April 26, 2021

Senate President Matt Huffman 1 Capitol Square Second Floor Columbus, OH 43215

Dear President Huffman,

I am writing on behalf of the Alliance for Regenerative Medicine (ARM) to express support for language to expand newborn screening in the House of Representatives’ budget proposal and to recommend passage by the .

ARM is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory, reimbursement, and manufacturing initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative medicines and advanced therapies.

In its 11-year history, ARM has become the global voice of the sector, representing the interests of 380+ members worldwide – including 13 members in Ohio with headquarters or large footprints in the state spread across major metro areas. Our members in Ohio and across the US and Europe include small and large companies, academic research institutions, major medical centers and patient groups.

As you know, the budget proposal (HB 110) approved by the Ohio House would require newborn children in Ohio to be screened for spinal muscular atrophy (SMA) and X-linked adrenoleukodystrophy, two genetic disorders included in the

1015 18th St Street NW, Suite 1102 [email protected] www.alliancerm.org Washington, DC 20036 @alliancerm

federal Recommended Uniform Screening Panel. HB 110 would also keep Ohio in step with federal guidelines by requiring the state’s newborn screening advisory council to determine whether to recommend that children in Ohio be screened for a particular disorder within six months of that disorder’s inclusion on the federal recommended uniform screening panel.

Children affected by genetic disorders are often only eligible to receive and/or benefit from transformative therapies if they are diagnosed soon after birth or in the early stages of life. The FDA in 2019 approved one such gene therapy, for children with SMA, that has shown durable, and possibly curative, treatment effects. This therapy is just one example of a coming wave of potentially life- changing medicines: The FDA expects to approve 10-20 gene and gene therapies each year by 2025. Many of the gene and cell therapies in the clinical trials pipeline target diseases for which there are no, or few, existing treatment options.

Newborn screening is therefore a vital tool to ensure that diagnoses can be made early enough for children to benefit from life-changing therapies that are either already approved by the FDA or that may be available through a clinical trial. Timely updates to the screening panel can therefore ensure that more children have access to the full range of treatment options.

These updates to Ohio’s newborn screening policies would complement the state’s creation of the Rare Disease Advisory Council in late 2020, which ARM also supported. There are more than 7,000 rare diseases and it is estimated that 1 in 10 Americans, thus 1 in 10 Ohioans, are impacted by one of them. Newborn screening is a vitally important tool to identify some of these diseases at their earliest stages.

ARM asks for your support of the newborn screening provisions in HB 110. We look forward to working with you and the original sponsor of these provisions, Representative Roemer, to advance this important initiative to the governor for his signature.

Sincerely,

Paige A. Bischoff Senior Vice President, Public Affairs

cc: Senate Finance Chair Representative Bill Roemer Ray DiRossi, Senate Budget & Finance Director