2022 Translational Research Conference for the Management of NCL’s November 3rd and November 4th , 2022

The 7th gathering of the Translational Research Conference for the Management of NCLs was on Thursday November 3rd and Friday November 4th at the Westin Chicago Lombard Hotel in Lombard Chicago. This conference organizing committee: Jill Weimer, David Pearce, Katherine White (Sanford Research), Steven & Dr. Sandra Nusinoff Lehrman (Lehrman Family Foundation), Jennifer and Tracy Van Houtan (Noah’s Hope & Hope 4 Bridget Foundation), Karan Khan & Carol Schwimmer (Fore Batten Foundation) and Dr. Chris & Heather Dainiak (Our Promise to Nicholas Foundation).

It’s hard to believe that a meeting that started 12 years ago in a small hotel conference room in Bethesda, Maryland with ~30 attendees has evolved into what we are hosting today. This year we had eighty-eight registered, thirty-two speakers and twenty-three joined virtually.  Although the agenda and attendance have grown, the initial goal has not changed: to create a forum to bring together scientists, clinicians, and patient advocates to explore new and innovative approaches for treating Batten Disease.  That initial meeting in 2010 focused entirely on treatment approaches for CLN1 and CLN2 disease, but recognizing we are all stronger together, our program and focus has evolved to include strategies for all forms of Batten disease.

Over this last decade plus, this meeting and others have been the spark for collaborative research partnerships between academia, industry and patient advocacy groups – really accelerating the path towards treatments for many forms of Batten disease. From the development and approval of an Enzyme Replacement Therapy (ERT) for CLN2 Batten disease to the more recent gene therapies and biologics being developed for other forms of Batten disease, our research community has persevered in thinking innovatively and strategically. As the march toward novel therapies continues, leading to increased clinical trials, this meeting creates a forum to ensure we are leveraging experience and know-how from both within our Batten disease community as well as from experts outside our field.

This conference was structured to promote dialog and drive conversations.  Speakers had been grouped by topics with a moderated panel discussion at the end of each session.  Jill Weimer and her committee invited a wide variety of speakers for each session, time restrictions prevent us from having everyone working in that space present.  We kicked off each panel discussion by opening the floor to the audience for anyone working in that session key focus area but who is not a speaker to provide a 2-3 min snapshot of their team’s progress.  After that, we took questions and comments from the audience and had a very productive meeting.

We also want to say thank you to all the wonderful sponsors that make this meeting possible and to say thank you to each of you for being a part of this exciting meeting.

Dr. Jonathan Cooper on behalf of 2021 NCL Conference

Noah’s Hope & Hope for Bridget Foundation

Our Promise to Nicholas Foundation

Fore Batten Foundation 

Drew’s Hope Foundation

Lehrman Family Foundation

Beyond Batten Disease Foundation




Sanford Research

Team Penelope

Taylor’s Tale Foundation

Hailys Heros Foundation

Ethan’s Reason Foundation

Garrett the Grand Batten Fighter Foundation

Precigen Exemplar

JCR Pharmaceuticals 

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