Hottest Gene Editing Events Causing Waves in 2024
The 5th Genome Editing Therapeutics Summit (formerly the CRISPR 2.0 Summit) this December will be your foremost drug development focused gene editing meeting, providing you with the latest and greatest insights from the year.
Can’t wait until then? Here’s a quick breakdown of some of the top headlines from 2024:
FDA Release Much-Anticipated Draft Guidance for Platform Technology Designations
This is seen as a big step forward by many in the gene editing field, who see this designation as crucial for streamlining the time and costs of R&D by reducing repeated testing of the same technology.
Nvelop Therapeutics Launches
Founded in 2022 and based on two in vivo validated, virus-like delivery approaches by David Liu and Keith Joung, Nvelop Therapeutics has officially launched. Delivery efficiency, tissue specificity, cargo diversity, and safety have all been cited by Nvelop as limitations their technologies can overcome.
FDA Clears IND for First Prime Editing Clinical Trial
In a landmark first for the field, in April – Prime Medicine announced it had cleared its IND application for PM359, a prime editing therapeutic used to treat chronic granulomatous disease. The therapy uses blood stem modified ex-vivo by prime editors to modify cells containing the disease-causing mutation.
Intellia Therapeutics Achieves Landmark First in NTLA-2001 Trial
Positive clinical proof-of-concept data has been reported from the ongoing phase 1 trial, studying a single dose CRISPR-based treatment for transthyretin (ATTR) amyloidosis. This data demonstrates, for the first time, the potential of administering a gene editing therapy twice, which could be a crucial next step for the field.
What do Nvelop Therapeutics, Prime Medicine and Intellia Therapeutics all have in common? Beyond being trailblazers in gene editing drug development, they are part of the 5th Genome Editing Therapeutics Summit expert speaking faculty, all sharing real-world learnings from their programmes to help attendees streamline their paths to the clinic.
Register your place today to be in the room for these crucial insights.