The organizations plan to continue preclinical evaluation towards the advancement of TSC1 and TSC2 programs using Apertura's TfR1 CapX™ capsid
SILVER SPRING, Md. and NEW YORK, July 30, 2026 /PRNewswire/ -- The TSC Alliance, an internationally recognized nonprofit that improves quality of life for everyone affected by tuberous sclerosis complex (TSC), announced the completion of a preclinical pilot study evaluating an adeno-associated virus (AAV) gene therapy approach for TSC1. The study was conducted in collaboration with Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines. The TSC Alliance and Apertura Gene Therapy plan to continue preclinical evaluation of TSC1 and TSC2 gene therapy programs usingApertura's capsid, TfR1 CapX™.
The investigational gene therapy approaches for TSC1 and TSC2 use Apertura's TfR1 CapX, an intravenously delivered AAV capsid designed to engage human transferrin receptor 1 (hTfR1) and support delivery across the blood-brain barrier (BBB) in preclinical models and enable broad distribution to the brain and other organs.
"We are encouraged by the initial findings from the preclinical pilot study," said Dean Aguiar, PhD, TSC Alliance Executive Vice President, Translational Research. "The results generated to date support further preclinical testing to evaluate the approach and determine its potential for advancement into clinical trials."
The TSC Alliance has committed to raise $1.76 million focused on gene therapy studies demonstrating effectiveness in TSC1 and TSC2 over the next 18 months. If successful, the TSC Alliance will begin its next fundraising phase to support IND-enabling studies and advance the program toward clinical trials. To learn more or support this initiative, visit tscalliance.org/gene-therapy.
Kari Luther Rosbeck, TSC Alliance President and Chief Executive Officer, shared, "The TSC Alliance, in partnership with the TSC community, has a long history of raising capital funds to drive TSC research. Advancing this innovative gene therapy could profoundly shape the future for people living with TSC."
Christopher Davis, PhD, Director, Nonclinical and Translational Research, Apertura Gene Therapy, said: "Apertura is proud to be developing gene therapy programs hand in hand with the TSC Alliance. This collaboration reflects Apertura's commitment to patient-centered drug development, and we are encouraged by the progress that has been made so far."
The TSC Alliance will host a session on the pathway for gene therapy research in TSC, at the 2026 TSC World Conference in Aurora, Colorado, on Saturday, August 1 at 1:00 p.m. MT.
About TSC
TSC is a rare genetic disease that affects people at all stages of life. TSC causes non-cancerous tumors to grow in different organs, primarily the brain, heart, kidneys, skin, eyes, and lungs, and can impair their function. Roughly 85% of individuals with TSC experience seizures, and two thirds of those individuals will have refractory epilepsy, highlighting the need for improved treatments to address the neurological impact of TSC.
About the TSC Alliance
The TSC Alliance is an internationally recognized nonprofit that improves quality of life for everyone affected by TSC by catalyzing new treatments, driving research toward a cure and expanding access to lifelong support. For more information, visit tscalliance.org.
About TfR1 CapX™
TfR1 CapX™ is a leading IV-administered, BBB-crossing capsid. Multiple for-profit and non-profit organizations have validated and licensed TfR1 CapX, and other groups are in discussions to license the technology. Clinical readiness has been supported by several preclinical development programs, including regulatory engagement and manufacturing by contract development and manufacturing organizations. TfR1 CapX is a proprietary, second-generation capsid that demonstrates superior CNS delivery compared to Apertura's first-generation capsid, BI-hTFR1. Research on the first-generation BI-hTFR1 capsid was published in Science.
About Apertura Gene Therapy
Apertura Gene Therapy develops genetic medicines and next-generation AAV capsids that engage human-relevant receptors, aiming to enable more effective and selective gene delivery. The company's lead capsid, TfR1 CapX™, leverages human transferrin receptor 1 to enable intravenous delivery to the brain and spinal cord. This established transport mechanism has a strong clinical track record in pediatric and geriatric populations, expanding its potential to treat serious neurological and genetic diseases. Apertura has licensed its next-generation capsids to multiple partners, with several programs expected to enter clinical trials over the next 12 months. Founded in 2021 on technology from the Broad Institute and supported by Deerfield Management, Apertura Gene Therapy is headquartered in New York City. Learn more at aperturagtx.com and follow us on LinkedIn.
Contacts
For the TSC Alliance
Dan Klein
Senior Director, Communications
(301) 562-9890
[email protected]
For Apertura Gene Therapy
[email protected]
SOURCE TSC Alliance
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