Aardvark Therapeutics’ Post

CCDS Day 1 - session 3 : Advances in therapeutic development for CCDS Ceres Brain Therapeutics : CBT1, an innovative patented drug to address metabolic disruption within neurons by releasing creatine. Positive short term tox. What’s next : IND/IMPD filings. Need funding to do long term tox study and get into clinical trials. A lot of hope for CTD patients CERES BRAIN Therapeutics Thomas Joudinaud #CTD #SLC6A8

Beacon Therapeutics presented 12-month data from its Phase II SKYLINE trial of its lead asset, AGTC-501, in XLRP at the Annual Macula Society Meeting, demonstrating encouraging efficacy and safety.   The presentation highlighted encouraging efficacy shown by improvements in retinal sensitivity, the primary endpoint for the trial, with a 63% response rate in the higher dose cohort. Beacon expects to announce longer term 24-month data from the trial in H2 CY2024.   Read the full announcement here: https://lnkd.in/d4Qfpq6d   #ophthalmology #blindness

Sarepta Therapeutics has announced positive data from Part B of its MOMENTUM study of exon skipping therapy SRP-5051 (vesleteplirsen). Data from the study found that at the higher, target dose, approximately 30 mg/kg dosed every four weeks, SRP-5051 resulted in mean dystrophin expression of 5.17%, and mean exon skipping of 11.11% at 28 weeks. You can read more about the study’s findings here: https://lnkd.in/ecukQjmh

How can generative AI accelerate peptide drug discovery? 💡 You don’t want to miss our CEO’s, Hans Melo, talk at #BioEuropeSpring! 👋 #hitdiscovery #drugdiscovery #drugdevelopment #peptides #peptidetherapy #cyclicpeptides #macrocycles #artificialintelligence #ai #generativeai #therapeutics #aminoacids #chemicalspace #mentenai #partnering 

The Future of Exosome Based Therapeutics: Key Clinical Updates ZEO ScientifiX™ invites you to a LIVE Webinar on The Future of Exosome Based Therapeutics with Dr. George Shapiro, MD https://lnkd.in/g5pnyr42

Great to see some positive news in the allogeneic CAR T space. Especially for a disease like Multiple Myeloma where commercial companies have struggled to meet demand. Also interest to see prominent use of a non-viral gene delivery platform, which I think we'll see more of very soon. #CART #celltherapy #genetherapy.

Potency development and regulatory expectations are the key for the success of CGT CMC development.

Halia Therapeutics, Inc.'s and Biolexis Therapeutics, Inc.'s partnership have identified a novel brain-penetrant small molecule candidate targeting NLRP3-driven neuroinflammation by leveraging Biolexis’ MolecuLern™ AI-enabled approach. This milestone represents a significant advancement in treatments for neuroinflammatory disorders, including Parkinson’s. #HaliaTherapeutics #BiolexisTherapeutics #BioUtah #UtahLifeSciences https://ow.ly/Jqow50SSyf2

Molecules of the Month – August 2024 | https://lnkd.in/gBWTKV32 August’s Molecules of the Month include Kannalife Sciences, Inc.'s GPR55 antagonist for chemotherapy-induced neuropathic pain and Boehringer Ingelheim’s zwitterionic inhibitor of KHK for metabolic disorders. We also feature bomedemstat, Merck's irreversible LSD1 inhibitor, a small peptide inhibitor of the Fas receptor from ONL Therapeutics, and Boehringer Ingelheim's PDE4B inhibitor with the potential to treat IPF. Read the full article to find out what compounds made our August 2024 Molecules of the Month list and check out recent articles for each. Full Article: https://lnkd.in/gBWTKV32

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