- In October 2024, GEMMABio, one of the two new companies emerging from the now-defunct Gene Therapy Program at the University of Pennsylvania, announced its first major initiative aimed at improving global access to gene therapies for rare diseases including krabbe. In addition, the Philadelphia-based biotechnology firm, led by Jim Wilson, M.D., Ph.D., is set to receive up to USD 100 million from the Oswaldo Cruz Foundation (Fiocruz) to support clinical research and manufacturing efforts
- In May 2024, TeleRare Health introduced a video-based virtual clinic tailored for individuals in the U.S. living with rare diseases including krabbe. In this way, the new resource aims to provide support for those with conditions such as aromatic l-amino acid decarboxylase (AADC) deficiency, enhancing access to specialized care and information
- In March 2024, Forge Biologics announced that its innovative AAV gene therapy program, FBX-101, received the Innovation Passport designation from the Medicines and Healthcare Products Regulatory Agency (MHRA), allowing it to join the Innovative Licensing and Access Pathway (ILAP). In this context, the designation is expected to accelerate regulatory processes and market access interactions within the U.K. for rare diseases treatment including krabbe
