Cureline Group - Global Translational CRO’s Post

Stoke Therapeutics is nearing FDA sign-off on a groundbreaking Phase 3 clinical trial for its experimental drug for Dravet syndrome. https://trib.al/CG93Uvp

𝐓𝐡𝐞 𝐂𝐑𝐈𝐒𝐏𝐑-𝐂𝐚𝐬13 𝐑𝐍𝐀-𝐞𝐝𝐢𝐭𝐢𝐧𝐠 𝐭𝐡𝐞𝐫𝐚𝐩𝐲 𝐢𝐬 𝐬𝐞𝐭 𝐭𝐨 𝐞𝐧𝐭𝐞𝐫 𝐭𝐡𝐞 𝐜𝐥𝐢𝐧𝐢𝐜 Earlier this month, HuidaGene Therapeutics announced that the FDA had cleared its application to begin a clinical trial of HG202 in patients with neovascular age-related macular degeneration, which is a leading cause of blindness in people over the age of 60. HG202 is the first CRISPR-Cas13 RNA-editing therapy to enter clinical trials. Read more in our clinical update: https://lnkd.in/dAyvEm6Q #crisprmedicinenews #crisprmedicine #crispr #geneediting #rnaediting #blindness #crisprtrial

It was a pleasure to give at the #ESWISummit https://lnkd.in/eQnmgfnT an update on the latest in #RSV #antivirals #therapeutics development

Small molecule #therapeutics have had a powerful impact on patients for generations. As #synbio-derived therapeutics offer a new generation of #medicine, what does this mean for small molecule modalities? Join the discussion at #SynBioBeta2024: https://lnkd.in/eShayXpi

FDA's CDER recently issued a draft guidance: Nonclinical Safety Assessment of Oligonucleotide-Based Therapeutics. In this new Advancing RNA article, Peter Calcott of calcott consulting provides a summary and analysis. The public comment period ends January 14, 2025. https://lnkd.in/dD7VrGuU #oligonucleotides #drugdevelopment

"Our goal in working with industry is to quickly bring a pipeline of cell-based therapeutics to patients with unmet needs." (via Mayo Clinic) https://ow.ly/Vprr50Uk5RB

Percheron Therapeutics ends clinical trial in Australia and Europe after data indicates no benefits after 6 months of treatment with avicursen (ATL1102) #CureDuchenne #duchennemusculardystrophy #duchenne #musculardystrophy #percheronTherapeutics https://lnkd.in/gP9TnMcr

Excited to share that the first patient has been dosed in the clinical trial of SN301A in #HepatocellularCarcinoma, in collaboration with Celest Therapeutics, to evaluate the safety and preliminary anti-tumor activity of SN301A, which utilizes our multi-armed off-the-shelf #CARNK investigational #celltherapy SENTI-301A #GeneCircuit. Learn more here: https://bit.ly/49EBE7j $SNTI #syntheticbiology #HCC  

The best way to predict the future is to create it.

"Our goal in working with industry is to quickly bring a pipeline of cell-based therapeutics to patients with unmet needs." (via Mayo Clinic) https://ow.ly/F4nj50Uk5RE