Erin Harris’ 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

2025 Outlook For Cell & Gene Therapy! I'm excited to see the incredible advancements in cell and gene therapies coming in 2025. Companies like Kiji Therapeutics, Orgenesis, and Precision BioSciences are pushing the boundaries, expanding treatments beyond oncology to genetic disorders, autoimmune diseases, and cardiovascular conditions. With improvements in gene-editing and scalable manufacturing, these innovations are set to make personalized medicine more accessible and impactful than ever before! https://lnkd.in/ee8WPsxU

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

I am happy to share that our latest research paper, "Rabies virus-mimicking liposomes for targeted gene therapy in Alzheimer’s disease," has been published in the International Journal of Pharmaceutics, one of the prestigious journals in our field. Thanks to all authors Hasan Akbaba, Ozan Karaman, Ezgi Turunç Özoğlu, Tian Tian for their valuable contributions and especially  Prof. Bakhos Tannous for his continuous support. Also, I would like to thank TÜBİTAK for supporting this study by 3501 - Career Development Program.   In this study, a gene carrier system combining the synergistic effect of virus-mimic RVG29 ligand with bioinspired liposome that imitates the natural lipid bilayers of cell membranes was developed for brain-targeted RNAi therapeutics. You can access the article from the link below:

Excited to share a new publication in the Journal of Molecular Therapy which highlights the eShunt®️ Platform as a CNS gene therapy delivery method. Thanks to the teams at UMass Chan Medical School and Tufts Medical Center for your work on this study. "We're very excited about these promising preclinical results, which suggest that the eShunt System delivery approach could provide biopharmaceutical and biotechnology organizations with a minimally invasive method for delivering gene therapies directly to the cortex and brain." #bbb #CNS #drugdelivery #neurovascular #endovascular https://lnkd.in/eeKCGPm7

What is Duchenne Muscular Dystrophy(DMD)? DMD is a rare and devastating neuromuscular disorder caused by mutations in the dystrophin gene. DMD leads to progressive muscle weakness that worsens over time, ultimately affecting a person's ability to walk, breathe, and perform everyday tasks. It is an X-linked disorder, meaning it primarily affects males. It is fatal and occurs in approximately 1 in every 3500-5000 male births worldwide. In a recent breakthrough, the FDA has expanded approval for Elevidys, the first gene therapy designed to treat DMD. Elevidys works by introducing an engineered gene that codes for a shortened version of dystrophin. Click on the link below to learn more! https://lnkd.in/grFNRYJp

REGENXBIO Gene Therapy Advances to Pivotal Trials in DMD “There remains a critical need for new therapeutic options for patients with Duchenne muscular dystrophy,” said Aravindhan Veerapandiyan, MD, child neurologist, Arkansas Children's Hospital. "The safety, functional, and biomarker data shared today reinforce the positive feedback from families, highlighting improvements in patients’ daily activities and underscoring the potential benefits of this treatment.”

Gene therapy has emerged as a promising frontier in modern medicine, offering revolutionary treatments for a range of diseases previously considered incurable. Dr. Bruce Levine, the co-inventor of Kymriah, the first FDA-approved gene therapy, recently shared some insights about what goes into spearheading such groundbreaking research and discoveries. Dr. Levine, who was also instrumental in advancing CAR T-cell therapy for cancer and driving progress in gene editing, notably with CRISPR, shared his visions for future projects aimed at improving patient outcomes and transforming healthcare. Read the summary and highlights from this recent discussion: What's Next in Cell and Gene Therapies: Anticipating the Next Advanced Therapy Breakthroughs. https://hubs.ly/Q02p12Ft0 #genetherapy #CGT #pharmaceuticalmanufacturing

Novartis to Acquire Kate Therapeutics, Expanding Gene Therapy Portfolio Novartis has agreed to acquire San Diego-based Kate Therapeutics in a deal worth up to US $1.1 billion. Kate specializes in gene therapies for neuromuscular diseases, including Duchenne muscular dystrophy (DMD). Read more: https://lnkd.in/d94-Ybmn #Novartis #GeneTherapy #DuchenneMuscularDystrophy #BiotechAcquisition #Neurology #PharmaInnovation

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.