The Advocate

April 2025: Volume 5, Issue 4

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In This Issue:

ASGCT Leadership Meets with Appropriators to Discuss NIH, FDA 
Congressional Briefing on Gene Editing Features ASGCT Experts
Senate Schedules Hearings on Biomedical Research and Innovation 
House and Senate Pass Budget Resolutions  
Priority Review Voucher Reauthorization Stalls in Congress
Senate Confirms NIH, FDA, CMS Leaders
HHS Announces Workforce Reduction and Restructuring  
Public Input Needed on Future Deregulation
First Company Announces Receipt of FDA's AMT Designation
Events
Policy News

ASGCT Advocacy

ASGCT Leadership Meets with Appropriators to Discuss NIH, FDA 

ASGCT President Dr. Paula Cannon met with legislators in the House of Representatives in March to advocate for robust National Institutes of Health (NIH) funding. The Society had bipartisan meetings with House appropriators to discuss NIH funding and the proposed 15% cap on NIH facilities and administration (F&A) reimbursement. Dr. Cannon emphasized the importance of these F&A funds for CGT programs, where therapies are often developed in highly specialized facilities shared between multiple labs at an institution. Earlier this month a federal judge permanently blocked implementation of the F&A cap, ruling that the change in policy violated federal statutes, failed to follow proper rulemaking procedures, and acted arbitrarily in imposing the cap retroactively. The Administration has appealed. ASGCT is pleased that the F&A rate system has been reaffirmed for now, but continued uncertainty will have a negative impact for the field and the Society remains concerned about potential caps in future funding packages.  

ASGCT signed onto a letter with 500+ other research- and patient-focused organizations asking for increased NIH funding and will continue engaging throughout this appropriations cycle to ensure scientists have the tools and resources they need to bring forth the next generation of gene therapies.

Congressional Briefing on Gene Editing Features ASGCT Experts 

On April 9, ASGCT hosted a virtual congressional briefing to educate Members of Congress and their staff on the potential of gene editing and the growing pipeline of therapeutic products. ASGCT members Dr. Paula Cannon, Dr. Charlie Gersbach, Dr. Janice Chen, and Scott McGoohan emphasized how foundational NIH basic research funding has been in advancing these technologies from the lab to the clinic, and consistently highlighted the importance of ensuring patient access as development progresses. The briefing featured expert perspectives on the science behind gene editing, its clinical applications, and the unique promise it holds for the rare disease community. With the first CRISPR-based therapy approved in the U.S. in 2023 and additional products in late-stage development, the session provided timely insights into the opportunities and challenges shaping the future of genetic medicine. 

Senate Schedules Hearings on Biomedical Research and Innovation 

The Senate Appropriations Committee has scheduled a hearing titled Biomedical Research: Keeping America's Edge in Innovation for April 30. This first hearing will feature researchers discussing biomedical research's importance and economic impact. A later hearing to be scheduled will include administration officials testifying about the government's role in supporting this research.  

House and Senate Pass Budget Resolutions  

The U.S. House of Representatives narrowly approved a budget framework on April 10 on a 216-214 vote. The budget resolution is significant because it provides a blueprint to begin the reconciliation process, which would allow Congress  to pass legislation with a simple majority in the Senate, bypassing the typical 60-vote threshold needed to overcome a filibuster. 

The budget resolution directs Congress to cut $1.5 trillion across the federal government, including $880 billion from programs overseen by the House Energy & Commerce Committee. Most likely, the source of those cuts would be federal funds for state Medicaid programs. While no specific plans have been announced, ideas circulated earlier this year suggest that per-capita spending caps, work requirements, and cutting subsidies for Medicaid expansion population could be on the table.  

Next steps in the process involve drafting the formal reconciliation bill, which will detail specific tax and spending measures as part of the Administration’s legislative agenda. This development will unfold over the coming months as lawmakers work to reconcile differences between the House and Senate versions.  

Priority Review Voucher Reauthorization Stalls in Congress

The bipartisan Give Kids a Chance Act (S.932/H.R.1262) includes the reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) program among other policies. The HELP Committee announced a markup for the Senate version bill earlier this month, which was subsequently postponed. The PRV program, which expired in September 2024, has been a critical incentive for developing treatments for rare pediatric diseases, resulting in over 50 new therapies since its inception. This mechanism has been especially beneficial for small and emerging biotech firms, enabling them to attract investment and bring innovative treatments to market without imposing costs on taxpayers. ASGCT will continue to emphasize the urgency of reauthorizing the program to prevent delays in drug development for children with rare diseases. 

Senate Confirms NIH, FDA, CMS Leaders

The Senate confirmed Dr. Marty Makary as Commissioner of the FDA on a 56-44 vote, gaining support from all Republicans and three Democratic Senators. Dr. Makary was previously a pancreatic surgeon at Johns Hopkins. Additionally, Stanford health economist Dr. Jay Bhattacharya was confirmed as NIH Director following a party-line Senate vote of 53-47. ASGCT will engage with incoming leadership to champion robust support for cell and gene therapy research. 

Dr. Mehmet Oz was confirmed in early April by the full Senate (53-45) as Administrator of the Centers for Medicare and Medicaid Services (CMS). In his confirmation hearing, Dr. Oz expressed strong support for cell and gene therapy access. When discussing sickle cell disease treatments, Dr. Oz specifically acknowledged the transformative potential of CRISPR technology and emphasized the value proposition of advanced therapies: "If we could offer these young children this treatment, they would be cured... That seems to be a pretty good investment." Dr. Oz also outlined his interest in novel payment approaches that could amortize costs while ensuring patient access to potentially curative therapies.  

HHS Announces Workforce Reduction and Restructuring  

​In response to President Donald Trump's Executive Order 14210, the U.S. Department of Health and Human Services (HHS) has announced a reduction in force (RIF) and restructuring plan. Secretary Robert F. Kennedy, Jr. also distributed a department-wide memo outlining broad HHS restructuring plans​.  

There has been broad interest from Congress. HHS staff briefed House Energy & Commerce staff in a closed-door briefing, with the potential for public testimony from Secretary Kennedy in the coming weeks. The Chair and Ranking Member of the Senate Health, Education, Labor, and Pensions (HELP) Committee also invited Kennedy to testify on the changes, though no hearing has yet been scheduled. Additionally, Committee policy leaders in the Senate and bicameral appropriations leaders have requested additional information on staffing cuts and restructuring plans. 

RIF notices impacted employees across HHS's major operating divisions, with particular concentration in administrative, policy, and communications functions. These reductions are on top of the 10,000 federal employees who had already departed the federal government since January, resulting in a total staff reduction of around 25%. There have been conflicting reports on whether HHS plans to rehire some of the employees impacted by RIFs. 

Some of the CGT-related agency staff reductions include:  

• Approximately 3,500 positions at the Food and Drug Administration (FDA). Drug, medical device, and food reviewers plus inspectors, are excluded from layoffs, but project managers and other support staff for those reviewers are not. FDA policy, communications, and senior staff have also seen deep cuts. There is some concern that the staff impact of this RIF could leave the Agency unable to meet its statutory agreements under PDUFA, potentially jeopardizing the future of the user fee program. The Agency plans to hire contractors to help complete active projects following the RIF. Separately, FDA has reportedly pulled back on some return-to-office mandates.   

• The National Institutes of Health (NIH) will decrease its workforce by approximately 1,200 employees, including the directors of five institutes and at least two senior leaders. Dr. ​Eric Green's term as director of the National Human Genome Research Institute (NHGRI) ended on March 17, as his appointment was not renewed. 

• Roughly 300 CMS staff were affected, including employees involved in policy and oversight functions. Details about the precise distribution of cuts within CMS are still emerging, but early reports suggest reductions targeted administrative personnel.  

For FDA, the proposed restructuring plan would dissolve product-specific offices and consolidate all product review divisions (drugs, biologics, devices, tobacco, and foods) into a single "Office of Product Evaluation and Regulation." Four additional offices would be created: an Office of Inspections, Compliance, and Enforcement; an Office of Scientific and Regulatory Policy; an Office of Administrative and Shared Services; and an Office of Strategic Programs and Innovation, along with a Digital Health Center of Excellence – consolidating the Agency's compliance, policy, administrative, strategic, and digital health functions into centralized offices.  

HHS also dissolved the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), which previously guided state-level newborn screening (NBS) recommendations. This federal panel's closure raises concerns for gene therapies as it may delay identification of eligible patients for early treatment. Decisions were anticipated on screening for metachromatic leukodystrophy and Duchenne muscular dystrophy, both conditions with recent gene therapy approvals. ASGCT has previously engaged with the ACHDNC to support NBS through the Recommended Uniform Screening Panel and will continue to monitor how this closure may impact patient identification and access.

Public Input Needed on Future Deregulation 

The Office of Management and Budget has released a Request for Information seeking public input on deregulation efforts, inviting stakeholders to identify regulations that should be rescinded. This initiative aligns with recent White House executive orders, including one directing a 60-day agency review (due April 19) to identify potentially unlawful regulations and another aimed at reducing what the Administration calls anti-competitive regulatory barriers. The timing is significant as reform proposals have been gaining traction through multiple channels, including congressional hearings, public letters from industry and investors, and various individual reports calling for regulatory changes. 

First Company Announces Receipt of FDA's AMT Designation

FDA's Advanced Manufacturing Technology (AMT) designation enables pharmaceutical manufacturers to receive early FDA feedback on innovative production methods before formal application submission. For cell and gene therapy developers specifically, the pathway supports novel production methods that improve consistency and scalability of these complex therapies, addressing unique manufacturing challenges while enabling more efficient processes that could reduce costs. FDA finalized their AMT guidance in December 2024; this month a company announced they have received AMT designation. FDA has not published a centralized list of designated companies. ASGCT has previously supported the AMT program and provided feedback on FDA’s strategic plan for implementing AMTs. The Society appreciates FDA’s recognition of the potential to advance and accelerate CGT development, and will continue to advocate for regulatory flexibilities in Congress to support CGTs.  

Events

Less Than One Month Until #ASGCT2025

Time is running out to register for cell and gene therapy's largest scientific meeting! Join your colleagues in New Orleans or virtually May 13-17 for the ASGCT 28th Annual Meeting. 

From AAV vector design to venture capital insights, this year’s expanded program includes brand-new session tracks to help you navigate it all. Don't miss this opportunity to stay at the forefront of cell and gene therapy innovation. Register today. 

Save the Date for the 2025 Policy Summit

The Policy Summit will return September 25-26, 2025, at the Washington Marriott Georgetown in Washington, D.C.! Save the date now and watch this newsletter in the coming months to learn the latest on speakers, topics, and registration. 

Inaugural Empowering Patients Summit Brings Together CGT Community 

ASGCT successfully hosted its first Empowering Patients 2025: A Cell and Gene Therapies Summit on March 12-13, bringing together patients, advocates, clinicians, and researchers. Sessions covered essential topics ranging from gene therapy approaches and clinical trial design to manufacturing basics and navigating the complex journey from early-stage research to treatment approval. You can still register and watch the sessions on-demand through the end of this week—after that, they’ll be available on ASGCT’s YouTube channel. Stay tuned for details on the next summit, coming March 11–12, 2026! 

Register for a Workshop on Cancer Therapies  

Friends of Cancer Research and the Parker Institute for Cancer Immunotherapy are hosting a joint public meeting called Unlocking Next-Generation Therapies: Exploring Innovative Development and Manufacturing Models for Cell Therapies on Friday, May 9, in Washington, DC (also available virtually). Register today to join this important discussion on advancing cell therapies, particularly for rare diseases.