Ractigen Therapeutics is a clinical-stage pharmaceutical company focused on developing first-in-class therapies that utilize RNA activation (RNAa) technology to enhance the expression of therapeutic genes in diseased cells. The core of their innovation lies in small activating RNA (saRNA), which selectively activates genes to stimulate the production of targeted endogenous proteins. This technology is particularly significant for addressing previously undruggable diseases, including various monogenic disorders and conditions affecting the central nervous system, neuromuscular systems, and oncology. Ractigen's pipeline includes a range of therapeutic candidates aimed at conditions such as spinal muscular atrophy, amyotrophic lateral sclerosis, and non-muscle invasive bladder cancer, showcasing the versatility and potential of RNAa in modern medicine.

To facilitate the effective delivery of their RNAa therapeutics, Ractigen has developed proprietary platforms, including Smart Chemistry-Aided Delivery (SCAD™), Lipid-Conjugated Oligonucleotide (LiCO™), and GLORY™. SCAD™ is designed for efficient delivery of duplex RNA directly to the central nervous system, ensuring broad distribution and long-lasting effects. LiCO™ enables targeted delivery to various tissues through multiple administration routes, while GLORY™ focuses on optimizing liver delivery of duplex RNA. These advanced delivery systems are crucial for maximizing the therapeutic impact of Ractigen's saRNA candidates, allowing for precise activation of gene expression in targeted tissues and enhancing the overall efficacy of their treatments.

China

Ractigen Therapeutics is an early-stage biotech company committed to bringing to market first-in-class therapies designed to selectively restore the expression of therapeutic genes silenced in diseased cells. Its core technology is based on a paradigm-shifting discovery known as RNA activation (RNAa). Founded in 2016 by the pioneers in the field, Ractigen is developing a rich pipeline of candidate medicines for patients with unmet need.

Cancer

Central Nervous System (CNS)

Genetic Diseases

Hematologic Diseases

Musculoskeletal Diseases

Nervous System Diseases

Urogenital Diseases

Ractigen Therapeutics

Spinal muscular atrophy

RAG-06

Biologics

Oligonucleotides

Amyotrophic lateral sclerosis

RAG-17

Duchenne muscular dystrophy

RAG-18

Bladder cancer

RAG-01

Thrombocytopenia

RAG-03

Gene Therapy

Lentiviral Vector (LVV)

RNA Therapies

Small Molecule Pharmaceuticals

Therapeutic

Ractigen Therapeutics

Company Details