Virtual Conversation: The Promise of Gene Therapy for Rare Disease in Texas


Eli Kirshbaum


State of Reform was proud to host an informative conversation with Amber Freed, founder and CEO of SLC6A1 Connect, Steven Gray, director of the UTSW Viral Vector Facility, and Brenda Gleason, president of M2 Health Care Consulting, about gene therapy’s capacity for treating individuals living with rare diseases in Texas.

These leaders sat down with us to discuss the obstacles associated with accelerating the use of gene therapy, including high costs and cross-system collaboration, as well as why these innovative therapies are worth putting so much time and resources into.


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Gleason spoke about the payment challenges associated with gene therapy, explaining that the current healthcare payment system isn’t designed for large, upfront, one-time payments, but rather more long-term payments. She recommends an “annuity” payment model for these therapies, in which payers pay for the treatment over a long period of time.

“Maybe something happens between a state, or a group of states, and a manufacturer, to say, ‘We’ll pay you a little bit, but over time. We’re not paying for the entire payment upfront, even though that’s how the patient would receive it.'”

She explained that Texas is one of around half of US states who have required insurance coverage of biomarker testing, which helps expedite the diagnosis of rare diseases.

Gray discussed potential solutions for navigating the high risk of bringing these expensive therapies to market; manufacturing costs, research and development costs, and all the other expenses involved in bringing the drug to market, coupled with the fact that there are very few precedents of approved gene therapies to draw from, make the task quite financially risky for companies. He cited the promising work of the Bespoke Gene Therapy Consortium in accelerating development of these therapies, which will help reduce this risk over time.

“Right now, there are only very few gene therapy products that are fully FDA-approved and marketed. So I think right now, any company is going to be very justified to say, ‘When we went into this, there was a high risk of failure.’ There weren’t a lot of precedents that they could rely on.

So I think any [companies] that are trying to develop these drugs, they are taking on a significant amount of risk right now, and some of that is regulatory risk, unknown risks … But as more drugs are approved, and there’s more precedents that are set, then hopefully this whole process gets streamlined, and then that essentially de-risks things, which, if you de-risk things, then it brings down costs.”

Freed—who has a personal stake in the topic as the mother of a son with a chronic disease—mentioned how difficult it can be to get a rare disease diagnosed, the effect this has on children living with rare diseases, and how widespread newborn screening can help.

“Oftentimes, just the journey to get a diagnosis is more expensive than doing genetic testing … Newborn screening is the gold standard we are all striving for, especially for children, because 50 percent of all rare diseases impact children, and only 30 percent will live to see their fifth birthday. Newborn screening is very difficult because it’s a state-by-state initiative …

Typically you’re not allowed to get a gene on the [newborn screening] panel until there’s a treatment existing, or a great standard of care for that condition, which takes a lot of time and falls on the back of a lot of patient organizations where we all volunteer and are stretched very thin.”

She recommended that Texas consider initiatives such as Project BabyDillo that would support full genome sequencing for infants born in the state requiring neonatal or pediatric intensive care unit as many of these infants have genetic conditions and full genome sequencing can identify genetic disorders in days versus years.

You can watch the full conversation above.