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Watch “The FDA's Role in Gene Therapy,” from ASGCT and NORD

ASGCT Staff | November 15, 2019

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

Education
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Learn About Gene Therapy for Hemophilia

ASGCT Staff | November 12, 2019

Hemophilia occurs when the genes that are vital to producing clotting factors are faulty. There are existing treatments for hemophilia that require lifelong administration, while gene therapy may offer a one-time treatment to address the cause of the disease. 

Education
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States Innovating on Gene Therapy Medicaid Reimbursement

ASGCT Staff | November 12, 2019

During the November 5 program at the ASGCT Policy Summit in Washington, D.C., state Medicaid experts shared their perspectives on developing coverage for these innovative new treatments.

Policy Summit
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Government Innovation is Key to Creating Regulatory Convergence

ASGCT Staff | November 11, 2019

During the Policy Summit’s Nov. 4 session, Developments in Gene Therapy Policy Landscape, speakers emphasized the importance of balancing predictability with flexibility as they discussed ways to align regulatory frameworks for gene and cell therapies.

Policy Summit
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NIH’s Collins Calls for Germline Gene Editing Moratorium at Policy Summit

ASGCT Staff | November 06, 2019

NIH Director Francis Collins, M.D., Ph.D. called for a worldwide moratorium on clinical uses of human germline gene editing, while defending ongoing and future applications of therapeutic somatic gene editing, on the third day of the ASGCT Policy Summit on November 6. 

Policy Summit
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New Payment Solutions Presented at ASGCT Policy Summit Could Help Offset Costs of Gene and Cell Therapies

ASGCT Staff | November 06, 2019

Speakers on the second day of the ASGCT Policy Summit discussed innovative payment strategies that they believe will help offset the cost of gene therapies and ensure more people gain access to these treatments.

Policy Summit
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FDA's Marks Urges International Collaboration at ASGCT Policy Summit

ASGCT Staff | November 05, 2019

Now is the time, Marks said during his keynote address, for regulators to work towards a globally harmonized framework that will facilitate the safe and ethical development of transformative gene therapy products. 

Policy Summit
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How Can Gene Therapy Help MPS Patients?

ASGCT Staff | October 29, 2019

There are seven different types of MPS, each caused by a different faulty gene. Gene therapy may be able to slow the progression of the MPS disorders I, II, III and IV, although it cannot reverse damage the disease has already caused. 

Education
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Register by Oct. 28 for the ASGCT Policy Summit

ASGCT Staff | October 24, 2019

Join us in Washington, D.C. for discussions about global regulatory, payment policy, and ethical issues surrounding gene therapy. 

Policy Summit
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Learn About Gene Therapy for Muscular Dystrophy

ASGCT Staff | October 24, 2019

Muscular dystrophy is a group of genetic diseases that cause progressive muscle weakness and loss of muscle mass. There are a wide variety of muscular dystrophies, each caused by different gene mutations.

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Six ASGCT Members Earn Career Development Awards

ASGCT Staff | October 23, 2019

ASGCT’s Career Development Awards support independent transformative pilot studies in gene and cell therapy conducted by ASGCT members.

ASGCT News   |   Development
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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.

Education
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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.

Education
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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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2019

ASGCT Policy Summit

November 4 – 6 | Washington D.C.