About Us

TNC 2025 Highlights

In the past year, the Zander Wyss TNC maintained its global leadership in neurodevelopmental clinical research. We expanded clinical trials, published discoveries in high-impact journals, and launched major international collaborations to advance therapies for rare disorders.

Here is a summary of this year’s accomplishments.
Download the PDF of our full Annual Report.

Scope of Research
Over 30 rare and ultra-rare neurodevelopmental disorders under investigation.

Clinical Trials
80 active clinical trials >

Publications
170 peer-reviewed articles since 2021. Contributions in 2025 advanced gene editing, neurogenetics, and translational neuroscience in leading journals.
Publications >

IMPACT Network
In 2025, Boston Children’s Hospital received a $7.5 million award to help lead the Aligning Research in Autism IMPACT Network, a global clinical trials infrastructure accelerating precision therapies for neurodevelopmental disorders. We serve as co-leader of the Clinical Coordinating Core.                          Read more about the IMPACT Network >

Clinical Research Unit
The Clinical Research Unit at Two Brookline Place, established in 2024, is now fully operational. This unit supports a growing portfolio of studies, providing integrated services for pharmaceutical, device, and behavioral trials. To date, 28 principal investigators have conducted study visits in our purpose-built, patient-centered space.
Read more about the Clinical Research Unit >

TNC Faculty
With the 2025 hiring of Dr Audrey Thurm, our full-time dedicated faculty is up to six principal investigators.
Visit our Faculty page >

Grants Giving
Our investment in early-stage innovation continues to drive impact, with pilot-funded investigators securing major follow-on funding and national recognition, while fellows advance into independent research careers.
Read more about TNC Pilot Grants >

TNC 2024 Highlights

In our fourth year we accomplished a significant expansion in the scope and output of our research community, new research awards, and the inauguration and operation of our Clinical Research Unit.

Clinical Trials
We are actively investigating 23 rare and ultra-rare brain disorders, with 66 clinical trials underway, and 24 additional trials prepared to launch.
Active Clinical Research Studies >

Publications
Publishing multiple basic, clinical and translational research projects focused on neurodevelopmental disorders in 2024
Publications >

Events
On September 23, 2024, we hosted over 200 scientists, clinicians, patient advocates, and families in Boston for our fifth full-day symposium entitled “Paving the Path: Clinical Trial Readiness and Developing Therapies for Rare Genetic Disorders”.
TNC 2024 Symposium >

Pilot Grants
Awards of $100,000 each have been made to three new projects.
TNC Pilot Grants >

TNC Clinical Research Unit
2024 saw the opening at our Two Brookline Place headquarters of state-of-the-art facilities for observational studies and therapeutic clinical trials specific for neurodevelopmental disorders. The new space is dedicated to research on rare and ultra-rare neurogenetic disorders, paving the way for later-stage external investment in new therapeutics.
Clinical Research Unit >

TNC 2023 Highlights

The 2023 report highlights key achievements in our second year of operations including:

Clinical Trials
Launching a new gene therapy trial for girls with Rett Syndrome in partnership with Neurogene
Opening a Clinical Research Unit at 2 Brookline Place for clinical trial study visits

Faculty Members
Recruiting three brilliant new faculty members: Mandana Arbab, Jordan Farrell and Emily Osterweil

Events
Partnering with Global Genes and hosting a panel on the “Power of Partnering” at the Rare Advocacy Summit on Collaborative Research for Neurodevelopmental Disorders

Training Initiatives
Hosting a translational neuroscience fellowship program
Awarding 4 TNC Pilot Grants
Launching a new Clinical Psychology Advanced Graduate Practicum

TNC 2022 Highlights

The 2022 report highlights key achievements in our second year of operations including:

Symposium
Hosting an inaugural symposium entitled Gene Therapy for Pediatric Neurological Disorders and multiple community/science meetings  LEARN MORE >

New Advisory Council
Launching a Neuroscience Family Advisory Council research committee to ensure our clinical research includes the patient and family perspective LEARN MORE >

Faculty Members
Recruiting two new faculty members to lead TNC Cores: Dr. Jed Hubbs, Director of Medicinal Chemistry Core and Dr. Maya Chopra, Director of Translational Genomic Medicine Core

Pilot Grants
Awarding two new $100,000 TNC Pilot grants

Human Neuron Core
Sharing of standard operating procedures and tools developed by the Human Neuron Core

Publications
Publishing a seminal paper proposing a new framework for evaluation of disorders for development of gene therapy, GENE TARGET  READ THE PAPER >

TNC 2021 Highlights

Leadership Team
Key appointments: Executive Director, new Core Directors, Strategic Advisory Council.

Fellowship Awards
Two Translational Neuroscience Fellowships: the postdoctoral training program is two years in duration.

Pilot Grants
Four $100,000 awards to principal investigators for one-year TNC Pilot grants, with the possibility for a renewal the subsequent year.

Scientific/Family Conferences
We partner with patient advocacy groups to plan and conduct meetings between families and researchers. Learn more about TNC Events.

New Gene Therapy Core
Completed national search for two new faculty. Read more about our Core Facilities.

New Clinical and Research Space
The TNC Research Center at 2 Brookline Place opens in July 2023.