Entrada Therapeutics reports preliminary data in healthy volunteers from Phase 1 ENTR-601-44-101 trial for Duchenne muscular dystrophy. Learn more here: https://lnkd.in/dMPZtt9a
Today we announced the selection of KB-7898, a p300 KAT inhibitor, our first development candidate for #autoimmunediseases, for the potential treatment of #Sjögrensdisease, a chronic #autoimmunedisease marked by the production of autoantibodies, ongoing inflammation and lymphocytic infiltration of the exocrine glands which may lead to uncomfortable dryness symptoms, known as sicca. Preclinical data will be presented at #ACRConvergence2024. Learn more: https://bit.ly/4eB3O54
See results from a 5-year follow-up of a phase 3 trial evaluating the efficacy and safety of two ALK inhibitors in patients with advanced ALK-positive non-small cell #LungCancerhttps://bit.ly/4e1Lvps
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Translating potential into benefit is a long journey when it comes to drug development, and initiatives such as the FDA’s RMAT (Regenerative Medicine Advanced Therapy) designation help accelerate promising drugs in development so that they can make their way to patients more quickly.
We are thrilled to announce that BlueRock Therapeutics cell therapy candidate for Parkinson’s disease has been granted RMAT designation, and look forward to seeing this candidate further advance in clinical trials.
Learn more: http://spr.ly/6046edwT8#TeamBayer
July Featured Article: Novel therapeutics for small abdominal aortic aneurysms: Recommendations for rodent model research @honglu227 @curciAAA @BloodVesselDoc1
https://lnkd.in/gKbpZ93r
🔊 It's great news that BlueRock received FDA Regenerative Medicine Advanced Therapy designation for Parkinson’s disease cell therapy candidate #bemdaneprocel. As of March 2024, the FDA had granted 109 RMAT destinations, of which only Neurocrine and Voyager's gene therapy (VY-AADC) was for Parkinson's disease.
Here are some drugs that have been approved by the FDA via RMAT designation:
◾ Gene Therapy #Lenmeldy by Orchard Therapeutics, for the treatment of children metachromatic leukodystrophy
◾ Cell Therapy #Amtagvi by Iovance Biotherapeutics, Inc. Biotherapeutics, for the treatment of adult metastatic melanoma
◾ Gene Therapy #Lyfgenia by bluebird bio for the treatment of sickle cell disease
◾ Gene Therapy #Casgevy by Vertex Pharmaceuticals for the treatment of sickle cell disease
◾ Gene Therapy #Roctavian by BioMarin Pharmaceutical Inc. for the treatment of adults with severe hemophilia A
◾ Gene Therapy #Vyjuvek by Krystal Biotech, Inc. for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa
◾ Tissue based therapy #Rethymic by Enzyvant Therapeutics for the treatment of Immune reconstitution in pediatric patients with congenital athymia
◾ Tissue based therapy #StrataGraft by Stratatech for the treatment of deep partial thickness burns
◾ Cell Therapy #Breyanzi from Juno Therapeutics/Bristol Myers Squibb for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma
NEWS: Today Bayer AG and BlueRock Therapeutics announced that BlueRock’s investigational cell therapy bemdaneprocel for the treatment of Parkinson’s disease has been granted Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA). Read the full release: https://lnkd.in/d7fDEDVs
✨ 𝗙𝗗𝗔 𝗚𝗿𝗮𝗻𝘁𝘀 𝗘𝘅𝗽𝗲𝗱𝗶𝘁𝗲𝗱 𝗔𝗽𝗽𝗿𝗼𝘃𝗮𝗹 𝘁𝗼 𝗜𝗼𝘃𝗮𝗻𝗰𝗲 𝗕𝗶𝗼𝘁𝗵𝗲𝗿𝗮𝗽𝗲𝘂𝘁𝗶𝗰𝘀 𝗔𝗺𝘁𝗮𝗴𝘃𝗶 𝗧-𝗖𝗲𝗹𝗹 𝗧𝗵𝗲𝗿𝗮𝗽𝘆 𝗳𝗼𝗿 𝗦𝗼𝗹𝗶𝗱 𝗧𝘂𝗺𝗼𝗿𝘀! 🌐
𝗜𝗻 𝗙𝗲𝗯𝗿𝘂𝗮𝗿𝘆 𝟮𝟬𝟮𝟰, The FDA authorizes Iovance Biotherapeutics, Inc. groundbreaking T-cell #therapy treatment 💉 for solid tumors. The "#Amtagvi" medication is the first tailored #tumor-infiltrating #lymphocyte (TIL) treatment available on the market. For patients with metastatic #melanoma who have previously had therapy with a PD-1 inhibitor—and a #BRAF inhibitor if the tumor possesses a BRAF V600 mutation—the FDA on Friday awarded it an expedited approval. Iovance is charging $515,000 per patient for the one-time therapy, which is the wholesale acquisition cost. The quoted price💲of the medication is somewhat more than that of the current CAR-T #cell treatments.
𝐁𝐫𝐨𝐰𝐬𝐞 𝐚𝐭 𝐨𝐮𝐫 𝐋𝐢𝐧𝐤𝐞𝐝 𝐑𝐞𝐩𝐨𝐫𝐭👉 https://bit.ly/42M63NI#tumor#immunotherapy#drugapproval#PD1Inhibitor#prophecy#maretresearch#prophecymarketinsights
How can you ensure your FMT assay is as efficient as possible? Our innovative Fibroblast-to-Myofibroblast Transition (FMT) Assay allows high-throughput in vitro evaluation of anti-fibrotic therapeutics.
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Fantastic update!