Last updated September 24, 2026

Advanced Therapy System

Developing non-viral gene therapies for rare brain and retinal diseases

Gene TherapyRare DiseaseNeurodegenerative Disease

Inherited brain and retinal diseases can progressively damage movement and sight, often beginning in childhood. Many lack treatments that correct the underlying genetic defect. Delivering useful genetic material safely and selectively into affected cells remains a major barrier.

Advanced Therapy System is developing NeurAll, a non-viral gene-therapy platform for rare neurological and retinal diseases. Its approach uses nanoparticles to carry genetic material into affected cells, with initial work involving and .

Key Investor Signals

Signed a public-research partnership for NeurAll

Fundación Progreso y Salud signed a March 2025 agreement with ATS covering development and exploitation of gene-therapy research led at .

Advanced nanoparticle testing in preclinical models

By June 2026, the research team had evaluated multiple particles for efficacy, compatibility and safety and was testing vectors in proof-of-concept studies.

Established a nearly €500,000 research-project budget

The March 2025 NeurAll agreement described a project budget close to €500,000, combining private and public resources with seed capital contributed by ATS.

Other Positive Signals

  • Gained project support from the and Mácula Retina-Gen CRB1 patient associations.

Team

Antonio López

Founder

  • Presides over ENACH, a patient association supporting families affected by rare brain-iron disorders and funding therapeutic research.
  • Took the initiative to establish ENACH in 2013 and built a patient-led organization focused on research funding and family support.
  • Led ENACH's research partnership with Pablo de Olavide University from 2014, including BPAN and drug-toxicity research lines.

Product

NeurAll is a preclinical platform using non-viral vectors to transport genetic material. Researchers at CABIMER are evaluating nanoparticle candidates for delivery to brain and retinal cells, with PANK2 and CRB1 among the initial genetic targets.

Nanoparticle gene delivery

The platform packages genetic material in nanoparticles instead of viral carriers. The research aims to improve delivery, cargo capacity and flexibility while evaluating the biological compatibility and safety of each candidate.

Brain and retinal targets

Initial work focuses on PANK2-linked neurodegeneration and CRB1-related retinal disease. Both programs seek to address inherited defects, while adapting the genetic cargo and delivery approach to the cells affected.

Preclinical candidate selection

Researchers compare candidate particles in preclinical models and run proof-of-concept tests. Results are intended to identify a lead for regulatory preclinical work before an application to begin a human trial.

Market Overview

Rare neurological and retinal disorders require gene-specific diagnosis and treatment development. Their small patient populations complicate recruitment, while irreversible loss of nerve or retinal cells makes timing important. Delivery platforms must reach the right cells and preserve function without unacceptable toxicity.

Approved retinal provides a commercial and regulatory precedent for treating inherited disease, but access and payment remain difficult. Development typically involves specialist centres, genetic testing and long-term follow-up. A new non-viral platform would need disease-specific efficacy, manufacturing and safety evidence.

Estimated PKAN prevalence
1–3 per million

Suggested worldwide prevalence of PANK2-related neurodegeneration

Retinitis pigmentosa prevalence
1 in 3,500–4,000

Estimated frequency of in the United States and Europe.

CRB1 share of early retinal disease
~10%

CRB1 accounts for about 10% of Leber congenital amaurosis or early-onset severe retinal dystrophy.

Retinal gene-therapy launch price
$850,000

US launch list price for treating both eyes with Luxturna in 2018

Comparable Outcomes

Retinal gene therapy

Gyroscope Therapeutics

Acquired by Novartis for up to $1.5B.

Developed gene therapies for retinal diseases by targeting biological pathways involved in vision loss. Novartis completed the purchase in February 2022 for $800M upfront and up to $700M in contingent milestones.

Neurological gene therapy

AveXis

IPO at ~$455M valuation. Later acquired by Novartis for $8.7B.

Developed gene replacement therapy that addressed the underlying cause of spinal muscular atrophy. Novartis completed its $8.7B cash acquisition of AveXis in May 2018, adding a gene-replacement program for spinal muscular atrophy.

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Sources

This profile was built from public company materials, ecosystem sources, market references, and professional profiles.

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