As the project partner, BLA congratulates BDGene on receiving the EU Orphan Drug Designations for the BD112 project in vivo gene editing therapy despite having only animal model data
Gaithersburg, MD (August 28, 2023) – BLA Regulatory, LLC, a full-service biopharma regulatory consultancy, congratulates Shanghai BDgene Therapeutics Co., Ltd. on receiving the Orphan Drug Designations from the European Commission (EC) for BD112 [1] in vivo gene editing therapy, despite having only animal model data. It is intended to be used to treat Huntington’s disease.
About BD112
BD112 is an innovative in vivo gene editing therapy based on original VLP delivery technology. VLPs enable efficient, transient delivery of CRISPR gene editing. The VLP of this gene guide has global leading advantages in terms of technical level, CMC, and clinical progress.
About BLA Regulatory, LLC
BLA Regulatory, LLC, is a full-service drug regulatory consultancy focusing on the US with global reach to other major markets. BLA provides comprehensive regulatory strategy and operation services from pre-IND strategic planning, IND preparation & submission, all the way to submission and approval of BLA/NDA. Our goal is to help move your product through the development cycle efficiently. With credits of 300+ INDs and 50+ NDAs/BLAs to our senior consultants, BLA brings clients a broad range of experiences from Big Pharma, CROs, and the FDA. Our services expand across regulatory, CMC, nonclinical, clinical, biostatistics, scientific writing, project management, eCTD publishing, and submission. We have extensive experience across therapeutic areas such as oncology, respiratory, addiction, autoimmune, inflammatory, metabolic, and cardiovascular diseases.
For further information contact:
Reference
[1] BDGene “Milestone of our company | Our in vivo gene editing gene BD112 has obtained Orphan Drug designation from the European Union” 27 August 2023.
https://mp.weixin.qq.com/s/Gu4kioccJ7SkxVrc4p1uKg