Asklepios BioPharmaceutical, Inc. (AskBio)’s Post

Osteoarthritis, one of the most common and debilitating chronic diseases, affects more than 30 million Americans, a number that is expected to increase exponentially as the population ages. Therapeutic options are limited. Current approaches start with nonpharmacological treatments such as walking aids, weight loss, and physical therapy, followed by acetaminophen and NSAIDS. Pioneers working on gene therapies for osteoarthritis and other chronic joint conditions won the prestigious OREF Award for 2024 at this year’s AAOS. One of the winners, Christopher Evans, PhD, discusses 30 years of research leading up to the founding of Genascence Corporation, which is now in clinical trials for a gene therapy for OA: https://bit.ly/3VDAh3R #medtech #osteoarthritis #chronicdisease #jointcondition #genetherapy

Gene Therapy: A New Hope for Rare Diseases Gene therapies are leading the charge in treating rare and life-threatening conditions. From spinal muscular atrophy to hereditary transthyretin amyloidosis, these breakthrough therapies are offering new hope by addressing diseases at their genetic root. Dive into the latest clinical trial successes and see how these advancements are reshaping the future of medicine. 🧬 #RareDiseases #GeneTherapy #FutureOfMedicine

📢 #FDA granted AMT-130 the first-ever Regenerative Medicine Advanced Therapy designation for #Huntington’sdisease. #UniCure shared positive interim data from two Phase I/II studies on its gene therapy for Huntington’s disease. The high-dose cohort showed an 80% slowing of disease progression, while the low-dose group saw a 30% slowing. 💥 Additionally, neurofilament light protein levels, a biomarker of neurodegeneration, reduced by 11% across all patients. #genetherapy #FDA #RMAT #Huntingtondisease #Clinicaltrial #Phase1/2

Cell and gene therapies (CGT), also known as “Advanced Therapy Medicinal Products” (ATMP), address diseases by directly repairing genetic defects within the body or introducing tissues or cells with new functions. These innovative therapies can correct or cure a wide range of conditions. The development and manufacture of these medicines are complex and novel, and they have only recently started gaining traction in the commercial sphere over the past decade. What sets them apart is their remarkable potential to offer permanent cures rather than temporary symptom reduction. Get an overview in the latest article by VTU experts Cornelia Haas and Birgit Krenn, which appeared in the magazine “botenstoff” of the Human.technology Styria GmbH (February 2024 issue, Precision Medicine special edition). #VTU #CellandGeneTherapy

Parkinson's disease is the second most common neurological disease. There is currently no cure, but there are treatments that can help to manage the symptoms. In a recent study published in Gene Therapy, researchers utilize the CRISPR-Cas9 system to induce the synthesis of dopamine (DA) in the brains of a rat model for Parkinson's disease (PD). Follow the link in the comments to learn more. #ParkinsonsDisease #LifeSciences #News #ClinicalResearch

As one of the contributing authors, I am proud to present Recent Advances in Targeted Therapy of Diseases (Nano Drug Delivery Systems and Gene Therapy). This book delves into the latest innovations in targeted therapeutic strategies, emphasizing the role of nano drug delivery systems and gene therapy in transforming disease treatment. It offers a deep dive into current research and breakthroughs, serving as a vital resource for healthcare professionals and researchers dedicated to the advancement of precision medicine.

#CellTherapy and #GeneTherapy are complex modalities and every program is truly unique. Does your commercial strategy take into account nuances specific to your drug?

What IF... together, we could overcome the challenges of LNP encapsulation at high mRNA concentrations? High mRNA levels are essential for therapies like protein replacement and gene therapy. At Innoforce, our advanced LNP technology delivers outstanding encapsulation efficiency, even at elevated mRNA concentrations. 💡For example, with mRNA concentrations of 2.5 mg/mL, we achieve over 97% encapsulation efficiency, keeping empty capsids below 6%. Explore how Innoforce can help you meet the challenges across a wide range of mRNA concentrations. ☞https://lnkd.in/gQRhKU-U #InnoforcePharma #PlasmidDNA #RNA #LNP #ViralVectors #CellTherapy

Mission possible: Gene therapy for inherited metabolic diseases Supported by improved knowledge gathered from numerous ongoing clinical trials for inborn errors of metabolism, the translation of innovative gene therapy for rare diseases, which remains a long and arduous journey, is gradually reaching patients, and is uplifted by the hope of a brighter outcome and a much-improved quality of life. Themed issue of Journal of Inherited Metabolic Disease https://lnkd.in/eSu-_8p8

We are honored to share that Andelyn has been selected by Armatus Bio, Inc. to accelerate manufacturing of their gene therapy treatment for Charcot-Marie-Tooth Type 1A (CMT1A), a rare genetic neurological disease associated with independence-limiting disability and risk of fatal complications that has no approved therapies today.   Charcot-Marie-Tooth disease type 1A (CMT1A) is a type of inherited neurological disorder that affects the peripheral nerves. People with this disease experience weakness and wasting (atrophy) of the muscles of the lower legs beginning in adolescence; later they can also have hand weakness and sensory loss. CMT1A is caused by having an extra copy (a duplication) of the PMP22 gene (NIH).   We are truly grateful to play an important role in directly transforming patient lives. https://lnkd.in/e3WBkyYU   #GeneTherapy #RareDisease #CDMO #aav