The Advocate

Volume 1, Issue 3

Subscribe to The Advocate 

May 2021

In This Issue:

Newborn Screening Advocacy Succeeds in Georgia
Society Supports Access to Genetic Testing and Screening
Policy News


ASGCT Advocacy

Newborn Screening Advocacy Succeeds in Georgia

Earlier this month, Georgia Governor Brian Kemp signed HB 567 into law, which will require the state to consider new federal newborn screening (NBS) recommendations within one year, implement them within two and a half years, and provide appropriate funding for the NBS program. ASGCT supported the bill earlier this year through a sign-on letter led by the EveryLife Foundation for Rare Diseases. Access to NBS and other genetic testing is an ASGCT policy priority to facilitate access to, and maximal benefit from, potentially lifesaving and life-altering gene therapies upon their approval.

Society Supports Access to Genetic Testing and Screening

ASGCT recently worked to enhance access to genetic testing and screening in two ways. The Society signed on this month to a letter to Congressmen Eric Swalwell (D-CA) and Scott Peters (D-CA) in support of legislation that would require state Medicaid programs to cover genetic and genomic sequencing tests. Specifically, the letter, led by The Assistance Fund, requests coverage of various diagnostic sequencing technologies (including whole genome sequencing, whole exome sequencing, and gene panels) to allow access to whichever type of diagnostic test a clinician deems necessary to accurately diagnose individual patients. The two representatives are working on a compromise bill that will reconcile differences between their two bills. Increasing consistent approaches to Medicaid coverage across states could improve timely diagnosis, and therefore treatment, of genetic diseases.

ASGCT also supported language in the Ohio state budget (HB110) that would expand and accelerate NBS in Ohio. The bill would add spinal muscular atrophy (SMA) and X-linked adrenoleukodystrophy (ALD) to Ohio’s NBS panel. In addition, the legislation would require the state to consider adding conditions that are added to the federal Recommended Uniform Screening Panel within six months and, if recommended to be screened for, to initiate screening with a year and a half. HB110 passed the Ohio House of Representatives on April 21 and has been referred to the Ohio Senate. ASGCT supported this legislation via an EveryLife Foundation sign-on letter.

Policy News

  • At the ASGCT 24th Annual Meeting last week, regulators Emer Cooke and Wilson Bryan shared insights on how COVID-19 may affect gene therapy clinical trials, including FDA flexibility for missing data. View the full session if you are a registered attendee, or you can register now for access to all Annual Meeting sessions through at least June 13.

  • Last month, ASGCT released a public education resource on the adenovirus-based vaccine for COVID-19 that is authorized by FDA for emergency use. Share this resource among your circles, along with the unit on mRNA vaccines, to inform, ease fears, and celebrate these accomplishments in the gene therapy field.

  • In late April, FDA lifted its clinical hold on UniQure’s gene therapy trial for hemophilia B. The Agency agreed with UniQure’s conclusion that the treatment was unlikely to have caused a case of liver cancer in a trial participant.

  • ASGCT applauds FDA’s plan to end its enforcement discretion on May 31 for compliance with regulatory approval requirements for human cellular and tissue-based products (HCT/Ps) that do not meet certain criteria (e.g., minimal manipulation, homologous use). Enforcement of these premarket approval requirements may protect patients from potentially unsafe and ineffective products.


  • Save the date: The third annual ASGCT Policy Summit will be held September 22-24, 2021.

  • ASGCT is hosting a free virtual Forum on Gene Therapy for Underserved Populations: Drug Development for Ultrarare Diseases and Lower-Income Countries to address the challenges of, and approaches to, creating sustainable models for development. Registration is now open for this June 22 event.


ASGCT Policy Summit

September 22-24, 2021

This site uses cookies to offer you a better user experience and to analyze site traffic. By continuing to use this website, you accept our use of cookies.