Ractigen’s ALS Drug Candidate Gains Orphan Drug Designation
The FDA has granted Orphan Drug status to Ractigen Therapeutics’ RAG-17, an investigational treatment of patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
RAG-17 is a candidate small interfering RNA (siRNA) therapy designed to reduce expression of the SOD1 enzyme in ALS patients who carry certain gene mutations.
Several preclinical studies have shown RAG-17’s potential as a treatment for this patient population, showing significant potency, the company said.
The FDA’s Orphan Drug designation is for drugs and biologics intended to treat a rare disease or condition that affects fewer than 200,000 patients in the U.S. The designation gives companies incentives such as seven-year marketing exclusivity from the date of FDA approval and a waiver of user fees.
RNAa Technology
RNAa (RNA activation) is a platform technology to selectively turn genes on by using short duplex RNA known as small activating RNA (saRNA).
RNAa is one of the few technologies that can stimulate production of endogenous targeted genes.
Ractigen Therapeutics
Ractigen is leading the translation of RNAa (RNA activation) into a new class of oligonucleotide therapeutics capable of specific activation of target gene expression. Ractigen pipeline focuses on previously undruggable diseases including monogenic disorders (e.g., haploinsuffiencies) in which RNAa can compensate or rescue loss in gene activity by boosting transcription. Therapeutic areas enabled by our SCAD™ and GOLD™ delivery platforms currently include the central nervous system (CNS), ocular, liver, and oncology.
March 7, 2023
https://www.fdanews.com/
RAG-17 is a candidate small interfering RNA (siRNA) therapy designed to reduce expression of the SOD1 enzyme in ALS patients who carry certain gene mutations.
Several preclinical studies have shown RAG-17’s potential as a treatment for this patient population, showing significant potency, the company said.
The FDA’s Orphan Drug designation is for drugs and biologics intended to treat a rare disease or condition that affects fewer than 200,000 patients in the U.S. The designation gives companies incentives such as seven-year marketing exclusivity from the date of FDA approval and a waiver of user fees.
RNAa Technology
RNAa (RNA activation) is a platform technology to selectively turn genes on by using short duplex RNA known as small activating RNA (saRNA).
RNAa is one of the few technologies that can stimulate production of endogenous targeted genes.
Ractigen Therapeutics
Ractigen is leading the translation of RNAa (RNA activation) into a new class of oligonucleotide therapeutics capable of specific activation of target gene expression. Ractigen pipeline focuses on previously undruggable diseases including monogenic disorders (e.g., haploinsuffiencies) in which RNAa can compensate or rescue loss in gene activity by boosting transcription. Therapeutic areas enabled by our SCAD™ and GOLD™ delivery platforms currently include the central nervous system (CNS), ocular, liver, and oncology.
March 7, 2023
https://www.fdanews.com/
