Gene therapy company Avrobio has stopped developing a treatment based on clinical trial results for the Fabry therapy. Avrobio attempted to treat Fabry disease through a stem cell gene therapy using a lentivirus.
ImmixBio has received Rare Pediatric Disease (RPD) designation from the FDA for the treatment of a rare pediatric cancer. The treatment IMX-110 has already received orphan drug designation (ODD) from the FDA as a treatment for soft tissue sarcoma.
Pacific Bioscience announced that it is collaborating with Care4Rare Canada Consortium to diagnose rare diseases through WGS testing. They plan to diagnose undiagnosed patients by using PacBio’s long-read sequencing technology on samples that have been sequenced with short-read technology .
Genomics England has announced plans to conduct Whole Genome Sequencing (WGS) for 5 million people over the next five years. The UK started the 100,000 Genome Project in 2013 and achieved the target of WGS for 100,000 people in 2018, and immediately announced its business expansion plan.
TikTok star Marky Jaquez, who suffered from a rare disease called epidermolysis bullosa, has passed away. Marky and her parents have been trying to raise awareness of living with a rare disease and give hope to patients. The parents said they will continue to do this in the future.
Nikki Deeley has been suffering from persistent pain for 10 years after being diagnosed with fibrous dysplasia. However, she continues to challenge herself to raise awareness of rare diseases, such as running for charity and fundraising.
Facebook has announced that it will ban patient-targeted advertising through messages based on a user's health status. This is likely to influence clinical patient recruitment that has been done through Facebook advertisements.
In 2021, the Australian government funded a total of $63.4M for 27 projects through RCRDUN (Rare Cancers, Rare Diseases and Unmet Need). Detailed project information and funding amount can be found at the link below.
Rare disease pharmaceutical industry trendsIonis Pharmaceutical and AstraZeneca announced that they had succeeded in Phase 3 clinical trial of co-developing transthyretin amyloidosis treatment(eplontersen). However, the…