Lauffer, Marlen C. http://orcid.org/0000-0003-1607-0428
van Roon-Mom, Willeke
Aartsma-Rus, Annemieke
,
Funding for this research was provided by:
ZonMw (10250022110002)
Deutsche Forschungsgemeinschaft (521414448)
Article History
Received: 10 May 2023
Accepted: 27 November 2023
First Online: 5 January 2024
Competing interests
: A.A.R. is a steering committee member of the N = 1 collaborative. A.A.R., Wv.R.M., and M.C.L. are members of the N=1 Collaborative working groups on preclinical development and patient identification. A.A.R. discloses being employed by LUMC, which has patents on exon skipping technology, some of which has been licensed to BioMarin and subsequently sublicensed to Sarepta. As co-inventor of some of these patents, A.A.R. is entitled to a share of royalties. A.A.R. further discloses being an ad hoc consultant for PTC Therapeutics, Sarepta Therapeutics, Regenxbio, Alpha Anomeric, Lilly BioMarin Pharmaceuticals Inc., Eisai, Entrada, Takeda, Splicesense, Galapagos and Astra Zeneca. Past ad hoc consulting has occurred for CRISPR Therapeutics, Summit PLC, Audentes Santhera, Bridge Bio, Global Guidepoint, and GLG consultancy, Grunenthal, Wave, and BioClinica. A.A.R. also reports having been a member of the Duchenne Network Steering Committee (BioMarin) and being a member of the scientific advisory boards of Eisai, hybridize therapeutics, silence therapeutics, and Sarepta therapeutics. Past SAB memberships: ProQR, Philae Pharmaceuticals. Remuneration for these activities is paid to LUMC. LUMC also received speaker honoraria from PTC Therapeutics, Alnylam Netherlands, Pfizer, and BioMarin Pharmaceuticals and funding for contract research from Italfarmaco, Sapreme, Eisai, Galapagos, Synnaffix, and Alpha Anomeric. Project funding is received from Sarepta Therapeutics and Entrada. W.v.R.M. discloses being employed by LUMC, which has patents on exon-skipping approaches for neurological disorders. In the past, some of these patents have been licensed to ProQR therapeutics. As co-inventor of these patents, W.v.R.M. is entitled to a share of milestone payments and royalties. W.v.R.M. further discloses being an ad hoc consultant for Accure Therapeutics and Herbert Smith Freehills. Remuneration for these activities is paid to the LUMC. LUMC also received funding for contract research from UniQure and Amylon Therapeutics.