same sentence a few decades ago? Here FDA appears true to its ambition of seeking to be conceptual and enabling of innovation in the world of the complex and ever evolving therapeutic modalities that CGT products comprise.

Alive vs Dead - I wonder if that's a measure that should be considered?

What was the market size 400 -500 kids? Unmet need? First rejected 2020 despite a 9-1 vote from the medical professionals.

1000 young lives- I actually paused here from my normal bluff and bluster- I truly hope this gets up - I feel for the families.


Below is the December 2023 Guidance from Matthew Klinker- January 2024- I think we provided requested data- March 2024, the FDA said refile.

This draft guidance provides recommendations for developing a science- and risk-based strategy to help assure the potency of a human cellular therapy or gene therapy (CGT) product. A potency assurance strategy is a multifaceted approach that reduces risks to the potency of a product through manufacturing process design, manufacturing process control, material control, in-process testing, and potency lot release assays. The goal of a potency assurance strategy is to ensure that every lot of a product released will have the specific ability or capacity to achieve the intended therapeutic effect.


The BLA resubmission is back on at the request of the FDA- the above appears sufficient?

Don't open the link below- it's for people who live in hope.

https://en.wikipedia.org/wiki/Hope,_British_Columbia

Reg