Latest Hotspot

Encouraging Results for Ractigen's RAG-17 in ALS-SOD1 Therapy from Independent Study

13 September 2024
3 min read

Ractigen Therapeutics, a pharmaceutical firm focusing on clinical-stage innovations, has reported favorable clinical findings from an Investigator-Initiated Trial (IIT) involving RAG-17, a small interfering RNA (siRNA) aimed at the Superoxide Dismutase 1 (SOD1) gene. The trial demonstrated positive outcomes in addressing amyotrophic lateral sclerosis (ALS) linked to SOD1 mutations (ALS-SOD1).

👇Discover comprehensive information about this drug, from its R&D status, core patents, clinical trials to approval status in global countries, by simply clicking on the image below. Dive deep into our drug database now.

Dr. Yilong Wang spearheaded a clinical study at Beijing Tiantan Hospital, a premier institution in China specializing in neurological conditions. The study involved six ALS-SOD1 patients and mainly focused on evaluating the safety profile of RAG-17. Administering RAG-17 intrathecally showed good tolerance at all dosage levels, with adverse events being mild in nature. Extensive safety assessments, including laboratory tests, vital sign monitoring, and electrocardiograms, further confirmed its favorable safety characteristics.

Additionally, preliminary signs indicated potential clinical benefits. Changes in clinical outcomes and key biomarkers suggested that RAG-17 could be effective for this patient group. These clinical findings are consistent with comprehensive preclinical data from Ractigen, which showed significant therapeutic benefits of RAG-17 in SOD1-G93A ALS mouse and rat models, such as delayed disease progression and extended survival.

"The initial clinical data is particularly encouraging and represents a step forward in our mission to bring new hope to ALS patients," noted Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. "The positive results from this trial highlight RAG-17's potential as a disease-modifying therapy for ALS-SOD1. We remain fully committed to advancing its clinical development and ultimately providing this critical therapy to patients."

The promising data will be showcased at three upcoming conferences: the 27th National Conference of Neurology in China in September, Neuroscience 2024 in Chicago, USA, in October, and the 35th International Symposium on ALS/MND in Montreal, Canada, in Decemberone of the major annual conferences for ALS and motor neuron disease research.

In March 2023, RAG-17 was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA), and its Investigational New Drug (IND) application for clinical trials in the U.S. was approved. Furthermore, in May 2024, the IND application received approval from the Center for Drug Evaluation (CDE) under China National Medical Products Administration (NMPA) for clinical trials in China.

👇Explore the latest research progress on drug-related developments, indications, therapeutic organizations, clinical trials, results, and patents by clicking on the targeted picture link below. Unfold a world of comprehensive information on this target in just a click!

图形用户界面, 文本, 应用程序, 电子邮件

描述已自动生成

According to the data provided by the Synapse Database, As of September 12, 2024, there are 58 investigational drug for the SOD1 targets, including 85 indications, 73 R&D institutions involved, with related clinical trials reaching 77, and as many as 14568 patents.

RAG-17 is a siRNA drug that targets the SOD1 gene and is being developed by Ractigen Therapeutics. The drug is indicated for the treatment of Amyotrophic Lateral Sclerosis (ALS) and has reached Phase 1 in both global and Chinese regulatory processes. Additionally, [RAG-17] is classified as an Orphan Drug, which provides regulatory and financial incentives for the development of drugs that target rare diseases.

图形用户界面, 文本, 应用程序

描述已自动生成

Weekly Insulin Efsitora Alfa Matches Daily Insulin in A1C Reduction for Type 1 Diabetes
Latest Hotspot
3 min read
Weekly Insulin Efsitora Alfa Matches Daily Insulin in A1C Reduction for Type 1 Diabetes
13 September 2024
Insulin efsitora alfa, administered weekly, achieves comparable A1C reduction to daily insulin in adults with type 1 diabetes.
Read →
Latest Results on HER2-Mutant NSCLC Drug BAY 2927088 Revealed at WCLC
Latest Hotspot
3 min read
Latest Results on HER2-Mutant NSCLC Drug BAY 2927088 Revealed at WCLC
13 September 2024
Recent findings on the investigational drug BAY 2927088 for HER2-mutant non-small cell lung cancer (NSCLC) showcased at WCLC.
Read →
Pharma Frontiers: Daily Digest of Global Pharmaceutical News – Sep 13
Pharma Frontiers: Daily Digest of Global Pharmaceutical News – Sep 13
13 September 2024
Sep 13th latest updates in the global new drug development field, including progress in new drug research and development, transaction information, and partnership developments.
Read →
Datopotamab Deruxtecan Shows 14.6-Month Median Survival in Phase 3 TROPION-Lung01 Trial for Advanced Nonsquamous NSCLC
Latest Hotspot
3 min read
Datopotamab Deruxtecan Shows 14.6-Month Median Survival in Phase 3 TROPION-Lung01 Trial for Advanced Nonsquamous NSCLC
13 September 2024
In the TROPION-Lung01 Phase 3 trial, Datopotamab Deruxtecan demonstrated a median overall survival of 14.6 months for patients with advanced nonsquamous non-small cell lung cancer.
Read →
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.