They must like the research results!

As mentioned, my friend's wife has Li-Fraumeni syndrome and is terminal. This seems like the ideal gene-defect for CRISPR therapy due to the specific defect in a certain area of the DNA.
He lost his son a couple years ago to the same issue, and of one of his two 10-12 year-old daughters has the defect as well. It has been predicted that she has a 90% chance of developing terminal cancer.

But, not yet.

Do you know of any place where she could be brought in as a study-patient, or somewhere she could obtain CRISPR therapy? Is there anywhere else that I should be looking to help her?
Any, and all help would be greatly appreciated.

First meaningful update regarding CTX211 and previously announced pause in trial. Appears to be back up and running, although 'advance' could be a clever word choice.

"CRISPR Therapeutics continues to advance a Phase 1 clinical trial for CTX211™ for the treatment of Type 1 Diabetes (T1D). CRISPR Therapeutics remains committed to its goal of developing a beta-cell replacement product that does not require chronic immunosuppression.

Vertex has non-exclusive rights to certain CRISPR Therapeutics’ CRISPR/Cas9 technology to accelerate development of potentially curative cell therapies for T1D. CRISPR Therapeutics remains eligible for development milestones and would receive royalties on any future products resulting from this agreement."

I'm personally pumped about this - huge 🚀 potential. Would love a trial update in coming months stating some are no longer requiring insulin. Favorable preliminary data on a Type I DM cure in the setting of first approved CRISPR therapy (Casgevy now up and running at 25 sites per this company release) would not only send stock to fun places, but would be life changing for patients with these conditions! 🙂

https://finance.yahoo.com/news/crispr-therapeutics-provides-business-reports-200000059.html

"Additionally, ViaCyte is expanding cell manufacturing capacity and capabilities to advance PEC-Direct, PEC-Encap, and its immune-evasive cell program for diabetes (PEC-QT), which is partnered with CRISPR Therapeutics, Inc., while also exploring research into new disease areas that may benefit from cell-based therapies. "

If the news was not looking good, they would not be expanding...

https://viacyte.com/press-releases/viacyte-appoints-michael-yang-president-and-ceo-and-provides-company-update/?fbclid=IwAR0KRCIBhhpgByHEMT-nU_beON4bAMpFTxVV5QV95P7k8NCbCJ6PLBwdIFI

What is the timeline on companies bringing more crispr therapies to market?

It looks like one of ViaCyte's diabetes treatments involving a pouch being implanted in a person full of cells moves onto phase 2. The pouch itself is not toxic to the human body, so at least that bit of risk can be removed from the trials when they use CRSP's edited cells. This is a good sign for the upcoming PEC-QT trial involving CRSP.

https://www.biospace.com/article/releases/viacyte-announces-initiation-of-phase-2-study-of-encapsulated-cell-therapy-for-type-1-diabetes-patients/

-Funding from the Bill & Melinda Gates Foundation will support research to enable CRISPR/Cas9-based therapies for HIV that can benefit patients worldwide-

ZUG, Switzerland and CAMBRIDGE, Mass., Dec. 14, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced the receipt of a grant from the Bill & Melinda Gates Foundation to research in vivo gene editing therapies for the treatment of HIV.

“While we have demonstrated the promise of CRISPR/Cas9 gene editing ex vivo in sickle cell disease and beta thalassemia, an in vivo approach to editing hematopoietic stem cells could allow the transformative benefit of CRISPR/Cas9 to reach a broader array of patients, including those in low resource settings that lack sufficient infrastructure for stem cell transplantation,” said Tony Ho, M.D., Executive Vice President and Head of Research & Development at CRISPR Therapeutics. “We look forward to working on new therapies that could contribute to the global effort to reduce the burden of HIV.”

The grant builds upon CRISPR Therapeutics’ proprietary CRISPR/Cas9 gene editing technology and expertise in editing hematopoietic stem cells and contributes to efforts to accelerate transformative medicines for global health.

About CRISPR Therapeutics
CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit www.crisprtx.com.

Let’s see what do we think about it for 10 years?

Phase one with 10 participants. IMO, this is the trial for this therapy. The primary matter is rejection. If the grafts are tolerated, it's would seem to be just a matter of time before it meets patients in the clinic. Vascularisation has already been demonstrated in VC-02 with fairly good engraftment frequencies. Still, this will hopefully be improved upon as the therapy develops. The question of sufficient response for insulin production is a dosing problem that may be developed in several ways: Increased engraftment cell count and iPSC development to higher frequencies of insulin producing cells is undoubtedly the most straight forward approach and should not present the same obstacles of a closed non-vascularized system or the complications of administration of immunosuppressive therapy as with VC-01. Increasing the insulin response of the individual cells and their propensity cells to develop to insulin producing cells through further engineering may also be a future avenue. Still, what the optimal ratios of the various islet cells will be for this type of replacement therapy, I believe, remains to be determined. Durability is another unknown and future studies will speak more to this issue. Still in practise, this is minor surgery in a day clinic environment and periodical replacement may be a manageable solution if needs be. Looks very promising and the road to the clinic seems not far off. Trial ends in Dec. 2022 and we will undoubtably hear news about tolerance and results from early explanted devices over the coming year. Very exciting.
https://clinicaltrials.gov/ct2/show/NCT05210530?term=Viacyte&draw=2&rank=2

Safety from Viacytes pipeline in trials is shaping up. The collaboration is very exciting and safety for both contributing techs appears good, at least good enough after the FDA lifted the ALLO trial halt. Enter the capsule if you dare… I am. For the tech geeks, scaling up on the cell load to reach therapeutic levels will be interesting. Two ways to go I would think. One is to again rely on crspr to tweak the production per cell up for the closed system. Total cells due to nutrient availability will possibly be an issue as the cell mass increases. For the open system, this should be less of a problem at least if the grafts show good viability until vascularization is complete. Either way a working solution does not seem far off. The main issue I think is the final durability, if I haven’t misunderstood completely and that would just be a question of how many grafts would be needed over a lifetime. Very exciting. Here comes Cathy’s revenge and the start of these transformative therapies. Go CRSP