American Society of Gene & Cell Therapy

Go behind the scenes of #MolecularTherapy (MT) with us! In this new video, join ASGCT President Paula Cannon, PhD, MT Editor-in-Chief Roland W Herzog, PhD, and MT Oncology Editor-in-Chief Timothy Cripe, MD, PhD, who discuss how these prestigious publications have shaped the landscape of #genetherapy and #celltherapy research for the past 25 years. Learn about: ?? The expanding scope of gene and cell therapy research ? How our rigorous review process ensures top-quality publications ?? Why leading researchers choose Molecular Therapy journals ?? The impact and growth of our journal family https://lnkd.in/gnYyVUS6

?? NEXT WEEK: Join ASGCT and the Brazilian Association of Cell and Gene Therapy (ABTCel-Gen) on Dec. 2 for Advanced Therapy Medicinal Production (ATMP) Research in South America. This free event will explore the latest developments in Advanced Therapy Medicinal Product (#ATMP) research across South America, including innovative production techniques and regional advancements driving the field forward. Moderator: Virginia Pican?o-Castro, PhD Speakers: Carlos Javier Alméciga Díaz, PhD and Maria Veronica Lopez, PhD Time: 1:00-2:00 p.m. BRT / 10:00 a.m.-11:00 a.m. CT Register here: https://lnkd.in/esyuuDAM

Congratulations to our #ASGCTBreakthroughs24 Outstanding Poster Award winners! - Saeed Anwar (University of Alberta) Enhancing Antisense Oligonucleotide Efficacy Using Small Molecule Oligonucleotide Activity Enhancers (OAEs) - Pranali Mistry, (Nationwide Children's Hospital) Developing a Second Generation Proviral Plasmid to Reduce Aberrant Cross Packaging and ITR Promoter Activity in AAV Vector Preparations You can read these abstracts and the rest of the collection via our website: https://lnkd.in/gsE-ptS7 Registrants can watch ALL sessions on demand on the virtual meeting platform as well!

This National Family Caregivers Month, we're highlighting a critical session at Empowering Patients 2025: "Exploring Goals of Care and Personal Values When Considering CGT Trials" Jennifer Siedman with Courageous Parents Network and Megan Maack with the Childhood Dementia Initiative will share their dual perspectives as caregivers and advocates. This session will: - Address the complexities of clinical trial decisions - Provide frameworks for managing expectations - Offer guidance on facilitating crucial conversations between families and clinical teams - Share insights for advocates supporting families through treatment decisions ??? Session: March 13 | 11:50 AM - 12:20 PM ET Full Summit Details: ?? March 12-13, 2025 ? 9:00 AM - 12:30 PM ET daily ?? Virtual & Free Register: https://lnkd.in/gNchxqcG Can't make it? Register for on-demand access to all sessions exploring the intersection of cell & gene therapy with patient & caregiver experiences. Want to stay current on news and events relevant to patients, caregivers,?and others affected by rare diseases? We just launched a brand new newsletter! Subscribe to the Patient Press here: https://lnkd.in/gvwwWuKT #CellAndGeneTherapy #RareDiseases #PatientAdvocacy #NationalFamilyCaregiversMonth

Thank You for an Incredible Breakthroughs in Muscular Dystrophy Conference! We are deeply grateful to all the researchers, clinicians, industry partners, and attendees who made this year's conference a remarkable success. The breadth and depth of scientific advancement shared over these days has been truly inspiring. This conference highlighted extraordinary progress in the field, from groundbreaking clinical trial results to innovative therapeutic approaches. We saw encouraging data from multiple gene therapy trials, bringing hope to patients and families affected by #Duchenne muscular dystrophy. The presentation of novel delivery technologies, from engineered AAV capsids to ultrasound-mediated approaches, demonstrated our field's commitment to overcoming current therapeutic limitations. Particular appreciation goes to our keynote speakers, Drs. Carsten B?nnemann and Louis Kunkel. Over the course of 1.5 days, we learned about: - Next-generation gene editing technologies - Advanced delivery systems - Novel therapeutic approaches - Disease mechanism insights - Improved preclinical models The progress shared at this conference gives us great optimism for the future of #musculardystrophy treatment. Thank you all for your dedication to advancing this critical field of medicine! Muscular Dystrophy Association

WATCH NOW: A new Community Quick Takes module on focused ultrasound in #CGT! Listen to quick takes from speakers Elisa Konofagou, PhD; Agessandro-Abrahao, MD; and Jonathan Linder, MD; who discuss the safety of focused ultrasound technology, the latest advancements and clinical applications of the technology, how it can enhance #genetherapy + #celltherapy in treating brain diseases, and more. https://lnkd.in/gpR-JMJj

ASGCT recently submitted feedback to the FDA’s Draft Strategic Plan on Advanced Manufacturing Technologies (AMT). AMTs such as continuous manufacturing, distributed manufacturing, and novel analytical methods have significant potential to improve product development speed, strengthen supply chains, and prevent drug shortages - key considerations for the rapidly growing CGT field. Check out these highlights and read the full response below! - AMTDP independence: ASGCT supports FDA’s AMTDP as a distinct pathway and recommends that AMT eligibility remain separate from the Advanced Technologies Team (CATT) criteria. That would prevent restrictive overlaps and support broader access for innovative CGT manufacturing approaches. - Biologics License Applications (BLAs), AMT data, and Drug Master Files (DMFs): ASGCT recommends that FDA permit BLAs to cite AMT data in DMFs, which would streamline regulatory submissions and support investment in new manufacturing approaches while addressing intellectual property concerns. - Clarification on designation criteria and benefits: ASGCT advocates for clear guidance on eligibility criteria, especially around “substantial improvement” metrics, and calls for a “Graduated AMT” status to maintain regulatory benefits as FDA familiarity with these technologies expands. The Society remains committed to assist?FDA in addressing scientific and regulatory challenges to enable more standardized and efficient manufacturing approaches.?#ASGCTAdvocacy https://lnkd.in/gi9QJKC8

Meet our ASGCT-AAAS Congressional Policy Fellow, Dr. Adriana Bankston! After her first few weeks in Washington, D.C., she says, "being here has provided a much broader view of how we can better support research both within and outside of government sectors." Learn more about why she applied to this one-year policy fellowship, what she's learned so far, and how YOU can contribute to policymaking on Capitol Hill beginning next year if you're interested! Applications for this fellowship are now open through Feb. 24, 2025! https://lnkd.in/gRrzWgnp

What was the status of the #celltherapy and #genetherapy pipeline in Q3? Our CEO, David Barrett, JD, recently spoke to CGTLive's Noah Stansfield about highlights in the quarterly report we publish with Citeline. In Q3, we saw: ?? FDA approval of #Tecelra for #synovialsarcoma ??An increasing proportion of nononcology CGT products in development ??A rebound in startup financing Watch the full clip and read the report! https://lnkd.in/g6ucNVis

Good morning, #ASGCTBreakthroughs24 attendees, and welcome to Day 2! Coming up at 8:30 a.m., we'll hear about RNA targeted therapies during our first session chaired by Scott Q Harper. Don't miss speakers Toshifumi Yokota, PhD; Lindsay Wallace, PhD; and Aurelie Goyenvalle, PhD! #musculardystrophy