Apertura Gene Therapy to Provide Lexeo Therapeutics Access to TfR1 CapX™ for Program Designed to Treat Friedreich Ataxia

Apertura Gene Therapy will provide Lexeo Therapeutics (LXEO) access to its intravenously delivered TfR1 CapX™ AAV capsid for a program designed to treat Friedreich ataxia.

Rhea-AI Summary

Apertura Gene Therapy will provide Lexeo Therapeutics (LXEO) access to its intravenously delivered TfR1 CapX™ AAV capsid for a program designed to treat Friedreich ataxia.

TfR1 CapX is engineered to target human transferrin receptor 1 and cross the blood-brain barrier to enable central nervous system gene delivery. Apertura describes TfR1 CapX as a proprietary, second‑generation capsid that shows superior CNS delivery versus its first‑generation BI‑hTFR1 capsid and notes that multiple organizations have validated and licensed the platform. Lexeo highlights that access to TfR1 CapX may allow evaluation of less invasive routes of administration to the CNS in Friedreich ataxia, alongside its existing LX2006 program.

Market Context

On Sep 22, Lexeo named Apertura in collaborations for sequential dosing and repeat administration co...

Analysis

On Sep 22, Lexeo named Apertura in collaborations for sequential dosing and repeat administration complementary to LX2006; this release specifies TfR1 CapX access for its Friedreich ataxia program.

Historical Context

1 past event · Latest: Sep 22

1 event

1. Sep 22

   Apertura collaboration

   24h Move

   -0.3%

   Apertura collaboration was included among three Lexeo collaborations to support FA program development.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

cns, cdmos, aav capsid

3 terms

cns medical

"a less invasive route of administration to the CNS"

CNS stands for the central nervous system, the brain and spinal cord that control thought, movement and bodily functions. For investors, CNS-focused products and research matter because therapies aimed at this “delicate wiring” are scientifically challenging, often carry higher development and regulatory risk, and can take longer to prove safe and effective — but successful treatments also tend to command large markets and premium pricing.

cdmos technical

"manufacturing by contract development and manufacturing organizations (CDMOs)"

Contract Development and Manufacturing Organizations (CDMOs) are independent companies that help drug and biologic developers by designing, testing, scaling up and producing medicines on a contract basis — think of them as a hired factory and research partner that turns a medical idea into a manufacturable product. They matter to investors because they provide steady, service-based revenue, spread development and production risk for drug makers, and their capacity, quality and pricing can directly affect the speed and cost of getting medicines to market.

aav capsid technical

"develops genetic medicines and next-generation AAV capsids"

AAV capsid is the protein shell that surrounds and protects the genetic payload in an adeno-associated virus used for gene therapies; think of it as the delivery truck or envelope that carries medicine to target cells. It matters to investors because the capsid determines how well the therapy reaches the right tissue, how the body’s immune system reacts, how easy the product is to manufacture at scale, and what patents or competitive advantages a developer may hold.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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NEW YORK, Sept. 23, 2026 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, will provide Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage company focused on reshaping the path of genetic diseases with high unmet need, access to TfR1 CapX™ for a program designed to treat Friedreich ataxia. Apertura's TfR1 CapX is an intravenously delivered AAV capsid designed to target human transferrin receptor 1 (hTfR1) and cross the blood-brain barrier.

"As an intravenously delivered capsid designed to cross the blood-brain barrier, TfR1 CapX holds the potential to help unlock new approaches to treat Friedreich ataxia," said Diego Garzón, Ph.D., Chief Business Officer at Apertura Gene Therapy. "We're proud to support Lexeo's research and development efforts and believe that this agreement can make a meaningful difference for the Friedreich ataxia patient community."

Friedreich ataxia (FA) is a genetic, progressive, degenerative multi-system disorder with a prevalence of approximately 1:50,000 in the United States. It is caused by a mutation in the FXN gene that disrupts the normal production of the protein frataxin, which is critical to the function of mitochondria in a cell and to the maintenance of cardiac function. Absence of fully functional frataxin leads to damage to peripheral nerves and the parts of the brain that control movement and balance, leading to neurological symptoms that include impaired muscle coordination, or ataxia, that worsen over time.

"We are excited to partner with Apertura Gene Therapy and gain access to this promising technology," said Louis Tamayo, Chief Financial Officer of Lexeo Therapeutics. "This agreement expands the tools available to us as we evaluate innovative approaches to address the significant unmet needs of patients living with FA, including the potential for a less invasive route of administration to the CNS following initial systemic administration of LX2006."

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 (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.

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.

About Lexeo Therapeutics

Lexeo Therapeutics is a New York City-based, clinical stage genetic medicine company dedicated to reshaping heart health by applying pioneering science to fundamentally change how cardiovascular diseases are treated. The Company is advancing a portfolio of therapeutic candidates that take aim at the underlying genetic causes of conditions, including LX2006 in Friedreich ataxia (FA), LX2020 in plakophilin-2 (PKP2) arrhythmogenic cardiomyopathy, and others in devastating diseases with high unmet need.

Contacts

For Apertura Gene Therapy
info@aperturagtx.com

For Lexeo Therapeutics
Media Response:
Media@lexeotx.com

Investor Response:
Ashley Kaplowitz
akaplowitz@lexeotx.com

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SOURCE Apertura Gene Therapy

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What is TfR1 CapX™ and how is it positioned in development?

TfR1 CapX™ is an intravenously administered AAV capsid designed to target human transferrin receptor 1 and cross the blood-brain barrier for gene delivery to the brain and spinal cord. It is described as a proprietary, second‑generation capsid that provides superior CNS delivery compared with Apertura’s first‑generation BI‑hTFR1 capsid. Several preclinical development programs, regulatory engagement, and manufacturing work with contract development and manufacturing organizations have supported its clinical readiness, and multiple for‑profit and non‑profit groups have already validated and licensed the technology.

How does this agreement relate to Lexeo’s existing Friedreich ataxia work?

Lexeo states that access to TfR1 CapX expands the tools available as it evaluates new approaches for patients with Friedreich ataxia. The company notes that the technology may enable exploration of a less invasive route of administration to the central nervous system following initial systemic administration of its existing candidate LX2006.

What is Friedreich ataxia as described in the announcement?

Friedreich ataxia (FA) is described as a genetic, progressive, degenerative multi‑system disorder with a prevalence of approximately 1:50,000 in the United States. It is linked to mutations in the FXN gene that disrupt production of frataxin, a protein important for mitochondrial and cardiac function. The absence of functional frataxin damages peripheral nerves and parts of the brain that control movement and balance, leading to neurological symptoms such as worsening impaired muscle coordination, or ataxia.