From The Editor | December 29, 2022

Tackling CGT's Top 3 Regulatory Impediments


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1


2022 brought a great deal of change and disruption to cell and gene therapy companies. The news of companies closing their pipelines, undergoing liquidations, etc. was loud this year. The reasons for these pitfalls are varied and sundry, but despite the noise, cell and gene therapies have made impressive clinical progress. But the sector continues to face big hurdles, and overcoming regulatory challenges is one of them.

Indeed, the cell and gene therapy regulatory landscape impacts the biotech’s every move. From accelerated approval legislative provisions and rare or ultra-rare study design considerations to the use of existing data to prospect of the direct benefit for pediatric populations and lack of animal models (and countless other key areas), cell and gene therapy companies are at the mercy of impactful and stringent regulations.

Assuming garnering a better understanding of how the regulatory world intends to improve is high on your 2023 priority list, be sure to listen in to episode 40 of Cell & Gene: The Podcast, out 1/5/23, featuring my guest, FDA’s CBER’s Dr. Peter Marks. During our discussion, Dr. Marks and I talk through innovations in clinical development, next steps in CMC, advice for biotechs as they engage with CBER, realistic improvements for the cell and gene therapy field, and much more.  

Our next Cell & Gene Live, Tackling Cell & Gene Therapies’ Top 3 Regulatory Impediments, is right around the corner as well. Join me on 1/10/23 at 11 a.m. EST as my expert panelists, BridgeBio's Adora Ndu and REGENXBIO Inc.'s Nina Hunter detail how to better understand and tackle the top regulatory challenges facing cell and gene therapy. Registration is free, and you will most definitely want to attend this one. During the hour, we’ll get into the major regulatory hurdles stymying the cell and gene therapy sector, ways to overcome them, as well as how changing regulations impact manufacturing and commercialization.

I’ve had the good fortune to feature both Ndu and Hunter on separate but equally insightful episodes of Cell & Gene: The Podcast. I encourage you to listen in – or re-listen in – to both episodes. Episode 34 featured Ndu, and at the time the episode aired, we were right on the heels of Congress' reauthorization of the Prescription Drug User Fee Authorization (PDUFA) Act. Ndu explained the Commitment Letter, the programs that may be rolled out under PDUFA VII, why right-sizing CBER will help the cell and gene sector going forward as well as how sponsor companies can prepare for 2023 from a regulatory perspective. Episode 17 featured Hunter, and she shared potential solutions to gene therapies' regulatory roadblocks and why the accelerated approval pathway is so pertinent. Both episodes are under 30 minutes and are well worth your time.

Regulatory standards are critical, but it’s clear that the FDA intends to reduce the regulatory barriers that keep cell and gene therapy professionals up at night. Be sure to register for our next Cell & Gene Live and subscribe to Cell & Gene: The Podcast, subscribe to our newsletter, and visit Cell & Gene regularly for the information you’ll need to help you and your team tackle changing expectations and market conditions.