1. Astellas Pharma. Astellas reports update to September 1 announcement on the ASPIRO clinical trial of at132 in patients with X-linked myotubular myopathy. 2021. https://www.astellas.com/en/ news/17161 (last accessed September 15, 2021).
2. BioMarin Pharmaceutical. U.S. FDA placed a clinical hold on BMN 307 phearless phase 1/2 gene therapy study in adults with PKU based on interim pre-clinical study findings. 2021. https:// investors.biomarin.com/2021-09-06-U-S-FDA-Placeda-Clinical-Hold-on-BMN-307-Phearless-Phase-1-2-Gene-Therapy-Study-in-Adults-with-PKU-Based-on-Interim-Pre-clinical-Study-Findings (last accessed September 15, 2021).
3. Shape Therapeutics. Shape Therapeutics enters into a strategic research collaboration with Roche to advance breakthrough AAV-based RNA editing technology for neuroscience and rare disease indications. 2021. https://www.globenewswire.com/ news-release/2021/08/24/2285515/0/en/Shape-Therapeutics-enters-into-a-strategic-research-collabo ration-with-Roche-to-advance-breakthrough-AAVbased-RNA-editing-technology-for-neuroscienceand-rare-disease-indications.html (last accessed September 9, 2021).
4. NeuExcell Therapeutics and Spark Therapeutics, a Member of the Roche Group. NeuExcell therapeutics and spark therapeutics announce research collaboration agreement to develop a novel gene therapy for Huntington's disease. 2021. https://www.prnews wire.com/news-releases/neuexcell-therapeuticsand-spark-therapeutics-announce-research-collabo ration-agreement-to-develop-a-novel-gene-therapyfor-huntingtons-disease-301369445.html (last accessed September 11, 2021).