American Society of Gene & Cell Therapy

🦓Today is #RareDiseaseDay, and it's more than just a day. It is a call to action for us to support research, push for policy changes, and empower patients in their journey toward treatments.  Here are a few actions you can take to show your support for those affected by #rarediseases: ➖ Register for our FREE, inaugural Empowering Patients Summit next month https://lnkd.in/ghKj7nJa ➖ Learn from our #PatientEducation resources + share them with your networks https://lnkd.in/gsq-UsA9 ➖ Use this social media kit from National Organization for Rare Disorders https://lnkd.in/gNTr5DFV

On tomorrow's #RareDiseaseDay, we're reminded of the 300 million people worldwide affected by rare diseases. Join #ASGCT for Empowering Patients 2025: A Cell and Gene Therapies Summit on March 12-13! #Raredisease treatment development requires all stakeholders to work together, and this Summit provides attendees with direct access to the scientific expertise and regulatory insights needed to navigate the complex #CGT landscape, while ensuring the patient voice remains central to the conversation. Featured Sessions Include: - A live conversation with FDA leadership on regulatory pathways for rare disease treatments - Expert insights on clinical trial design for rare diseases - Discussions on managing expectations and navigating difficult conversations when considering CGT trials - Strategies for addressing challenges when promising innovations face setbacks Turn awareness into action. Register now: www.asgct.org/ep2025 #RareDiseaseDay #EmpoweringPatients2025 #CellAndGeneTherapy

🚨 New resource alert! 🚨 Check out our quarterly Community Quick Takes module on immune responses to AAV #genetherapy. In this module, Drs. Allison Bradbury and Barry Byrne give patient-friendly insights on how the immune response to AAV gene therapies impacts treatment outcomes. Let us know what you think and please share with your networks! Visit the link (in comments) to view the full video on our #PatientEducation site.

#GeneEditing has gained a lot of attention in recent years, but this might leave some wondering: what is it? 👍 Gene editing is: - A technique used to correct, remove, or add genetic material to treat or prevent disease. - A method that can be performed inside the body (in vivo) or outside the body (ex vivo) before returning cells to the patient. - A tool that uses various technologies, such as CRISPR-Cas9, TALENs, and Zinc Finger Nucleases (ZFNs), to modify DNA. - A strategy being closely researched in preclinical studies and clinical trials for a range of disorders. 👎 Gene editing is not: - The same as traditional #genetherapy, which typically delivers a new, working copy of a gene instead of altering existing DNA. - Without risks—off-target edits and unintended genetic modifications are still being studied. - A guaranteed cure—while promising, gene editing therapies are still in early research and clinical trial phases. - Widely available yet—most gene editing treatments are still undergoing rigorous safety and efficacy testing. #PatientEducation https://lnkd.in/gScdZhbE

Calling all #genetherapy + #celltherapy researchers! Do you have late-breaking data that is: • high impact • groundbreaking • innovative and • newsworthy? Send us your work through March 14 for the opportunity to present it at #ASGCT2025! Associate members: If you're a first and presenting author of an accepted late-breaking abstract, you can register for the meeting for free! https://lnkd.in/gGfWFXNT

📰 OUT NOW: The February issue of The Patient Press! 📰 In this issue, we're inviting patient advocacy organizations, academic institutions, and other nonprofits to show their support for The National Institutes of Health research funding and sign onto a group letter to congressional appropriators by March 4. ICYMI: The Patient Press is our bimonthly newsletter for the patient advocate community. Subscribe to receive it directly in your inbox every other month and please share with other #raredisease advocates who might be interested. #PatientEducation #ASGCTAdvocacy https://lnkd.in/ehmdz4RG

Celebrating Groundbreaking Achievements in #GeneTherapy We are excited to share the remarkable story of a two-and-a-half-year-old girl who is showing no signs of spinal muscular atrophy after becoming the first person to receive gene-targeting treatment while still in the womb! This success story aligns with the work of one of #ASGCT's upcoming Annual Meeting keynote speakers, Dr. Tippi MacKenzie. She is pioneering fetal therapies and in-utero treatments for genetic disorders, and her innovative research is advancing our understanding of early intervention approaches for genetic conditions. As ASGCT prepares for its 28th Annual Meeting in New Orleans (May 13-17), the Society is increasingly inspired by physician-scientists who are transforming possibilities into realities for patients. From laboratory discoveries to clinical applications, their dedication changes lives before they even begin. This is why ASGCT gathers each year - to celebrate these advances, share knowledge, and forge the collaborations that will lead to the next breakthrough. Learn more about attending the 2025 Annual Meeting, virtually or in-person, at www.asgct.org/2025.

❓ Did you know? February is Low Vision Awareness Month, a time to highlight inherited retinal disorders (IRDs) like retinitis pigmentosa, Stargardt disease, and Leber congenital amaurosis (LCA) that impact vision. ✔️ #Genetherapy is a promising approach to slow or even stop vision loss caused by IRDs. ✔️Learn more about how gene therapy can be delivered into the eye on our #PatientEducation site, and share with your #raredisease network! https://lnkd.in/g_CjwTAN

Join us on Wednesday, March 19 at 10 a.m. CST for the next installment of #ASGCT's Molecular Therapy Presents: #Immunotherapy in Autoimmune Disease! With seven CAR T #celltherapies currently approved in the United States to treat certain forms of cancer, researchers and clinicians are exploring other applications to treat #autoimmune diseases. Following the ground-breaking first report of a patient with severe systemic #lupuserythematosus who was successfully treated with CD19 CAR T cells, clinical trials of CAR-T cell therapies for #lupusnephritis, #scleroderma, #myositis, and #multiplesclerosis have taken flight across the world. Join moderator Boris Fehse, PhD, and speakers Lili Yang, PhD; Lena Kristina Pfeffer, MD; and Fabian Mueller, MD. Register today here! https://lnkd.in/e9Npuwur #celltherapy #moleculartherapy

📆 There are a few days left to apply for the Congressional Policy Fellowship! ASGCT Members: Apply through Monday, Feb. 24, to spend a paid year in Washington, D.C. providing CGT guidance to legislators. Learn more about what you'll do in this position + apply today! #ASGCTAdvocacy #genetherapy #celltherapy https://lnkd.in/g4xPm2Vc