First CRISPR Treatment Poised for Historic Approval

PLUS: Complexity Challenges Progress in Autism

Hi friends 👋🏼,

Big news this week as the first-ever CRISPR therapy seeks approval. Plus, more stories for Autism Awareness Month:

  • First CRISPR Treatment Poised for Historic Approval

  • Complexity Challenges Progress in Autism READER REQUEST

  • Current & Emerging Treatments for Tuberous Sclerosis Complex READER REQUEST

Upgrade to Premium for access to weekly articles and to make custom requests from our team of scientists.

(if you’re already a premium member, ignore this little box)


🧬✂️ First CRISPR Treatment Poised for Historic Approval

1/ Vertex and CRISPR Therapeutics have submitted the first-ever CRISPR-based cell therapy for approval 🥳


Exciting news for patients with #sicklecell disease: the first #CRISPR #genetherapy is seeking approval for clinical use. This groundbreak... See more

2/ The therapy, called Exa-cel (previously CTX001), stopped painful crises in sickle cell and led to 90% of beta-thalassemia patients no longer needing regular blood transfusions!

3/ Patients will continue to undergo a 15-year safety follow-up, but we will likely see the FDA’s final decision in the next 8-12 months.

♾️ Complexity Challenges Progress in Autism

Image courtesy of Dr. Alysson Muotri, Ph.D. presented at the Autism Tree Global Neuroscience Conference, 2022

April is Autism Awareness Month, so let’s dive in to understand this complex and nuanced condition.👇🏼

Subscribe to The Cure to read the rest.

Become a paying subscriber of The Cure to get access to this post and other subscriber-only content.

Already a paying subscriber? Sign In

A subscription gets you:
Submit your disease request and we'll find and report on clinical trials, emerging medical technology, and innovative therapeutics.
Easy to understand science written for the everyday person.
Articles delivered to your inbox every Sunday.
Plus - No ads. Ever.