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news.The US Food and Drug Administration (FDA) has granted clearance for an investigational new drug (IND) application of Epicrispr Biotechnologies’ EPI-321, intended for treating genetic neuromuscular condition, facioscapulohumeral muscular dystrophy (FSHD).
Plans are underway to commence the global Phase I/II trial this year. Credit: Lucas Vasques on Unsplash.
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This clearance comes after the company’s receipt of approval from New Zealand’s Medsafe last month for clinical trial application to commence a first-in-human study of the therapy.
During the month, the company has also raised $68m in the first close of a Series B funding round for supporting the therapy’s clinical development.
Plans are underway to commence a global Phase I/II trial of EPI-321 this year.
An investigational single-time gene-modulating therapy, EPI-321 is tailored to suppress aberrant expression of the double homeobox 4 (DUX4) gene, which is not activated correctly in this genetic neuromuscular condition, resulting in progressive muscle degeneration.
It is said to be systemically delivered through a “clinically validated” adeno-associated virus (AAV) vector.
In preclinical models, the therapy has shown a DUX4 expression suppression and muscle tissue protection.
The FDA granted rare paediatric disease and orphan drug and fast-track designations to the therapy.
Epicrispr Biotechnologies CEO Amber Salzman said: “For too long, individuals with FSHD have faced a progressive disease with no treatment options.
“EPI-321 is the first therapeutic candidate designed to silence DUX4 through epigenetic reprogramming, offering a potentially transformative approach to halting disease progression in FSHD. We look forward to initiating our study in the US and bringing EPI-321 to patients who urgently need therapies.”
Epicrispr develops gene-modulating therapies, focusing on treatments for neuromuscular conditions.
Its Gene Expression Modulation System (GEMS) allows for gene expression control, offering treatments for conditions that were untreatable before.
The company’s lead programme, EPI-321, is currently being studied in trials for FSHD, and Epicrispr is also further progressing the development of gene-modulating therapies.
It formed a research partnership with Kite Pharma for developing chimeric antigen receptor (CAR) T-cell therapies.
