That’s a wrap!!

The 5-day international NCL2021 conference wrapped up on Sunday morning, and what a brilliant meeting it was!
With over 300 delegates (114 attending in-person), 71 speakers from around the globe and 64 poster presentations, the Batten research community is thriving indeed!
The quality of the research presented was consistently outstanding – from basic science (which investigates the molecular and cellular mechanisms of disease), right through to the translational and clinical research (which enables those scientific discoveries and potential therapies to be applied and studied in human patients).

Some of the highlights of the program were:

• Demonstration of new cellular and animal models of Batten disease to investigate disease mechanisms and to test potential new treatments. These included new stem cell and ameboid models, and pig and non-human primate whole animal models.
• Biomarker discovery studies in a number of Batten sub-types, helping to identify ‘biological indicators’ specific to Batten disease.
• Presentation of data from ongoing preclinical studies in a variety of pharmacological and gene therapy-based treatments.
• Discussion of study designs for upcoming in-human clinical trials, including gene therapies for CLN1 and CLN7 disease, and BBDF/Theranexus therapy for CLN3 disease.
• New natural history data helping to characterize the typical progression of different Batten disease subtypes.
• Clinical learnings and observation data from what is now 6 years’ experience of Brineura (cerliponase alfa) treatment for CLN2 disease.
• Discussion of new clinical assessment rating scales for different Batten disease subtypes.

A huge THANK YOU and congratulations to the NCL2021 organising team, presenters and sponsors for enabling this outstanding event.

For any queries or further information on the above, please contact Dr Ineka Whiteman, Head of Research, Medical and Scientific Affairs at research@bdsraaustralia.org