Our novel gene therapy utilizes an adeno-associated virus (AAV9) to deliver a functional copy of the faulty BBS gene in key tissues.
Since AAV is not known to cause human disease and can be tightly controlled (it does not replicate like disease-carrying viruses), it has been the gene delivery method of choice for a number of therapies including Luxturna for retinal disease and Zolgensma for spinal muscular atrophy with 149 AAV-based clinical trials underway/complete.1
Available clinical data, covering more than 3,000 people treated over more than 20 years, indicate that AAV gene therapy is a well-tolerated and efficacious form of therapy.1
We will utilize synergies from BBS1 to bridge our other pipeline programs, expediting our commercial opportunities.
|Disease: BBS1||Program: AXV101||
|Disease: Undisclosed Ciliopathy Asset||Program: AXV201||
|Disease: Undisclosed Ciliopathy Asset||Program: AXV301||
In preclinical studies, our BBS1 novel gene therapy modified the underlying disease of BBS, rescuing vision loss by halting retinal degeneration, stopping BBS-induced weight gain and the development of obesity.1,2
Human phase 1 trials are anticipated to enrol at end of 2024.View all data
Intracerebroventricular (ICV) delivery of an Adeno-associated virus 9 (AAV9) expressing human BBS1 (hBBS1) is able to ameliorate the neurometabolic phenotype in the Bbs1M390R/M390R mice.More info
Our results show that a precise analysis of different constructs and codon-optimised sequences, is a requisite first step to test new constructs for AAV vectors before assessing for actual pharmacological efficacy.More info
The low levels of expression of BBS1 in photoreceptors may explain the inability to successfully rescue the retinal phenotype. We therefore explored the therapeutic effect of delivering BBS1 to the retinal pigmented epithelium cells, which are highly ciliated and crucial for photoreceptor survival.More info
1Goswami R, et al. Front. Oncol. 2019;9:297. 2Fernandes Freitas Martins M, et al. ESCGT; 19–22 Oct 2021, Virtual Congress. 3Jeyabalan-Srikaran J, et al. ESCGT; 19–22 Oct 2021, Virtual Congress.