Rejuvenate Bio’s Post

Rejuvenate Bio Awarded $4M Grant By CIRM For Gene Therapy Development Learn more & get our take 👇 https://lnkd.in/gASTSZ9A “DSP ACM is a rare, severe, life-threatening, and debilitating disease that typically manifests in young adults as a high risk of life-threatening ventricular arrhythmias, sudden cardiac death, and progression to heart failure. There is currently no disease modifying therapy for patients with this disease, and our gene therapy RJB-0402 addresses a significant unmet medical need.” — Daniel Oliver, CEO & Co-Founder at Rejuvenate Bio “The funding and strategic support from CIRM will accelerate development of our gene therapy candidate RJB-0402 into clinical trials for DSP ACM patients in desperate need of new therapies. We are confident that RJB-0402, a one-time gene therapy, could be a transformative regenerative medicine and the first disease modifying therapy to address the unmet medical need of DSP ACM patients, and the recent funding from CIRM will enable us to initiate our first in human clinical trial.” — Noah Davidsohn, Ph.D., Chief Scientific Officer & Co-Founder at Rejuvenate Bio “Our goal is to move the most promising research forward. A one-time gene therapy treatment for patients with this rare cardiac disease DSP ACM, would have significant impact for patients with this degenerative disorder. We look forward to supporting Rejuvenate Bio in bringing this regenerative therapy to patients with this degenerative disease.” — Dr. Abla Creasey, Vice President of Therapeutics Development at California Institute for Regenerative Medicine (CIRM) #aging #genetherapy #SoHCNews

🔹 In an unprecedented breakthrough, scientists at the Children's Medical Research Institute have successfully conducted the first gene therapy trials on a complete human liver, marking a milestone in the quest for effective treatments against severe inherited diseases. Using a normothermic liver perfusion system, this pioneering study opens up new possibilities for directly assessing adeno-associated virus-based therapies in the target organ, potentially overcoming current limitations of preclinical models and accelerating the development of more efficient and safer gene therapies. This innovative approach not only promises to improve the precision of therapeutic dosing and the identification of side effects but also to reduce reliance on animal trials, bringing us one step closer to real solutions for patients facing diseases with limited treatment options. Follow the link below to learn more 👉🏼 https://lnkd.in/dznmPyAW #techtitute #medicine #genetherapy #perfusion

4D Molecular Therapeutics is revolutionizing cystic fibrosis (CF) treatment with its innovative inhaled gene therapy candidate, 4D-710. Promising interim data from a Phase 1/2 clinical trial have shown that a single dose of 4D-710 can improve or stabilize lung function in adults with CF who are ineligible or intolerant to CFTR modulators. The trial also revealed a dose-dependent increase in CFTR protein production in lung samples, highlighting the therapy's potential. Buoyed by these encouraging results, 4D Molecular Therapeutics is planning a Phase 3 trial to seek regulatory approval for 4D-710. At the upcoming 15th Australian Cystic Fibrosis Conference in Brisbane, Alan H. Cohen, MD, Senior Vice President of Clinical Development and Therapeutic Area Head of Pulmonary Medicine at 4D Molecular Therapeutics, will present the promising interim results of gene therapy. Dr. Cohen, who oversees the gene therapy programs at 4D Molecular Therapeutics, is also leading pioneering research in other cardiopulmonary diseases, including Fabry Disease and Alpha1-Antitrypsin Deficiency. His visionary leadership is crucial in advancing these innovative treatments that have the potential to significantly improve patient outcomes for these serious conditions. To read more article: https://lnkd.in/g9_tnih2 To know more the conference: https://lnkd.in/gNH__PNN #4DMT #CysticFibrosis #CFAustralia #ModecularTherapeutics #CFTR #Conference

Check out this "Behind the Paper" blog post from Tenaya describing how we selected some of the critical features for TN-401 , our investigational gene therapy for PKP2 ARVC.

Humanized mouse models are crucial in cell and gene therapy development, providing adaptable platforms for disease research and therapy evaluation. Learn about the versatility of these models and how they're accelerating the path to effective treatments. https://lnkd.in/gtjC_jBZ

In 2023, cell and gene therapy saw seven FDA approvals, with expectations of 17 more in 2024. The FDA's accelerated approvals are encouraging for these costly therapies. Notable approvals included BMS's Breyanzi, Iovance's #Amtagvi, and Orchard's #Lenmeldy. Vertex and CRISPR's Casgevy was the first #CRISPR-based gene editing therapy approved in the U.S. Bluebird bio's #Lyfgenia and Sarepta's #Elevidys also received approvals. Looking ahead, Rocket's #Kresladi, BMS's #Breyanzi for follicular lymphoma and mantle cell lymphoma, and Pfizer's #Beqvez for hemophilia B are anticipated for 2024. #OBI4You #CellTherapy #GeneTherapy https://zurl.co/YQsL

Scientists at the Children's Medical Research Institute have made a groundbreaking advancement in gene therapy by testing AAV-based therapies in whole human livers using an ex situ normothermic perfusion system. This novel method allows for the direct evaluation of gene therapies within the human liver, overcoming the challenges posed by traditional preclinical models. Key findings include the superior performance of AAV-SYD12 and AAV-LK03 variants under non-neutralizing conditions and the identification of challenges related to neutralizing antibodies. This research opens new possibilities for developing effective liver-directed gene therapies, marking a significant step forward in personalized medicine. https://lnkd.in/g4YHcvxd

Ocugen completes dosing in second cohort of OCU410 geographic atrophy trial Biotechnology company Ocugen has completed dosing in the second cohort of its Phase I/II ArMaDa clinical trial of modifier gene therapy candidate OCU410 (AAV-hRORA). OCU410 is a modifier gene therapy candidate targeting geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD).  It makes use of AAV delivery platform for the retinal delivery of the RORA (ROR Related Orphan Receptor A) gene.  Ocugen chief medical officer Dr Huma Q. said: “OCU410 regulates multiple pathways involved with the disease, including lipid metabolism, inflammation, oxidative stress, and membrane attack complex (complement) with a single sub-retinal injection.” Last month, Ocugen completed subject dosing in the first cohort of its Phase I/II ArMaDa clinical trial of OCU410 (AAV-hRORA). #ocugen #biotechnologyresearch #clinicaltrials https://lnkd.in/ewuwX4DV

We're proud to share an exciting milestone reached by Zhongmou Therapeutics Technology (Wuhan) Co., Ltd. in the fight against inherited retinal diseases. Their latest clinical data reveal significant advancements in optogenetic gene therapy, specifically targeting Retinitis Pigmentosa (RP). This therapy, known as ZM-02, has demonstrated notable improvements in patient vision, offering hope to millions worldwide suffering from this degenerative condition. A universal solution to a previously incurable challenge, ZM-02 stands as a testament to the power of innovation and dedication in healthcare. Congratulations to the team at Zhongmou Medical for their groundbreaking work in ophthalmology. A pivotal component of this success is the collaboration with PackGene Biotech, Inc., who provided the essential Adeno-Associated Virus (AAV) vectors used in the therapy. This partnership exemplifies how collaboration and innovation can lead to groundbreaking advancements in medical science. Learn more: https://lnkd.in/gVvdF6-M #GeneTherapy #RetinitisPigmentosa #Optogenetics #AAV