AviadoBio Announces Agreement with Apertura Gene Therapy to License TfR1 CapX™, a Next-Generation Blood-Brain Barrier-Crossing Capsid

AviadoBio Ltd. (“AviadoBio”) and Apertura Gene Therapy (“Apertura”), a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, today announced a licensing agreement for Apertura’s TfR1 CapX™, a novel, intravenously-delivered AAV capsid designed to target human transferrin receptor 1 (hTfR1), cross the blood-brain barrier, and enable broad distribution to the brain and spinal cord.

Under the agreement, AviadoBio will leverage TfR1 CapX to drive the development of its pipeline of genetic medicines, built on AviadoBio’s proprietary vMiX™ RNAi platform. This includes AVB-406, an investigational, preclinical stage gene therapy for Alzheimer’s disease and other tauopathies.

“This agreement with Apertura strengthens a key component of our strategy to develop transformative genetic medicines for neurodegenerative diseases,” said Lisa Deschamps, Chief Executive Officer, AviadoBio. “We believe this agreement enhances our ability to advance AVB-406 and broadens the potential of our vMiX platform in Alzheimer’s disease, other tauopathies, and additional high-need indications.”

TfR1 CapX has demonstrated widespread transduction of neurons and astrocytes throughout the brain and spinal cord, as shown in preclinical testing conducted by AviadoBio and independently by several groups.

“TfR1 CapX has been designed to specifically target human TfR1, the most extensively studied, clinically validated brain shuttle receptor,” said Alex Bloom, Ph.D., Chief Technology Officer, AviadoBio. “Our agreement to license TfR1 CapX enhances our ability to advance our vMiX gene-silencing platform to enable one-time, IV-delivered gene silencing in rare and common disorders with high unmet medical need.”

AviadoBio’s proprietary vMiX platform is designed to enable targeted, durable gene silencing using AAV-delivered RNA interference. By harnessing the natural cellular mechanism of RNA interference, vMiX has the potential for lifelong reduction of disease-causing gene expression following a single administration. The platform is intended to support flexible therapeutic approaches, including the ability to target one or multiple genes, and is being applied across AviadoBio’s pipeline of genetic medicines for neurodegenerative diseases.

“We are thrilled that AviadoBio has selected TfR1 CapX to pair with its vMiX platform, which has shown compelling potential to address the root causes of neurodegenerative diseases,” said Dave Greenwald, Ph.D., Managing Director at Deerfield Management and Executive Chairman of the Board at Apertura Gene Therapy. “We believe this combination has the opportunity to provide transformational benefit to patients in a safe and scalable approach.”

In addition, AviadoBio will present three oral presentations on AVB-406 at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting taking place May 11-15, 2026 in Boston. These presentations will highlight preclinical, translational, and manufacturing data from the program, which will leverage TfR1 CapX as part of its ongoing development. Presentation details are as follows:

Preclinical development of AVB-406: An intravenous AAV-miRNA achieving sustained MAPT knockdown for the treatment of Alzheimer’s disease

  • Session: Neurologic diseases IV
  • Presenter: Carlos J. Miranda, Ph.D.
  • Date: Wednesday, May 13, 2026 at 4:00 PM EDT

Development and translational validation of AVB-406: An intravenous AAV-delivered miRNA targeting MAPT for the treatment of Alzheimer’s disease

  • Session: Neurologic diseases V
  • Presenter: Romain Joubert, Ph.D.
  • Date: Thursday, May 14, 2026 at 10:45 AM EDT

Development of a scalable platform production process for AVB-406, a blood–brain barrier–crossing AAV vector for MAPT knockdown in Alzheimer’s disease

  • Session: AAV downstream manufacturing II
  • Presenter: Andrea Martorana, Ph.D.
  • Date: Thursday, May 14, 2026 at 4:00 PM EDT

About AviadoBio

AviadoBio is relentlessly chasing cures by developing and translating groundbreaking science and precision delivery into life-changing medicines across neurodegenerative diseases. Powered by a precision approach built on three integrated levels — payload, target and delivery — the company is advancing a pipeline of targeted, one-time AAV gene therapies across frontotemporal dementia with GRN mutations (FTD-GRN), retinitis pigmentosa (RP) and other retinal dystrophies, as well as Alzheimer’s disease and related tauopathies.

Founded in 2019 from pioneering research at King’s College London and the UK Dementia Research Institute, AviadoBio combines deep neuroscience expertise with proven biopharma leadership. Its capabilities include gene supplementation, optogenetics, and its proprietary vMiX™ RNAi gene-silencing platform, enabling durable, disease-modifying approaches across multiple degenerative conditions. Headquartered in London, the company operates across the UK and the U.S. and is backed by leading global life sciences investors and strategic partners.

For more information, please visit www.aviadobio.com and follow us on X @AviadoBio and LinkedIn at AviadoBio.

About Apertura

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 Company, Apertura Gene Therapy is headquartered in New York City. Learn more at aperturagtx.com and follow us on LinkedIn.

About vMiX™

vMiX™ is AviadoBio’s proprietary vectorized RNA interference (RNAi) platform designed to enable one-time, durable gene silencing using AAV delivery. Unlike non-vectorized RNAi approaches that require repeat dosing, vMiX is engineered to provide sustained suppression of disease-causing genes following a single administration. The platform combines potent knockdown, cell-specific expression, and the flexibility to target multiple genes or incorporate knockdown-and-replace strategies, supporting the development of next-generation genetic medicines across a range of diseases.

About TfR1 CapX™

TfR1 CapX™ is a leading IV-administered, BBB-crossing capsid. Multiple for-profit and non-profit organizations have validated TfR1 CapX and 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 (CDMOs). 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.

AviadoBio and Apertura partnership in gene therapy, featuring TfR1 CapX™ and vMiX™ platforms for CNS treatment

Contact

For AviadoBio:
Farah Speer
SVP, Head of Communications and External Relations
[email protected]
+1-312-543-2881

For Apertura Gene Therapy:
[email protected]

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