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Luke Rosen had hoped a new treatment would slow the progression of his daughter’s KIF1A Associated Neurological Disorder (KAND), but was amazed to see an apparent reversal of her rare disease symptoms.
“She used to wake up and say my feet feel like they’re on fire inside,” but by the second dose of the new drug, her mornings had changed dramatically, Rosen, founder of KIF1A.ORG, told an Oct. 28 panel at the BIO 2025 Patient Advocacy Changemakers Event (PACE) in Washington, DC.
“The neuropathic pain: We haven’t had that as part of our life in three years now. Three years without our child writhing in pain every morning—without my son, who’s two years older, having to walk into the living room to see his sister on the ground in severe pain,” Rosen said.
Rosen’s daughter Susannah is taking an ASO (antisense oligonucleotide) drug, one of a new class of treatments called Genetically Targeted Technologies (GTTs). The PACE panel, entitled The Next Small Thing (Small Molecules, Big Promises), outlined the promise of these drugs and the need to protect incentives for their development through policy, like the MINI Act that panel moderator Emily Roberts, Director – Issue Advocacy – Genentech highlighted.
PACE is an annual event organized by the Biotechnology Innovation Organization (BIO) to bring together the biotech industry and patient advocates with the goal of forwarding patient access to medical innovation. The panel on GTTs offered a reminder of the importance of patients as a motivation for biotech.
Rosen recalled preparing for the PACE discussion with a Zoom call that included panelist Holly Kordasiewicz, Senior Vice President, Neurology – Ionis Pharmaceuticals.
“We were actually in the hospital with Susannah when I was on the panel prep call, and Holly reached over to her desk and said, ‘Look, I have a picture of Susannah and her service dog that’s always on my desk,’ ” said Rosen, his voice filling with emotion. “And that was so heartwarming for me because I realized that all of these innovators, Holly and people like you who are developing these treatments, don’t forget how urgent this is.”
What are GTTs?
Kordasiewicz opened the panel by explaining the science of how GTTs address problems caused by genetic mutations. “In the central dogma of biology, you start with DNA. DNA is made into RNA. RNA is made into protein,” she began.
“Most small molecules are targeting the protein. That’s the end of that process,” said Kordasiewicz. “Now, if you go that step earlier, to the RNA, it’s different. If you know the DNA sequence, you know the RNA sequence. And so it then becomes very straightforward to make a drug that’s upstream in the disease process.”
After about 30 years of development “and foundational science,” GTTs are now being used as medicines, such as the ASO drug Susannah Rosen takes for her KAND. Other GTTs include a type called small interfering RNA (siRNA).
Why GTTs matter
Kordasiewicz’s company, Ionis, is currently developing a range of GTT drugs for neurologic and cardiometabolic diseases such as spinal muscular atrophy. The panel in particular highlighted one important GTT with Food and Drug Administration (FDA) approval for amyotrophic lateral sclerosis (ALS) who have a mutation, or change, in the superoxide dismutase 1 (SOD1) gene.
The approval of this ALS drug “is changing the game, not just for ALS, but for the entire neurodegenerative disease space,” according to panelist Melanie Lendnal, Senior Vice President of Policy & Advocacy – ALS Association.
“Before this was approved, we all in the neurodegenerative disease space thought that the goal was to slow progression of the disease,” she said. “What we found is it is not just stopping the progression, but in 40% of people who are treated early in their journey, they’re actually regaining function. This was something that was out of the realm of possibility in the neurodegenerative disease space.”
Lendal described the challenges of ALS
“Between two to five years from diagnosis, you lose the ability to eat, talk, walk, eventually breathe, but your mind is all there the entire time. So in many ways, it’s one of the cruelest diseases,” Lendal said. “The whole family is impacted. We have kids as young as four, five years old who are giving their parents their medications and helping them get through the day.”
For reasons we still don’t understand, perhaps due to some kind of toxic exposure, one in eight of those diagnosed with ALS are military veterans, even though veterans only make up about 1% of the population, according to Maureen Elias, Government Relations Deputy Director – Veterans – Wounded Warrior Project.
“It’s like being in the military alone gives you 1.5 to 2 times the risk of contracting ALS,” Elias said.
“A dear friend of mine, Lauren Price, a combat veteran who served in Afghanistan multiple times and was a huge advocate for our community, helping raise awareness of toxic exposures, was diagnosed with ALS and was gone in nine months,” Elias said. “So what we lose is this huge contribution of people to our nation because of this disease that takes them away so quickly and so fast. And not just the veterans themselves, but then we pull people from our workforce and from our communities to care for those who are living with ALS.”
Elias said more research is needed to understand the high incidence of ALS among veterans. She said veterans also need better screening for conditions like ALS or breast cancer that are apparently influenced by toxic exposures experienced by people who served in the military. In general, doctors need to understand the unique needs of veterans.
“No one’s asking a veteran in a doctor’s office if you served in the military, so they’re not being screened for things that they should be screened for,” Elias said.
How policy could help
Given the remarkable results of GTTs in addressing a range of ailments, panelists urged policies that protect innovation and help us realize the full potential of these new drugs.
“The technology and the science is there,” Kordasiewicz said. “It’s just a matter of applying it and then having the right policy so that we can advance these ideas forward.”
Despite the benefits of GTTs, and despite their complexity, the law currently classifies them as “small molecule” drugs. Under the Inflation Reduction Act (IRA) of 2022, the small molecule drugs that make up the vast majority of drugs on the market have a maximum of nine years’ protection from price controls, while biologics—which are often more complex to develop—receive 11 years of protection.
Investors have already become more hesitant to back small molecule drugs since the IRA, and there are policy proposals to address the problem, including the EPIC Act, which would give all small molecule drugs 11 years of protection from price controls. Another proposal, the MINI Act, is specifically targeted at giving GTTs the same protections as biologics, as they share many traits with biologics, including that they are complex to develop and their novelty means they need more support for development.
“In order to support this type of innovation, we need public policies that keep up with the science and encourage continued investment in this space. There is current legislation in Congress right now that does exactly that. It’s a bill called the MINI Act that stands for Maintaining Investment in New Innovations,” said panel moderator Roberts.
She said an additional two years of protection from IRA price controls “is a critical shift that would give GTTs the runway they need and allow the innovation and promise of patients to continue.”
Rosen also noted the importance of continued research, and policy to protect that research.
“My urgent call to action is to really put your shoulders behind things like genetically targeted treatments, therapies, and the MINI Act,” he told the PACE audience of patient advocates and members of the biotech industry, “because without all of this, we wouldn’t be where we are right now.”
