ASGCT 2026 Recap: How Personalized Gene Therapies Are Becoming Real for Rare Disease Families

Key Takeaways

• The field’s biggest honors went to individualized, patient-led medicine: the Penn and CHOP team behind Baby KJ, Timothy Yu for N=1 therapies, and Terry Pirovolakis, the father who built Elpida Therapeutics to treat his own son.

• The viral delivery science was defined by AI-driven vector design, and new strategies to target the brain. Encoded, Dyno, Apertura, and others showed meaningful improvement in difficult to target tissues like the brain.

• The ophthalmology session had largely stopped asking what these genes do. It was demonstrating how to create genetic therapies to address these mutations.

Why the Biggest ASGCT 2026 Awards Went to Individualized and Patient-Led Gene Therapy

This year, the field gave its biggest prizes to people who developed medicines for one patient at a time.

The Catalyst Award went to the team behind Baby KJ Muldoon, the infant treated with a custom CRISPR base-editing therapy for a rare urea cycle disorder: Rebecca Ahrens-Nicklas, Kiran Musunuru, Fyodor Urnov, and Danaher. It is the first time the award has gone to a group plus a company, and the first time ASGCT has ever honored a corporation for a scientific result. Accepting for the team, Musunuru did not give a victory speech. He told the room it was not a clinical trial, was not a cure, and that it is too early to know what KJ’s outcome means for the next patient. The candor provided a clear-eyed view of progress that still requires much to be done to reach more patients.

The Mendell Award went to Timothy Yu of Boston Children’s Hospital for pioneering N=1 trials and founding the N=1 Collaborative, now past 2,000 members. He argued these therapies should be treated less like small drug programs and more like surgery: custom tools and procedures for a single patient. He called it genetic surgery.

And in the patient-advocacy session, Terry Pirovolakis of CureSPG50 and Elpida Therapeutics took the stage. He got his son’s SPG50 diagnosis in 2019. He raised the money, ran the program, and treated three children in three years. He is a parent who built a drug because no one was going to build it for him.

The message was clear: the most highly respected leaders in the field are focused on blazing new trails forward for personalized medicines.

How Next-Generation Gene Therapy Delivery Is Making Treatments for Brain Safer

Today’s capsids often need very high doses to reach tissues like the brain. High doses cost more, are harder to manufacture, and carry more safety risk, mostly to the liver. These issues have historically made gene therapy for CNS disorders very difficult.

Dyno Therapeutics launched two new AI-designed delivery vectors and updated two more. Dyno-9zh is built to reach the brain through a simple IV infusion, with no injection into the spinal fluid. In monkeys it reached up to half the neurons in a key region of the cortex and stayed largely out of the liver. In addition, Dyno-n96 effectively targets muscle tissue; it hit about three quarters of skeletal muscle fibers in monkeys at a dose roughly 25 times lower than what some approved muscular dystrophy gene therapies require. A 25-fold lower dose changes who can afford to make the medicine. Dyno also showed updated data on its yp2 brain-targeting vector and opened its AI design tools to rare disease foundations and patient groups. This is especially meaningful for ultra-rare disease families, who can benefit greatly from access to these technologies.

Apertura presented data on its brain-targeting vector, TfR1 CapX, which can be manufactured at clinical scale. A vector that looks brilliant in a mouse but cannot be made consistently in large batches never reaches a child. Showing it can be made is the breakthrough that turns a research result into a medicine. Apertura also presented strong work on which animal models actually predict human results, and it is giving academic labs free access to the vector through its Open Apertura program. That same vector is already licensed into clinical programs, including AviadoBio’s AVB-406 for Alzheimer’s.

Both Dyno’s yp2 vector and Apertura’s TfR1 CapX latch onto the transferrin receptor, called TfR1, a shuttle the body already uses to carry iron into the brain, and ride it across the blood-brain barrier. They are not alone, as an increasing number of delivery-focused companies are utilizing this and similar mechanisms to effectively cross the blood-brain barrier. Historically, this has been a critical limitation for CNS therapies, so this level of progress is highly encouraging for the field.

Gene Therapy for Inherited Retinal Diseases is Moving From Research to Real Treatments

In past years at ASGCT, retinal disease sessions focused on discovery science, seeking to clarify the impact of various genetic factors in disease symptoms and progression.

This year, it was clear across numerous talks that the entire field had moved forward. Rather than focusing on discovery, multiple sessions highlighted transitional science and mid preclinical stage, across a range of animal models, with the discussion centered on dose, delivery route, durability, and the path to an IND. Inherited retinal diseases like retinitis pigmentosa, Stargardt, and Leber congenital amaurosis came up less as scientific puzzles and more as engineering problems with clear next steps.

Examples of this progress include Beacon Therapeutics’ laru-zova, a gene therapy for X-linked retinitis pigmentosa caused by RPGR mutations, which finished enrollment in its pivotal VISTA trial in 2025, dosed its first patients in the bilateral LANDSCAPE study, and has 12-month data expected in the second half of 2026.

How Rare Disease Families Can Access Gene Therapy Today

The field spent a decade asking whether individualized medicine could work. This year it gave that work its top awards and showed the delivery tools to scale it. For families, the variable that has always mattered is time. Every lower dose, every capsid that can actually be manufactured, every program already in preclinical development gives the next family months back.

If your child has a confirmed genetic diagnosis and you want to understand what is possible, submit it to Nome for a free evaluation. We assess which modalities apply to your child’s specific mutation, estimate timeline and cost, and identify the execution path. No charge. No commitment.

About Nome

Nome Therapeutics is the Operating System for Personalized Therapeutics, turning every rare disease patient’s genome into an actionable treatment plan and helping execute on it at the lowest cost and highest speed in the industry.

REFERENCES

ASGCT. “Congratulations to our 2026 Honorific and Funded Award Winners.” February 2026.

Inside Precision Medicine. “ASGCT 2026: Timothy Yu Wins Jerry Mendell Award For N-of-1 Therapies.” May 2026.

GEN. “It Was Not a Cure: Musunuru Cautions ASGCT on Baby KJ Promise.” May 2026.

ASGCT Annual Meeting. Keynote and General Session Speakers.

Dyno Therapeutics. “Two New AAV Capsids and AI Platform for Rare Disease Therapeutic Development at ASGCT 2026.” May 2026.

Apertura Gene Therapy and the Broad Institute. “TfR1 CapX Data at ASGCT 2026.” April 2026.

AviadoBio. “Licensing Agreement for Apertura’s TfR1 CapX.” April 2026.

Beacon Therapeutics. laru-zova (laruparetigene zovaparvovec) for X-linked retinitis pigmentosa, VISTA and LANDSCAPE trial program. 2025-2026.

This article is for informational purposes only and does not constitute medical advice.