It has been an exciting start to the summer for our team, as we exited stealth mode and announced $40M in seed and Series A financing on June 20. Last month our leadership team of CEO Pankaj Bhargava, CSO ?Victoria Smith, and CMO Deborah Wenkert attended the FOP Drug Development Forum and the International Conference on Children’s Bone Health (ICCBH). During those events we had the chance to share details of our upcoming clinical trial of andecaliximab for the treatment of fibrodysplasia ossificans progressiva (FOP). It was a pleasure to connect with the FOP community, hear their stories, and share our goals. In case you missed our news, you can see it here: ?https://bit.ly/3Rw9TG7 International FOP Association #raredisease #biotech #lifesciences #biopharma
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Take a look at the?Spotlight interview with Eduardo Jule, PhD?in the September AAPS LBDDS Community Newsletter. Dr. Jule, our esteemed trainer, will be leading the upcoming "Lipid-Based Formulations" course. With over 20 years of experience in lipid-based drug delivery and pharmaceutical development, this training offers an unmatched opportunity to gain valuable insights! ?? Check out all Lipid Based Formulations news in the newsletter below! ?? #interview #Newsletter #LipidBasedFormulations #SymmetricTraining
Pharmaceutical/ Early and Late stage Formulation Development/ Process Development / Parenteral and Semisolid dosage form/ ANDA/NDA
Our September AAPS LBDDS Community Newsletter is here, brimming with invaluable insights and updates! ?? ?? Dive into the latest USP news on lipid-based drugs ?? Explore a wealth of information on lipid-based posters and talks for the AAPS PharmSci 360 meeting ?? Enjoy our spotlight interview featuring Eduardo Jule, PhD and myself Dimple Modi Ph.D. Mark your calendars with upcoming events. Celebrate the honors and rewards of renowned scientists Jayachandra Babu Ramapuram, PhD, FAAPS and Ben Boyd This newsletter is your go-to source for staying up-to-date with the latest advancements. A special thank you to Jayachandra Babu Ramapuram, PhD, FAAPS and his dedicated team for their exceptional effort in publishing this newsletter! #PharmSci360 #LBDDS #PharmaceuticalScience #AAPS #Newsletter
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Today was the last day of the 17th EBF Open Symposium in Barcelona. Esther van Duijn presented a poster on behalf of Peregrion, TNO’s AMS spin-off. Within Peregrion we anticipate on the new Mass Balance guideline of the FDA, generating the human ADME data package (already) in early clinical development. Colleague Rob Vreeken of TNO was on the program and presented on the AMS application of 14C-labeled substrates for efficacy measurements in drug development. Learning more about Peregrion: https://www.peregrion.com/ Interested in innovative AMS applications of TNO: https://lnkd.in/g4skHAr3 #TNO #Peregrion #EBF2024 #Bioanalysis #AMS #LifeScience #DrugDevelopment #ClinicalDevelopment #Innovation
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Avidity Biosciences, Inc. which received early funding from CureDuchenne, announced positive initial data from their EXPLORE44 Trial for individuals with Duchenne amenable to skipping exon 44. Their experimental exon-skipping agent, AOC 1044 (del-zota), at 5 mg/kg increased exon 44 skipping by an average of 37% (from 7% at baseline to 44%) and increased average dystrophin levels by 25% (from 7% of normal at baseline to 32%) after 3 doses received once every 6 weeks. Creatine kinases levels were reduced to near normal levels. Register for the webinar on August 16, 2024, at 9 AM PST: https://lnkd.in/gEEhp-kc Learn more and read the letter to the community: https://lnkd.in/gq562NU7 #DMD #duchenne #exonskipping #CureDuchenne #AvidityBioscience #FDA #clinicaltrials
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?? There's still time to contribute to our collection "The Potential of Transferrin as a Drug Target and Drug Delivery System'' ?? led by the team: Dr. Nermi Parrow, Dr. Mallikarjuna Rao Pichika, Dr. Sidharth Chopra and Dr. Patricia Carrilho. ?? Check out the details here https://lnkd.in/dTSATNPG This Research Topic is committed to extending our understanding of Transferrin as a two-pronged weapon: as a drug target and an efficient drug delivery system. Link here to participate ?? https://lnkd.in/dmUbE5ki Manuscript Submission Deadline 15 March 2024 #Transferrin #DrugDelivery #ironmetabolism #metabolicpathways #translationalpharmacology
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?? CMTx Biotech, Inc. wins $299,993 award from Department of Health and Human Services, National Institutes of Health! ?? ?? The award is for a novel host-directed therapeutic for the treatment of sulfur mustard induced lung injury. CMTX Biotech is working to commercialize a pipeline of proprietary non-antibiotic chemically-modified tetracycline compositions and formulations for the host-modulatory treatment of diseases with high unmet needs. ?? ?? This is a project grant (B) award, categorized under Assistance Prime Transaction, and it will last for 1.92 years, starting from July 9, 2024, and ending on June 30, 2026. The research type is Grant/Subgrant/Subcontract. #SBIR #STTR #CMTXBiotech #DHHS #NIH #Innovation #Research #Awards
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At @Bristol Myers Squibb, we are all in on #TargetedProteinDegradation (TPD) and its potential for patients. As one of our differentiated research platforms, we’re expanding on our legacy in TPD by exploring potential new options across a broad range of diseases. We had great discussions about these advancements during?our annual European Scientific Innovation Summit this week. You can learn more about our efforts in TPD?here:?Science Firsthand: The pathway to drug discovery – Bristol Myers Squibb (bms.com) #BMS_Employee, #EUScientificInnovationSummit
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On Rare Disease Day, we send our congratulations to the PharmAust team on the recently released top line results from their Phase 1 MEND study, assessing monepantel?as a potential therapy for Motor Neurone Disease (MND) / Amyotrophic Lateral Sclerosis (ALS). This work has found that "monepantel displays a superior safety, tolerability to the leading FDA approved drug Relyvrio?", while "preliminary efficacy data shows a 58% reduction in the rate of disease progression for Cohort 2 (High Dose) using the FDA primary efficacy endpoint, ALSFRS-R". More detail on these results can be found in the below presentation, delivered by PharmAust CEO, Dr Michael Thurn. Alithia Life Sciences is extremely proud to be associated with this groundbreaking research and we look forward to supporting future phases of this study when they commence in the second half of 2024. #rarediseaseday #raredisease #rarediseases #motorneuronedisease #mnd #als #clinicalresearch #clinicaltrial #medicalresearch #scientificresearch #medicaltreatment
PharmAust Phase 1 MND Study Top-Line Results investor webinar
https://www.youtube.com/
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The US FDA has approved Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT)' Zoryve (roflumilast) for treating atopic dermatitis, the most common type of eczema affecting millions in the US. #FDAApproval #EczemaTreatment #ArcutisBiotherapeutics #Zoryve Find out more on Scientist Live - https://loom.ly/x3nYMy8 Subscribe to our newsletter here - https://loom.ly/km0X6OM #science #sciencenews #scientificresearch
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The US FDA has approved Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT)' Zoryve (roflumilast) for treating atopic dermatitis, the most common type of eczema affecting millions in the US. #FDAApproval #EczemaTreatment #ArcutisBiotherapeutics #Zoryve Find out more on Scientist Live - https://loom.ly/x3nYMy8 Subscribe to our newsletter here - https://loom.ly/km0X6OM #science #sciencenews #scientificresearch
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The video Mark mentions below is great to learn more about protein degradation!
At @Bristol Myers Squibb, we are all in on #TargetedProteinDegradation (TPD) and its potential for patients. As one of our differentiated research platforms, we’re expanding on our legacy in TPD by exploring potential new options across a broad range of diseases. We had great discussions about these advancements during?our annual European Scientific Innovation Summit this week. You can learn more about our efforts in TPD?here:?Science Firsthand: The pathway to drug discovery – Bristol Myers Squibb (bms.com) #BMS_Employee, #EUScientificInnovationSummit
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