Myrtelle gets ILAP status from UK for Canavan disease therapy candidate

Myrtelle has secured innovative licensing and access pathway (ILAP) designation from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for its gene therapy candidate, rAAV-Olig001-ASPA, to treat Canavan disease (CD).

The newly granted ILAP helps Myrtelle to gain quick access to much-needed development services and tools in the UK for life-threatening or seriously devastating conditions.

These services cover possible recurrent interactions with MHRA, accelerated evaluation of marketing authorisation applications, new and flexible licensing routes and engagement in market access events.

Myrtelle Global Regulatory Affairs senior vice-president Nancy Barone Kribbs stated: “The ILAP designation by MHRA for rAAV-Olig001-ASPA provides access to tools that aim to accelerate the development of this potential treatment for children with Canavan disease who currently have significant unmet medical needs and are without approved treatment options.”

Myrtelle is developing rAAV-Olig001 in a Phase l/ll clinical trial for CD, a fatal childhood genetic disorder characterised by the collapse of white matter in the brain due to a mutation in the aspartoacylase gene (ASPA). This affects the creation of myelin, leading to a deficiency in the aspartoacylase enzyme.

rAAV-Olig001 is a new vector from a class of recombinant adeno-associated viruses (rAAVs) that selectively target oligodendrocytes, the cells in the brain that produce myelin.

It has already received orphan drug designation and advanced therapy medicinal product classification from the European Medicines Agency.

The gene therapy candidate also obtained orphan drug, rare paediatric disease and fast track statuses from the US Food and Drug Administration.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

Free Whitepaper

Optimise your cell therapy process: a guide to cell thawing

Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies.

By Cytiva Thematic

By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy