2018 ASGCT

I will be attending the 2018 ASGCT and look forward to hearing the latest developments (poster or oral talks) about cell and gene therapy. I am excited most by Sangamo Biosciences and Homology Medicines. I hope to get a feel for the science hands-on and talk to the people actually doing the work. I will try to post my thoughts on this blog and maybe write something after the conference. Its crazy how hot the field is right now and its great to be part of this.

CRISPR as diagnostic tools?

I feel that the CRISPR companies (NTLA, CRSP, EDIT) are now moving into the diagnostic industry. Is it because this a hedge against their main Cas9 patents not working? I see the C2Cc  (Cas13a) crispr enzyme which targets RNA as the new and exciting direction.

http://science.sciencemag.org/content/early/2018/02/14/science.aaq0179 (zhang group)

http://science.sciencemag.org/content/early/2018/02/14/science.aar6245 (Doudna group)
The next patent battle?

On other CRISPR news,
Use of Cas9 to record events in cells (great for tracking cellular evolution)

http://science.sciencemag.org/content/early/2018/02/14/science.aap8992

Congressional Hearing on Gene editing

Crazy how fast gene editing has progressed. Hope Congress embraces this new technology instead of succumbing to fear mongering.

https://www.help.senate.gov/hearings/gene-editing-technology-innovation-and-impact

Older house hearing 2015

https://www.youtube.com/watch?v=7kU5EiT-0nY

NYT profile of FDA commissioner

https://www.nytimes.com/2018/02/11/health/gottlieb-fda-drugs.html

I think this has been trumps most well balanced pick in his cabinet. I think he balances both the left and right demands on regulations. I’m excited that he is pursing gene therapy and accelerating certain approval processes. On drug pricing I think he has a tough balancing act and his approval of generics should help. It’s nice with someone with industry and finance background try to streamline the process. With the new hhs secretary should be interesting how well they work together. 

Paying for Gene Therapy

https://icer-review.org/wp-content/uploads/2017/03/ICER-Gene-Therapy-White-Paper-030317.pdf

Illustrates some of the problems and potential solutions for gene therapy reimbursement models.

Cool New CRISPR journal

http://www.crisprjournal.com/

Wow, a new journal dedicated just for crispr.

Great Review of Gene Therapy

https://www.ncbi.nlm.nih.gov/pubmed/29326244

Gene Therapy comes of Age

Just shows how a promising technology can take years to finally work out the problems.

I suspect that CRISPR's rapid progress owns a debt to the pioneering work of the first gene therapy products.

I think one of the main issues will be reimbursement.