Ex-Novartis Exec Unveils Neurodegeneration Gene Therapy Start-Up AviadoBio
Lisa Deschamps Commercialized Novartis’s Zolgensma
Executive Summary
AviadoBio believes it can overcome barriers to making gene therapy work in neurodegenerative diseases, and its new CEO knows what it takes to make a commercial success in the field.
AviadoBio, a new biopharma company focused on developing gene therapies for neurodegenerative disorders, has been launched with completion of an $80m (£58.6m) series A financing round.
The London, UK-headquartered company is based on next-generation gene therapy design and neuroscience expertise from King’s College London, and its lead candidate is currently in preclinical development for frontotemporal dementia (FTD), with therapies for amyotrophic lateral sclerosis (ALS) among its other targets.
The company is not giving too much away about its gene therapy platform just yet, but has disclosed that it is using a “novel neuroanatomy-led” approach to drug delivery. This will also involve precision micro-dosing to achieve extensive gene expression throughout the nervous system.
The start-up’s launch reflects growing momentum in the broader field of neuroscience, where advances in understanding of underlying disease mechanisms, and encouragement from regulators – such as the US approval of Biogen's antibody-based Aduhelm (aducanumab) – is spurring increased investment.
AviadoBio has been co-founded by leading neurologist and neuroscientist Christopher Shaw, molecular neurobiologist Youn Bok Lee and vector biologist Do Young Lee from King’s College and the UK Dementia Research Institute.
Lisa Deschamps
The company’s newly appointed CEO is Lisa Deschamps, former senior vice president and chief business officer at Novartis Gene Therapies. Deschamps brings 25 years of industry experience, including the worldwide commercial launch of Novartis’s Zolgensma (onasemnogene abeparvovec), its breakthrough gene therapy for spinal muscular atrophy.
The $80m (£58.6m) financing round follows an initial $16.5m (£12m) seed financing, and will be used to advance AviadoBVio’s lead program and other preclinical assets, including for amyotrophic lateral sclerosis (ALS), whilst continuing to expand its key personnel.
The financing was led by New Enterprise Associates (NEA) and co-led by Monograph Capital, with participation from LSP, as well as seed investors Advent Life Sciences, Dementia Discovery Fund (DDF), F-Prime Capital, JJDC and medical research charity LifeArc.
Novartis’s Zolgensma was a milestone in the field of CNS gene therapy. The one-time treatment targets the genetic root cause of spinal muscular atrophy (SMA) by replacing a missing or non-working SMN1 gene with a new, working copy of a human SMN gene. The therapy is also remarkable because Novartis has made it a commercial success, despite its very high upfront cost. (Also see "Novartis Steers Zolgensma Towards Commercial Success In Europe" - Scrip, 12 Mar, 2021.)
However, many other recent candidates in neurodegenerative conditions, such as Huntington’s disease or ALS have failed, whether they worked through gene therapy or other modalities such as antisense oligonucleotides. (Also see "Roche Shelves Phase III Huntington's Candidate Tominersen" - Scrip, 23 Mar, 2021.)
Shaw said he believed AviadoBio’s approach could overcome one of the key hurdles to successful gene therapy in neuroscience.
“We have seen that modifying gene expression can be curative, but achieving widespread distribution is the greatest challenge. We have shown that precision micro dosing to neural networks will deliver broad CNS expression, providing safe and effective treatments,” he said.
“We are directly exploiting insights into the causes of diseases to design therapies that have the potential to cure patients for whom there are no effective treatments. I believe that AviadoBio has the potential to move neurodegeneration from palliation to prevention.”
Other companies looking for a neuroscience breakthrough include Pfizer andvSarepta, who are developing rival gene therapy candidates for Duchenne muscular dystrophy. Among those who have already faltered include Voyager Therapeutics which saw its efforts with multiple partners to develop a Parkinson’s disease gene therapy ultimately fail. However, the company recently signed a new deal with Pfizer, reviving hope that its proprietary TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) technology can overcome gene expression problems encountered by traditional AAV platforms. (Also see "Pfizer Gets AAV Capsid Rights From Voyager For Gene Therapy R&D" - Scrip, 6 Oct, 2021.)