Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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The Genomics and Epigenetic Guided Safe Harbor mapper will aid in the future design of gene-editing therapies.
BrainStorm received indication from the FDA that it is able to request a Type A meeting to discuss the content of the refusal to file letter.
The FDA action follows a slew of recent cell and gene therapy updates in the field of amyotrophic lateral sclerosis.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The trial was launched through a partnership between Cure Rare Disease and UMass Chan Medical School.
The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s work with regulatory members to advance gene therapies towards review and approval.
Review top news and interview highlights from the week ending November 4, 2022.
The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s goals and strategies in educating regulatory decision-makers.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The DSMB has recommended the trial to continue with additional risk mitigation procedures.
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.
MRI revealed that participants who achieved sustained or confirmed disability improvement showed significantly less ventricular enlargement at 12 months.
Review top news and interview highlights from the week ending October 28, 2022.
CGTLive highlights the PROVIDE trial of PR001 for Gaucher Awareness Month.
The trial expects to enroll its first patient in the first half of 2023.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
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.
The proportion of patients with concentrations of NfL and MBP above the upper limit of normal decreased from 67% and 63%, respectively, to 12% at 5 years.
With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.
The director of the Regenerative Medicine Institute at Cedars Sinai discussed research being conducted with CNS10-NPC-GDNF in multiple indications.
Participant 3 showed an 89% decrease in NAA in the CSF at 3 months post-treatment.
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