C&G Editor in Chief, Erin Harris

1. Accelerating NAMs For A Human-Based Future In CGT Development 8/1/2025

   The Complement-ARIE Consortium, led by FNIH’s Stacey Adam, Ph.D., is advancing the development, validation, and regulatory adoption of human-based new approach methodologies (NAMs) to transform preclinical testing and accelerate cell and gene therapy development.

2. Navigating The Complexities Of NK Cell Therapy Development 7/30/2025

   Deloitte's Amit Agarwal and Ashraf Husain share expert insights on how NK cell therapy developers can overcome manufacturing, supply chain, and commercialization challenges to successfully scale these promising therapies.

3. Redefining CGT With Exosomes And EVs 7/16/2025

   July’s Cell & Gene Live featured a comprehensive look at the scientific advances, clinical promise, manufacturing hurdles, and regulatory considerations shaping extracellular vesicle and exosome-based therapies. 

4. Inside AviadoBio And Astellas's Shared Mission To Bring Gene Therapy To Patients With FTD-GRN 7/2/2025

   AviadoBio and Astellas have joined forces to advance an investigational gene therapy designed to treat frontotemporal dementia with progranulin mutations (FTD-GRN), aiming to address a critical unmet need in neurodegenerative disease.

5. Advancing Gene Delivery: LNPs, Adenovirus, Lentivirus, And More 6/24/2025

   During this month’s Cell and Gene Live, Advancing Gene Delivery: LNPs, Adenovirus, Lentivirus, And More, expert panelists Francesca Barone, M.D., Ph.D., CSO at Candel Therapeutics and Karen Kozarsky, Ph.D., President of Vector BioPartners shared their perspectives on current challenges, technological innovations, and future directions in gene therapy vector development and application.

6. Rethinking Academic Translation In CGT: Inside The RISE Framework At Mass General Brigham 6/23/2025

   The RISE framework — focused on Resource sharing, Interdisciplinary collaboration, Sustainable funding, and Educational outreach — is helping academic medical centers such as Mass General Brigham overcome translational barriers in cell and gene therapy development and bring promising therapies closer to clinical application.

7. The Promise Of Focused Ultrasound For AAV Gene Therapy Delivery 6/11/2025

   Dr. Bernie Owusu-Yaw, a research fellow at Brigham and Women’s Hospital and Harvard Medical School, is pioneering a promising gene therapy approach that combines focused ultrasound with novel AAV capsids to safely and effectively deliver therapeutics across the blood-brain barrier, offering new hope for treating challenging neurodegenerative diseases.

8. Unlocking The Potential Of iPSC Therapies In Regenerative Medicine 5/21/2025

   In a recent Cell & Gene Live, leaders from Mayo Clinic and Astellas discussed the transformative potential of iPSC therapies in regenerative medicine, highlighting regulatory alignment and manufacturing innovation to realize their full impact.

9. Ferring Builds A Global Gene Therapy Manufacturing Network Around ADSTILADRIN 5/16/2025

   Ferring Pharmaceuticals has built a global manufacturing and support network around its flagship gene therapy, ADSTILADRIN, enabling scalable production, provider education, patient access, and real-world data collection to ensure sustainable and widespread adoption.

10. Atsena Therapeutics Advances Gene Therapy For XLRS 5/13/2025

    Atsena Therapeutics exemplifies the field’s growing shift toward non-oncology indications with its targeted gene therapy for X-linked retinoschisis, a rare inherited retinal disorder with no approved treatments.