Ractigen Therapeutics

Ractigen is developing a class of groundbreaking saRNA therapeutics which treat disease by turning on genes. Our immediate focus are monogenetic rare diseases.

[Ractigen Therapeutics @Ractigen_Tx]

Thrilled to unveil late-breaking obesity therapy data at #ADA2026! The saRNA approach redefines weight loss: achieving 45% fat loss while preserving muscle—outperforming standard treatments.
Abstract: https://t.co/iGSHYF7oTX
Details: https://t.co/CvIfJ4lFmL
#saRNA #LiCO #Obesity

[Ractigen Therapeutics @Ractigen_Tx]

RAG-17 Phase I data showcased at #AAN2026! A single intrathecal dose achieved up to 58% reduction in CSF SOD1, 81% reduction in plasma NfL, and encouraging functional stability in SOD1-ALS patients.
Abstract: https://t.co/6YPRE0pmsG
Discover more: https://t.co/5d0cfcGxpV
#SCAD

[Ractigen Therapeutics @Ractigen_Tx]

Ractigen Therapeutics' RAG-01 just received China NMPA CDE IND approval for Phase II clinical trials in Non-Muscle Invasive Bladder Cancer (NMIBC). A significant step forward for saRNAtherapeutics globally & in China! #saRNA #NMIBC
Learn More: https://t.co/Fsze1boRZ6

[Ractigen Therapeutics @Ractigen_Tx]

Ractigen Therapeutics has successfully dosed the first patient in the Phase II trial of RAG-17, targeting SOD1-mutated ALS. This milestone brings us closer to innovative treatments for this challenging disease. #ALS #RAG17 #siRNA
Learn More: https://t.co/mPH4BDzvbt

[Ractigen Therapeutics @Ractigen_Tx]

Ractigen Therapeutics has successfully dosed the first patient in the Phase II trial of RAG-17, targeting SOD1-mutated ALS. This milestone brings us closer to innovative treatments for this challenging disease. #ALS #RAG17 #siRNA
Learn More: https://t.co/mPH4BDzvbt

[Ractigen Therapeutics @Ractigen_Tx]

Milestone achieved! Ractigen Therapeutics has dosed the first patient in our RAG-18 trial for Duchenne Muscular Dystrophy. As an agnostic saRNA therapy, RAG-18 has the potential to transform the treatment landscape for all DMD patients. #saRNA #DMD #RNAa https://t.co/mlq8bGqx4M

[Ractigen Therapeutics @Ractigen_Tx]

Exciting news! Ractigen’s foundational study on the SCAD platform—a modular system for efficient duplex RNA delivery to the CNS & beyond—has been published in Molecular Therapy - Nucleic Acids! 🧠💡
It’s open access—read here: https://t.co/VXnM19CyaF
#CNSdelivery #siRNA #saRNA

[ALS News Today @alsnewstoday]

Review findings showing the therapy’s potential to “significantly” impact the lives of those with SOD1-ALS: https://t.co/NGrrbfNb5G

#ALS #AmyotrophicLateralSclerosis #ALSDisease #ALSTreatment #ALSResearch

[Ractigen Therapeutics @Ractigen_Tx]

Ractigen’s RAG-17 data makes a distinguished debut at #AAN2025! Awarded “Abstract of Distinction” & Phase I trial progressing well, showing potential to slow functional decline in #ALS patients. Find Abstract here: https://t.co/irO343KTBB Discover more: https://t.co/XeTD0utPvt

[Ractigen Therapeutics @Ractigen_Tx]

New approach to #BladderCancer! Ractigen's RAG-01, a first-in-class #saRNA therapy, demonstrates remarkable efficacy in early Phase I data for BCG-unresponsive #NMiBC #EAU25 @urotoday @UrologyTimes https://t.co/8muEH4jyRP

[Ractigen Therapeutics @Ractigen_Tx]

"Our CEO, Dr. Long-Cheng Li, has published a review on RNAa in Molecular Therapy - Nucleic Acids! This article explores the fascinating world of this gene regulation mechanism and its therapeutic applications. Check it out here: https://t.co/lib1ExAQl1"

[Long-Cheng Li @RNAa_biologist]

Excited to share our latest review on RNAa in Molecular Therapy - Nucleic Acids! Learn about this fascinating gene regulation mechanism and its therapeutic applications: https://t.co/6Yy84nZpsV #RNAa #saRNA #GeneRegulation #MolecularTherapy

[Ractigen Therapeutics @Ractigen_Tx]

Our CEO is going to present the progress of RAG-18 program which is an IND-enabling stage saRNA drug candidate for the treatment of DMD irrespective of DMD gene mutation. #dmd #sarna

[Long-Cheng Li @RNAa_biologist]

Excited to announce that I'll be speaking on our RAG-18 DMD program at the @RNALeaders #Leaders Europe! Register now and join my session in Basel on 4-6 March! 👉 https://t.co/E5zgglYXAc #rnaleaders #RNA #mRNA #RNAi

[Ractigen Therapeutics @Ractigen_Tx]

Our Founder & CEO, Dr. Long-Cheng Li, receives the 2024 Life Science Ice Breaking Award for advancing innovative RNA-based therapies for ALS. A testament to his contributions to ALS treatment and Ractigen's vision for patient-centric innovation. https://t.co/4O3Je9Jvsy

[Ractigen Therapeutics @Ractigen_Tx]

We have successfully completed the first patient dosing in the Phase I clinical trial for our innovative ALS treatment, RAG-17. This milestone highlights our commitment to advancing RNA therapies that have the potential to transform lives. Read more: https://t.co/F2P1HzO1zF

[Ractigen Therapeutics @Ractigen_Tx]

Ractigen Therapeutics has received FDA ODD for RAG-21, our novel RNA-based therapy targeting FUS-ALS. This marks our second ODD, reflecting our commitment to developing innovative treatments for ALS. Together, we can conquer this disease! https://t.co/hIgcafKP36

[Ractigen Therapeutics @Ractigen_Tx]

Discover the advanced capabilities of our SCAD™ (Smart Chemistry Aided Delivery) system, designed to leverage the self-delivering properties of ASO, ensuring safe, robust and sustained gene knock-down in CNS. https://t.co/glQEp6RXTm

[Ractigen Therapeutics @Ractigen_Tx]

Exciting News! We're thrilled to announce positive clinical data for RAG-17 in treating ALS-SOD1 from our Investigator-Initiated Trial! Results show strong safety and early signs of efficacy. Together, we’re bringing new hope to ALS patients. Read more: https://t.co/OioGOMlW8d

[Muscular Dystrophy News Today @mdnewstoday_]

Here is this week's most-read article featured on our website. Are you caught up? https://t.co/O9LRXUUEew

#MuscularDystrophyNews #MuscularDystrophy

[CGTLive® @CGT_Live]

RAG-18 is intended to function through targeted activation of the UTRN gene in muscle cells, leading to increased production of utrophin, the protein encoded by UTRN.

#MuscularDystrophy #RNAtherapy #saRNA @Ractigen_Tx

Read more:

https://t.co/RdxiIlj1EB

[Ractigen Therapeutics @Ractigen_Tx]

Ractigen’s RAG-18 receives FDA Orphan Drug Designation, a game-changer in treating Duchenne Muscular Dystrophy and Becker Muscular Dystrophy. Read more: https://t.co/jz6tEmYijY