16 Minutes News by a16z

CRISPR: Policy, Platform, Trials (#11)

Episode Summary

News and trends covered this week -- all about the latest policy and in practice implication for CRISPR -- include: * California law requiring labels for self-editing kits (that don't exist yet) * Alliance (including 13 of the most active companies in gene editing for therapeutics) statement against germline editing at this time -- and parallels/differences given China CRISPR babies scandal & recombinant DNA from 1970s * clinical trials for CRISPR come of age, with first publicly identified patient (for sickle cell disease) and first study inside the human body (for inherited congenital blindness) * CRISPR as a platform, applications, and what all of this means for innovation …with @jorgecondebio @andy23tran and host @smc90.

Episode Notes

This is the 11th episode of 16 Minutes, a weekly show where we quickly cover the top headlines of the week, the a16z Podcast way: why are these topics in the news; what’s real, what’s hype -- from our vantage point in tech.

This week, we do a 20-ish minute deep-dive on CRISPR, to tease apart the FUD from the facts given a bunch of recent news (not research!) around the gene-editing platform. The news implications range from policy to practice:

• At the end of July, one of the first laws to directly regulate CRISPR was signed in the state of California by Governor Gavin Newsom, making it illegal for "gene therapy" kits to be sold, unless they have a clear warning label that the kit is "not for self-administration” -- this policy may affect future "biohacking"... but also, such kits don't really exist yet!
• At the end of August, the Alliance for Regenerative Medicine (ARM, an TKTK) released a Therapeutic Developers’ Statement of Principles for the use of gene editing in therapeutic applications -- endorsed by 13 of the most active companies in the field -- while it supports somatic cell editing, it also asserts that germline gene editing "is currently inappropriate in human clinical settings".
• The above news is also relevant given news of/ the scandal from earlier this year (first revealed by MIT Technology Review) around "CRISPR babies" with germline edits born in China;
• Meanwhile, clinical trials for CRISPR are coming of age, with the first publicly identified CRISPR patient (treating sickle cell disease) and the first CRISPR study inside the body (for LCA, the most common cause of inherited childhood blindness); how do these show pacing and containment of risks (somatic cell, monogenetic phenotypes, ex vivo, etc.)?

Finally, how does all this news affect innovation in gene therapies and other applications? When it comes to engineering the genome -- including tech challenges and startup opportunities -- what might we  borrow from the history of innovation here? If CRISPR is not a single tool or set of proteins but a platform, what becomes possible? General partner Jorge Conde and partner Andy Tran, both of the a16z bio team, share their thoughts on all this and more with host Sonal Chokshi in this episode of 16 Minutes.