top of page

News & Insights 

First Gene Editing FDA Approval - A Day to Celebrate Biotech!

A day to celebrate biotech! 🥳


Congrats to Vertex and CRISPR Therapeutics on FDA approval of CASGEVY

• 1st product that uses gene editing (See Exa-cel below for more info)


And Bluebird for FDA approval yesterday of Lyfgenia • another autologous stem cell-based gene therapy to treat Sickle cell disease


Interesting that both were approved on a single-arm (no control arm) trial, as the FDA is more willing to accept this data for a rare genetic disease with a high unmet need, and a strong biological basis.


From the FDA press release about yesterday's approvals, it seems that Lyfgenia will have a Black-Box safety warning (re: hematologic malignancies), but not CASGEVY (safety data looked impressive)


Two more approved cell therapy products,

helping solve medical challenges


Now,

biopharma will continue to find even better therapies that are easier to tolerate/available to more sickle cell patients with similar efficacy.


And Dx companies will help to ID the small number of patients that won't respond to CASGEVY


We know our therapies and Dx are not perfect...


But over time, we are giving more people, more healthy years

Because that's what we do in biotech...

Continue to find ways to make life better


Here's more on CASGEVY


What is CASGEVY?

CASGEVY (Exa-cel) is a cell therapy that uses CRISPR/Cas9 to edit the patient’s own (autologous) hematopoietic stem cells (HSCs) outside their body (ex vivo) to produce high levels of fetal hemoglobin in red blood cells after the gene edited HSCs are delivered back to the patient.


An excellent option for many sickle cell patients

• See FIG. 9 📸 for impressive efficacy data

⇨ After years of painful VOC events, CASGEVY reduces/eliminates them in almost all patients


The FDA characterized the clinical efficacy results of exa-cel to reduce VOCs in SCD patients as "strongly positive".



 

 


Want to get notified about our future posts with patent law insights and patent prosecution and clearance process excellence? Sign up for our email list today.


Post History:

12/10/23 posted (EJV)


This blog post is not legal advice and does not necessarily represent the viewpoint of any of the clients of Double Helix Law. Please seek the guidance of a registered and licensed patent attorney or patent agent for any patent-related questions you have. Please note, especially regarding statements made for jurisdictions outside the U.S., that the authors of this article are U.S. patent practitioners.

Comments


Get the latest DHL news and life science patent and investor insights right in your mailbox!

Thanks for subscribing!

bottom of page