FAQ
Most frequent questions and answers
The dose of MaavRx is small and is delivered locally to a small area of the brain. Animal studies have shown this dose is well tolerated with no evidence of toxicity. Similar AAV gene therapies delivered locally to the same area of brain or other indications have already been shown to be safe and well tolerated in children and adults. Indeed, the first AAV gene therapy administered in this way has already been approved by the FDA in the USA, the EMA in Europe and other national regulatory authorities.
The liver toxicity reported with AAV gene therapies only occurs when very high doses are given to whole body via intravenous injection. This is not relevant to MaavRx therapy. The per patient dose of MaavRx is over a thousand times less than the dose associated with severe toxicity.
Most other gene therapies have been developed for very rare diseases (“Orphan” indications). Many of these also require high doses delivered in large volume to the whole body.
In contrast MaavRx is being delveloped for a very common and growing indication. Preclinical studies suggest that each patient will require only a very small volume at a comparatively low concentration.
The combination of a high number of patietns and a low dose allows for large scale production with great economoies of scale.
Our strategic intention is to ensure that MaavRx will be more cost-effective alternative than continuous infusion of L-DOPA.
MaavRx patents are approved in the USA and Europe.
MaavRx Ltd has filed other patent application worldwide.