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Learn About Gene Therapy for Huntington’s Disease

ASGCT Staff | October 15, 2019

The American Society of Gene & Cell Therapy (ASGCT) released its latest resource profiling disease-specific treatment efforts, this time focusing on developing gene therapies for Huntington’s disease.

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ASGCT Policy Summit Highlights Patient and Advocate Perspectives

ASGCT Staff | October 14, 2019

ASGCT will have patient, caregiver, and advocate speakers participating in all three days of our upcoming Policy Summit, held at The Westin in Washington, D.C. November 4-6.

Advocacy   |   Policy Summit
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ASGCT Education Program Returns, Covers Parkinson's Disease

ASGCT Staff | October 10, 2019

ASGCT is proud to launch a second round of disease-specific resources in its Patient Education program. The first of the seven new resources focuses on developing gene therapies for Parkinson’s disease.

ASGCT News   |   Education
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Watch “The Science Behind Gene Therapy,” from ASGCT and NORD

ASGCT Staff | October 08, 2019

The webinars in this series are designed to inform patients, patient advocates, families, and the public about various aspects of gene therapy.

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Rhoda Baer acquired from National Cancer Institute

Bridging the Gap Between Scientists And Patients In Gene Therapy Development

David Barrett, J.D. | September 27, 2019

Patients with rare diseases must rely collectively on scientists, physicians, pharmaceutical and biotechnology companies, and rare disease advocates to learn about these disorders, says ASGCT Executive Director David Barrett.

Advocacy   |   Education
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FDA photo

Margaret Hamburg, M.D. to Speak at Policy Summit

ASGCT Staff | September 26, 2019

Margaret Hamburg, M.D., an internationally recognized expert in medicine and public health, will be one of the keynote speakers on November 6, the third and final day of the ASGCT Policy Summit in Washington, D.C.


Education   |   Policy Summit   |   Policy Updates
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Watch “Gene Therapy: Yesterday, Today, and Tomorrow” from ASGCT and NORD

ASGCT Staff | September 24, 2019

“Gene Therapy: Yesterday, Today, and Tomorrow,” the first of five educational webinars from ASGCT and the National Organization for Rare Disorders (NORD), is now available for free.

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Dose escalation of AAV-PHP.B in the mouse brain following intravenous and intra-arterial delivery

Effectiveness of Gene Therapy Delivery to the Brain Varies by Species

Edith Pfister, Ph.D. | September 19, 2019

In reviewing a recent Molecular Therapy article, Edith Pfister, Ph.D., associate editor of ASGCT's newsletter The Vector, outlines patterns of virus CNS transduction efficiency in non-human primates in comparison with mice.

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ASGCT's Role in Treating Sickle Cell Disease

Guangping Gao, Ph.D. | September 16, 2019

September is Sickle Cell Awareness Month, and a major goal of ASGCT’s new strategic plan is to expand our outreach to countries outside the U.S.

President's Message
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2020 Annual Meeting Website is Now Live

ASGCT Staff | September 09, 2019

For the most up-to-date information on our upcoming Annual Meeting, you can now visit

Annual Meeting
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Credit: Daniel Soñé Photography, LLC

NIH Director Collins to Speak at ASGCT Policy Summit

ASGCT Staff | September 05, 2019

Francis Collins, M.D., Ph.D., director of the National Institutes of Health, will deliver his keynote address, "The Need for an International Moratorium on Clinical Uses of Human Germline Gene Editing," on day three of the ASGCT Policy Summit, November 6.

Education   |   Policy Summit   |   Policy Updates
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NIH Director Francis Collins, M.D., Ph.D
Credit: NIH

What to Expect at the ASGCT Policy Summit

ASGCT Staff | September 04, 2019

With a new name and expanded programming, the 2019 ASGCT Policy Summit brings a wide range of gene therapy experts and stakeholders from around the world to Washington, D.C. November 4-6 to discuss the challenges of and solutions to successful gene therapy development, access, and use.

Policy Updates
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Credit: U.S. Mission Photo by Eric Bridiers,

ASGCT Submits Comments to WHO Advisory Committee

ASGCT Staff | August 22, 2019

ASGCT submitted comments on August 16 to the World Health Organization’s (WHO) Advisory Committee on Developing Global Standards for Governance and Oversight of Human Genome Editing regarding guidance to governments, institutions, and researchers on scientific, ethical, social, and legal challenges related to both germline and somatic cell human genome editing.

Advocacy   |   Policy Updates
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ASGCT, NORD Partner for Free Educational Gene Therapy Webinars

ASGCT Staff | August 19, 2019

Have you heard about gene therapy? Do you want to learn about the history of gene therapy, which gene therapies are currently available and what the future may hold? ASGCT and NORD partner to host the first of five educational gene therapy webinars, "Gene Therapy: Yesterday, Today and Tomorrow," on August 29 at noon EST.

Advocacy   |   Education
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Credit: Rita Elena Serda, Duncan Comprehensive Cancer Center at Baylor College of Medicine, National Cancer Institute, National Institutes of Health

CAR T-Cell Therapy Covered by Medicare, but Still Inadequately Reimbursed

ASGCT Staff | August 14, 2019

An announcement that the Centers for Medicare & Medicaid Services will cover CAR T-cell therapy is welcome news, but ASGCT remains concerned about reimbursement levels for CAR T-cell therapies for Medicare beneficiaries provided in the inpatient setting.

Advocacy   |   CAR T-cell Therapy   |   Medicare   |   Payment Policy
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What's It Like to Attend the ASGCT Annual Meeting?

ASGCT Staff | August 01, 2019

Ever wondered what it's like to attend the ASGCT Annual Meeting? Join four of our gene and cell therapy pros, all of whom hold very different roles in ASGCT, as they attend the premier event in the booming field of gene and cell therapy.

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ASGCT Policy Summit

November 4 – 6 | Washington D.C.