GOOD NEWS丨YEEDOZENCOM assisted Ractigen in the IND approval of small nucleic acid drug RAG-17

On 15 May 2024, our cooperative client Ractigen Therapeutics (hereinafter referred to as " Ractigen ") received a notification from CDE, that the self-developed small nucleic acid drug, RAG-17 injection, has obtained IND approval. Warmly congratulations to Ractigen!

About ALS

ALS, the full name is Amyotrophic Lateral Sclerosis, is a chronic progressive neurodegenerative disease characterized by damage to upper and lower motor neurons, manifested by muscle weakness, muscular atrophy, medullary palsy and pyramidal signs. ALS is an incurable disease, with a survival period of 3-5 years after its onset. ALS is classified as sporadic or familial, and familial ALS can be caused by mutations in a variety of genes, SOD1 is one of the common genes. In China, SOD1 is the most commonly mutated gene causing ALS, and about 20% of familial ALS and 5% of sporadic ALS are caused by mutations in the SOD1 gene.

About RAG-17

RAG-17 is a small double-stranded interfering RNA (siRNA) targeting SOD1 for the treatment of ALS caused by the SOD1 gene, it is used to treat patients with ALS caused by mutations in SOD1 by decreasing the expression of SOD1 protein.

Preclinical efficacy studies have demonstrated that treatment with RAG-17 can extremely significantly delay the onset of the disease, prolong the survival of animals and improve their motor function. An IIT has enrolled and treated six participants, and preliminary results show positive efficacy and a favorable security. The availability of RAG-17 offers new hope for people struggling with ALS.

Cooperation

In the process of IND application of the product, Yeedo and Ractigen worked closely in the review and evaluation of non-clinical material, clinical protocol and related documents, filing and submission of application material. Through the joint check of both parties, RAG-17 obtained IND approval successfully.