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Prenatal Delivery of a Therapeutic Antisense Oligonucleotide Achieves Broad Biodistribution in the Brain and Ameliorates Angelman Syndrome Phenotype in Mice

Wan Du, MD, PhD | April 11, 2024

Read our monthly summary of a Molecular Therapy article from the April issue of The Vector.

Molecular Therapy
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FDA Hosts Third Installment of CGT Science Series on CMC Guidance

Watch now! | April 11, 2024

Watch this virtual event, as well as past installments of this series, on our YouTube channel.

Policy & Advocacy
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Society Responds to FDA’s Potency Assurance for Cellular and Gene Therapy Products

Margarita L. Valdez Martínez  | April 11, 2024

Potency assays are a key part of product development. ASGCT believes that addressing the overarching concerns with FDA’s outlined potency assurance strategy through these comments will enhance the value of the final guidance document to assist CGT product developers as they address product potency. 

Policy & Advocacy
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Congratulations to Our 27th Annual Meeting Award Winners

Read more about them on our blog | April 10, 2024

We're looking forward to celebrating our 2024 award winners at the upcoming Annual Meeting May 7-11 in Baltimore, MD.

Annual Meeting 2024  |  Awards
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Register for #ASGCT2024 Before Rates Increase Next Month

May 7-11, 2024 | April 09, 2024

Join more than 8,000 of your colleagues at the largest gathering of gene and cell therapy professionals in Baltimore, MD, May 7-11, 2024.

Annual Meeting 2024
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CAR T for Acute Myeloid Leukemia with Dr. Marco Davila—Molecular Therapy Podcast

Listen now! | April 09, 2024

Listen to Dr. Davila discuss a recent article he co-authored, Bispecific CD33/CD123 targeted chimeric antigen receptor T cells for the treatment of acute myeloid leukemia.

Podcasts
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Vote in ASGCT's 2024 Election

Vote through May 1 | April 03, 2024

Help us fill four spots on ASGCT's Board of Directors and cast your ballot by May 1.

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Meet Ben Kleinstiver, PhD: ASGCT Volunteer Rockstar

Learn more about Dr. Kleinstiver | March 29, 2024

Research goals in the Kleinstiver lab include engineering and improving genome editing technologies, optimizing methods to accelerate the development of CRISPR enzymes, and transforming these tools into new classes of genetic therapies. 

Volunteer Rockstars
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Regulatory Landscapes in CGT with Sabrina Mogle—ASGCT's The Issue

Listen now! | March 26, 2024

In this episode, Dr. Walsh Martin talks to Sabrina Mogle about dedicating her career to the orphan drug and advanced therapy space, and how helping clients navigate regulatory conversations on the path from idea to BLA keeps her motivated today.

Podcasts
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Hot Topics in CGT with WIRED's Emily Mullin: Annual Meeting Preview

Listen now! | March 19, 2024

Today's guest is Emily Mullin, a staff writer at WIRED, and we're looking ahead to the major topics + themes that will be highlighted at the upcoming ASGCT Annual Meeting.

Annual Meeting 2024
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FDA Approves Lenmeldy for Metachromatic Leukodystrophy

Jeffrey S. Chamberlain, PhD | March 18, 2024

Lenmeldy is the first and only treatment in the U.S. for early onset MLD.

Cell & Gene Therapy Approval
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National Gene Vector Biorepository Will Begin Storing Human Clinical Samples

Visit NGVB's website to learn about its services. | March 14, 2024

Free services provided by NGVB benefit qualified NHLBI investigators at all stages of clinical development. 

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Hear Drs. David Schaffer and Adam Schieferecke on the Molecular Therapy Podcast

Listen now! | March 12, 2024

Listen to their discussion about a recent Molecular Therapy article, Evolving membrane-associated accessory protein variants for improved adeno-associated virus production.

Podcasts
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AI in CGT: Previewing the Annual Meeting with Dr. Rory Bricker-Anthony, Chris Leidli, and Catherine Gillespie

Listen now! | March 11, 2024

Get a preview of our AI-focused symposium during the 2024 Annual Meeting.

Podcasts
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ASGCT Responds to FDA’s Advanced Manufacturing Technologies Designation Program Draft Guidance

Margarita L. Valdez Martínez | March 11, 2024

Given the Society's mission, ASGCT expressed concerns about the limited scope of the proposed program and its ability to be a catalyst for innovation in CGT manufacturing. 

Policy & Advocacy
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Pediatric Gene Therapy Group Holds Fourth Annual Lecture Series to Discuss Ethical Issues

Ayden Eilmus, Nevaeh Everett, and Cara Hunt | March 08, 2024

Read about and watch the lunchtime lecture series from the Pediatric Gene Therapy and Medical Ethics Working Group.

 

 

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2024

Register for the 27th Annual Meeting

May 7-11, 2024 | Baltimore, MD

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