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4DMT's Fabry gene therapy on hold, but 'remains active' .
March 28, 2023

4DMT’s Fabry gene therapy on hold, but ‘remains active’ .

The National Institute for Health and Care Excellence (NICE) in the UK has approved the use of guided, self-help digital cognitive behavioral therapy (CBT) for children experiencing anxiety. This decision comes after the agency carried out a thorough cost-effectiveness analysis of the treatment option. Digital CBT aims to give children access to mental health support in a format that is more accessible, convenient, and affordable than traditional therapy. This is especially important considering the COVID-19 pandemic has made access to mental health services more challenging for many. By allowing for self-directed therapy, children can work through their anxiety at their own pace, and parents or carers can offer support when needed. This decision by NICE is a positive step towards prioritizing mental health and making effective treatments available to those who need them.

4D Molecular Therapeutics (4DMT) has faced a setback as its Fabry disease gene therapy program, known as 4D-310, has been placed on clinical hold by the US Food and Drug Administration (FDA). However, the company remains optimistic about the program’s future. Despite the clinical hold, 4DMT has stated that the program remains active and that they will work closely with the FDA to address any concerns and move the program forward. Although there is uncertainty about the program’s future, the company is continuing to advance its gene therapy using an adeno-associated viral vector. While 4DMT navigates this obstacle, Sangamo is expected to gain a significant market share as its therapy is likely to receive approval before 4DMT’s. The patient perspective on gene therapy and gene editing, including concerns and needs, will be an important factor in the future of this field. Despite this setback, 4DMT is committed to continuing its work on this promising therapy for Fabry disease, and the industry will be closely watching its progress.

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