Lysosomal Disorders

The pediatrician from Royal Manchester Children’s Hospital discussed how further research in Gaucher disease can benefit the field of rare lysosomal diseases as a whole.

Review top news and interview highlights from the week ending October 28, 2022.

CGTLive highlights the PROVIDE trial of PR001 for Gaucher Awareness Month.

AVR-RD-02 also received ILAP designation in the UK earlier this month.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The pediatrician from Royal Manchester Children’s Hospital discussed progress with the PROVIDE trial of PR001.

The trial is being conducted at the University of California – San Diego under the leadership of Stephanie Cherqui, PhD.

Rossano discussed unmet needs for patients with Danon disease and the potential of investigational gene therapy RP-A501.

As we learn more about genomics and identify more genes tied to rare disorders, the role of genetic counselors will become even more critical.

Review top news and interview highlights from the week ending October 21, 2022.

Pam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access to advanced care.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.

With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.

Participant 3 showed an 89% decrease in NAA in the CSF at 3 months post-treatment.

Review top news and interview highlights from the week ending October 14, 2022.

The chief scientific officer of cell therapy at Poseida Therapeutics discussed the company’s CAR-T and TCR technologies.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

All 5 patients on ERT at study start have since been able to withdraw after gene therapy treatment.

The professor of genetics at Federal University of Rio Grande do Sul discussed how RGX-121 has the potential to change the treatment landscape of MPSII.

Review top news and interview highlights from the week ending October 7, 2022.

The FDA recently cleared Freeline Therapeutics to begin treating patients in the United States.

Karen Walker, chief technology officer, Kyverna Therapeutics, discussed the company’s CAR T-cell and regulatory T-cell technologies.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Data from the first 3 of the 8 patients dosed indicates that the patients improved on the GMFM and the MSEL.