What’s on the horizon for  neurological treatments?

Leading scientists from across North America  share how advanced therapeutics will rewrite the future for patients with the most complex diseases

October 23,  2026 |  by Sophie Lorenzo

According to the World Health Organization, neurological conditions affect more than 40% of the global population. These remain among the most complex diseases to treat, made all the more difficult by the challenge of developing therapies that can cross the blood-brain barrier.

It is not overstating to say that advanced therapeutics are poised to revolutionize neurological care by addressing genetic conditions at the source. At the Future of Advanced Therapeutics in Neurology symposium, a satellite event of the American Society of Human Genetics’ (ASHG) Annual meeting, distinguished speakers shared emerging approaches.

The event brought together 130 participants, including ASHG attendees from across Canada, the US, Asia and Latin America; PIs and trainees from McGill University, Université de Montréal and Université Laval;  members of the life sciences ecosystem in Montreal; as well as patient advocates for rare neurological conditions for a day of engaged conversations.

Expanding access to genome editing 

We were very fortunate to have Fyodor Urnov from the Innovative Genomics Institute (IGI) at UC Berkeley give the first keynote of the day. Prof. Urnov is at the forefront of pioneering novel therapeutics. He co-developed the toolbox of human genome and epigenome editing and was on the team that advanced its first-in-human applications to the clinic.

Speaking from San Francisco, he inspired the audience by discussing his work to expand “CRISPR on-demand” — leveraging academic and private sector infrastructure to create a standardized platform that would allow each new treatment to benefit from the work already completed for similar conditions, with the goal of accelerating the development of personalized genetic medicines, particularly for rare neurodevelopmental conditions.

CRU medical director Massimo Pandolfo spoke of how new therapeutics are being brought to clinical trials, and how the Neuro CRU helps investigators and industry get promising research from the lab to the patients who need it, faster.

Patients in perspective

Dr. Stalin Gill and Dr. Navpreet Sangha, founders of SPG4Hope Foundation spoke movingly about the urgent need for multi-institutional collaborations in the development of treatments for rare genetic diseases.

Their three-year-old son Gurmoh lives with a rare form or spastic paraplegia which will severely limit his mobility and independence. While cutting-edge therapies could allow us to address the root cause of a disease, the delay in producing personalized treatments can make a dramatic difference in the debilitating symptoms of patients with a progressive disease. 

Their talk was a potent reminder to the scientists in the room of the need to leverage skill sets and technological platforms across universities to accelerate therapeutic development. This is exactly what Neuro CRU executive director, Dr. Ziv Gan-Or,  The Neuro’s Platform for Advanced Clinical Therapies are doing by collaborating with leading scientists in the US and Canada to accelerate the development of a treatment for Gurmoh.

Gene editing methods and therapies

We were privileged to hear from Dr. Yong-Hui Jiang, Chief of Medical Genetics at the Yale School of Medicine about the innovative Stimuli-responsive Traceless Engineering Platform (STEP) which he and his team developed. This chemical delivery system uses tiny particles that can bypass the size and diffusion limits of viral vectors and lipid nanoparticles (LNPs). His current research is investigating its use in the treatment of Angelman’s or Willi-Prader Syndromes.

Benoit Gentil, Director of the Genetics and Epigenetics of Motor Development and Performance Lab at McGill and an associate member of the department of Neurology and Neurosurgery, shared exciting progress in developing a gene therapy treatment for Charlevoix-Saguenay Ataxia (ARSACS), the second most common autosomal recessive ataxia and an important area of research for Quebec patients. He and his team created a ‘mini gene’ using only the essential parts of the gene needed to fight the disease which fits into a viral vector for delivery to nerve cells. This approach is showing promise in tests in mouse models.  

Developments in brain delivery methods

Three stellar speakers weighed in on novel brain delivery methods,  highlighting the range of promising approaches being investigated to enable therapeutics to cross the blood-brain barrier more effectively.

Dr. Andres Lozano, a professor and neurosurgeon at University Health Network in Toronto discussed how his programme has mapped cortical and subcortical circuits in the brain. He showed striking examples of how functional neurosurgery is advancing the use of deep-brain stimulation and brain-machine interfaces to promising results in treating akinesia, rigidity and gait in movement disorders and other neurological conditions.

Derrick Gibbings, a professor in the Department of Cellular and Molecular Medicine at the University of Ottawa, presented his lab’s work on small extracellular vesicles (sEV), capable of delivering RNA between cells and across the blood-brain barrier. By packaging autophagy-targeting chimeras into sEVs, they can help degrade long-lived, complex structures like damaged mitochondria or aggregates like alpha-synuclein in neurodegenerative diseases.

A senior scientist who leads the Brain Repair Group at the Sunnybrook Research Institute in Toronto, Isabelle Aubert shared her lab’s work on MRI-focussed ultra-sound, which was shown to more effectively mediate blood-brain barrier opening. This non-invasive approach also allowed researchers to use a lower dose of therapies for effective treatment, opening a very promising avenue for further research.

Lessons from a decade of RNA therapeutics

We were honoured to have Phillip Zamore, a giant in the field and the Chair of the RNA Therapeutics Institute at UMass Chan School of Medicine to close out the day as our second keynote speaker. Many of our attendees were there to hear about a novel platform for cell type-specific RNA silencing his lab is developing using micro-RNAs. A promising avenue as these are abundant, long-lived, tissue and cell specific, and can be used to guide argonaute proteins to degrade a target mRNA with only a small patch of complementarity required to bind to the target.

He was joined by Dr. Maryam Oskoui, a pediatric neurologist and clinician-scientist from the Azrieli Research Centre at the CHU Ste-Justine with extensive experience in clinical research for neuromuscular disorders. She spoke of the complex elements that continue to make developing treatments for these conditions so challenging, despite advances in RNA therapeutics for rare diseases. Factors include variable genetic factors and heterogenous disease progression to limited natural history data and the challenge of determining relevant clinical endpoints.

Emerging research on the boards

Graduate students and post-docs from McGill, Université de Montréal and Université Laval contributed to a lively poster preserntation session. Presenters were rated on their study design, the quantity and quality of the data they collected as well as their presentation skills.  

1st place: Rahul Suresh (McGill University) for his work on “A safe, efficient, and compact single-AAV gene restoration platform via CRISPR activation for neurodevelopmental disorders.”

2nd place: Justine Schweizer (McGill University) for her study of “Nociceptor-targeted viral therapy for the treatment of inflammatory chronic pain.”

3rd place: Marina Broomfield (McGill University) for her study of the “Cellular Drivers of Edema in Glioblastoma.”

A successful scientific meeting takes more than great speakers (as privileged as we were in that regard); it takes engaged participants.  We are thankful to all those who attended for bringing insightful questions, sparking great conversations and making new connections, all with a common goal of helping to advance treatments for rare neurological disorders for all the individuals affected.