Ractigen Secures FDA Orphan Drug Designation for RAG-21 in ALS
Wednesday, November 20, 2024
Ractigen Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RAG-21, a novel small interfering RNA (siRNA) therapy.
RAG-21 is an RNA interference therapy designed to lower FUS mRNA levels, preventing the production of toxic proteins linked to FUS-ALS, a severe form of amyotrophic lateral sclerosis. Delivered via the SCAD™ platform, it ensures precise and sustained gene knockdown within the central nervous system.
ALS is a neurodegenerative disease with no cure, often causing death within 2–5 years of diagnosis. FUS mutations lead to aggressive ALS, characterised by early onset, rapid progression, and toxic protein build-up in neurons. Existing treatments offer limited benefits, but RNA-based therapies like RAG-21 target the root cause, providing new hope.
This therapy is designed to target the FUS gene in amyotrophic lateral sclerosis (ALS), a rare and severe neurological condition.
RAG-21 addresses FUS-ALS, a particularly aggressive form of the disease. Using RNA interference (RNAi) technology, the therapy reduces levels of FUS protein, which is linked to motor neuron damage in this ALS subtype.
Preclinical research has shown that RAG-21 effectively mitigates the harmful effects of FUS mis-localisation and aggregation, offering a potential solution where no curative treatments currently exist.
The Orphan Drug Designation programme recognises therapies targeting rare conditions affecting fewer than 200,000 individuals in the United States. It provides benefits to developers, such as tax credits for clinical trials, seven years of market exclusivity, and a waiver of certain regulatory fees.
RAG-21 represents the company’s second orphan designation for ALS therapies, following an earlier approval for RAG-17 targeting SOD1-ALS.
This milestone highlights the continued efforts to develop RNA-based solutions for rare diseases with high unmet medical needs.
Source: ractigen.com
