Both patients dosed experienced a stable increase in cerebrospinal fluid HexA activity.
Lysosomal Disorders
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Review top news and interview highlights from the week ending March 4, 2022.
David Taylor, PhD, an assistant professor in the department of molecular biosciences at UT Austin, discussed the Cas9 research conducted between 2 labs at UT Austin.
The senior vice president and head of clinical development at Mustang Bio discussed the importance of Rare Disease Day.
The decision is the latest action in a line of patent interferences over the revolutionary gene-editing technology.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The cofounder and chief scientific officer of Cartesian Therapeutics discussed the importance of Rare Disease Day.
The therapy was well-tolerated in 15 patients and no clinically relevant liver findings were observed.
The professor from National Taiwan University Hospital discussed the importance of Rare Disease Day for raising awareness.
Clinical and industry leaders share their perspectives on the importance of collaboration in developing treatments for rare diseases.
The president and chief operating officer of Rocket Pharmaceuticals discussed the importance of Rare Disease Day.
The director of the Powell Gene Therapy Center at the University of Florida discussed the importance of Rare Disease Day.
Review top news and interview highlights from the week ending February 25, 2022.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The trial design was informed by both patient and physician perspectives.
The pheNIX clinical hold follows an announcement that BioMarin’s PKU hold may last several quarters.
Review top news and interview highlights from the week ending February 18, 2022.
The company also announced that the phase 3 study of val-rox has completed enrollment.
Matthew Gantz, president and chief executive officer, Castle Creek Biosciences, discussed FCX-013 for the potential treatment of scleroderma.
Myrtelle’s therapy targets oligodendrocytes to deliver a functional copy of the ASPA gene.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Updated data were presented on LYS-SAF302 and LYS-GM101 at WORLDSymposium.
The director of research and development at AVROBIO discussed the analytical pipeline the company has developed for molecular follow-up of cell therapies.
The associate professor from UC San Diego discussed the curative potential of gene therapy in cystinosis.
Passage Bio presented data on both the Krabbe and GM1 gangliosidosis programs at WORLDSymposium.
