News

CMN Weekly (29 October 2021) - Your Weekly CRISPR Medicine News

Some of the best links we picked up around the internet

By: Gorm Palmgren - Oct. 29, 2021

Top picks

Research

Industry

Clinical News

  • The FDA has granted orphan drug designation to Intellia Therapeutics' NTLA-2001 to treat transthyretin (ATTR) amyloidosis. NTLA-2001 is an in vivo investigational CRISPR therapy that knocks out the transthyretin (TTR) gene and reduces the concentration of TTR protein in serum.
  • FDA has placed Gamida Cell's GDA-201 on hold pending modifications to donor eligibility procedures and sterility assay qualification. GDA-20 is a cryopreserved, off-the-shelf cell therapy candidate for treating patients with follicular and diffuse large B cell lymphomas. The company recently submitted an IND application to FDA for a Phase 1/2 trial with GDA-201.

COVID-19

Reviews

Opinion and Prizes

Huh, heh, wow

Sign-up Free Newsletter here

Tags

HashtagArticleHashtagMissing linksHashtagNewsHashtagCMN WeeklyHashtagIntellia Therapeutics, Inc.HashtagMammoth Biosciences, Inc.HashtagVertex Pharmaceuticals, Inc.

News: CMN Weekly (29 October 2021) - Your Weekly CRISPR Medicine News
CLINICAL TRIALS
Sickle Cell Disease, SCD
Sponsors:
Beam Therapeutics
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Hereditary Angioedema, HAE (NCT05120830)
Sponsors:
Intellia Therapeutics
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Acute Myeloid Leukemia, AML, (NCT05066165)
Sponsors:
Intellia Therapeutics
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
View all clinical trials
close
Search CRISPR Medicine