Gene editing is evolving rapidly – now there is prime editing that can add sequences to cells to help them repair faulty sequences – taking control of our genetic futures with genome intervention
From here:
David Liu: A Master Class on the Future of Genome Editing (substack.com)
“David Liu is an gifted molecular biologist and chemist who has pioneered major refinements in how we are and will be doing genome editing in the future, validating the methods in multiple experimental models, and establishing multiple companies to accelerate their progress.
The interview that follows here highlights why those refinements beyond the CRISPR Cas9 nuclease (used for sickle cell disease) are vital, how we can achieve better delivery of editing packages into cells, ethical dilemmas, and a future of somatic (body) cell genome editing that is in some ways is up to our imagination, because of its breadth, over the many years ahead.”
As always, there is a positive ad a negative aspect to this leap in capability.
There is a full transcript to the 48 minute interview on the SubStack link plus lots of diagrams and links to relevant pieces.
Here’s a taste of what is in the interview.
“David Liu (10:41):
“So you can basically take any DNA sequence of up to now hundreds of base pairs and replace it with any other sequence of your choosing of up to hundreds of base pairs.”
Onwards!
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Pity they decided to experiment on global population first though, eh?
Concepts are one thing, reality not keeping up? - can they lean up their manufacturing processing (this is on Hedley's substack)
Hedley Rees wrote this earlier: Kymriah GMP issues in NJ led to FDA letter for Novartis
This is potentially a watershed event:
The US FDA has highlighted GMP deficiencies in the production of CAR-T therapy Kymriah at Novartis’ Morris Plains plant.
In November and December last year, the United States Food and Drug Administration (FDA) inspected the Morris Plains, New Jersey facility, resulting in it issuing a Form 483 [inspection report] with an undisclosed number of observations to Novartis. This is an extract:
Among the deviations, the Agency found Novartis failed to establish laboratory controls, particularly citing sampling plan and test procedure issues relating to freezing bags – or ‘Cryobags’ – the primary container for Kymriah.
“[The plans] are not appropriate to assure that Cryobags are “free of…particulate matter” as required by your acceptance criteria,” the Agency states. “Between December 2018 and the date of the inspection, you identified approximately 100 batches of Kymriah contaminated with foreign particulate matter, such as wood, cellulose, brass, and steel.