2019 NF2 Gene Therapy Consortium

On March 29, 2019, the non-profit organization NF2 BioSolutions hosted a first-of-its-kind consortium focused on gene therapy (GT) strategies for the rare genetic disorder Neurofibromatosis Type 2 (NF2). The one-day interactive meeting which was held at the SAP America office in Boston, MA, USA was attended by a diverse group of key researchers and clinicians in NF2, as well as gene therapy experts from both academia and industry. The forty-five attendees of the expert meeting included 11 NF2 researchers, 12 NF2 clinicians, 10 gene therapy researchers, and 6 biotech executives from all over the US and the UK.

Some of the institutions represented:

 

 

The meeting was carried out in a participatory workshop format wherein attendees were grouped into five teams, each comprised of representatives from every expert group. Four brainstorming exercises were prepared for the consortium which were completed by each team in parallel, after which a general discussion amongst all attendees was held for each exercise. The four group exercises focused on:

1. Existing gene therapy strategies and their applicability on NF2
2. Challenges in gene therapy and NF2 research
3. Deriving the needs from the challenges in gene therapy and NF2 research
4. Developing the process to bring gene therapy for NF2 to the patients

This report below summarizes the top outcomes derived from the consortium and highlights the key points that the focus groups established for each exercise. The immediate steps to be undertaken towards developing the ideas gathered from this expert meeting will also be discussed.

In the report appendix A is the attendees list and appendix B is the agenda.

*​DISCLAIMER*​ ​: This summary report is a compilation of notes and video transcriptions of oral presentations during the consortium. Some information may have been inadvertently missed or misinterpreted during the process.