In this video, Charles Sabine talks us through the highlights from the CHDI Conference that took place between 28 February and 03 March in Palm Springs, California.


This annual conference is a special event for the Huntington's community that brings researchers together for three days of innovation, inspiration and hope. Last year it took place remotely due to the pandemic but this year returned to a much-needed in-person event.


In this video Charles spoke to Peter McColgan MD and Lauren Boak PhD from Roche and Michael A Panzara MD from Wave Life Sciences about the halting of both trials that shook the community in an already difficult time.


Brian Beers BSc spoke on behalf of PTC Therapeutics about their current trials using less invasive dosing methods including liquid and pills that could be administrated at home.


Beth Borowsky PhD and Rajeev Sivasankaran from Novartis spoke about how their orally administrated drug, branaplam differs from the more invasive ASO (gene silencing) treatments and where they are at with their phase 2 study, VIBRANT-HD.


Charles also spoke to Ricardo Dolmetsch from uniQure about their study that is currently being trialed on 20 patients. This one-dose gene therapy treatment involves an adeno-associated virus (AAV) being surgically placed into the degenerating part of the brain in order to lower the Huntingtin gene.


A quote that we feel really sums up the dedication of the researchers within the Huntington's community came from Ricardo Dolmetsch. He said:

"I come to this meeting and I am so impressed by all the developments, the enthusiasm of all the scientists, but also all the technology that is being brought to bear on the disease. Diseases are never cured by any individual or even single company, it requires this really complicated ecosystem and you need as many shots on goals as you possibly can. I've spent my entire life on genetic diseases of the brain and I think this is one of the best organised communities. I've seen more effort dedicated here than I've seen in a lot of places. While there have been some failures that have been deeply disappointing, I think we've learned a lot from those failures." 


You can watch Charles' full update from the conference below.