BloodStream Media’s Post

September is Inherited Retinal Disease Awareness Month. There are currently 2 million+ people worldwide who are at risk of losing their eyesight due to an inherited retinal disease, and we’re aiming to raise awareness and bring hope to those who may be suffering. Kiora Pharmaceuticals’ KIO-301 is an innovative drug aiming to restore functional vision by making retinal ganglion cells light sensitive. KIO-301 is not a gene therapy, but a small molecule potentially capable of restoring lost vision in patients with inherited retinal diseases. Learn more about KIO-301 here: https://lnkd.in/eA9HQ6My #kiorapharmaceuticals #visionrestoration #retinaldiseases #innovativemedicine #sightloss #InheritedRetinalDiseaseAwarenessMonth

The Cell and Gene Therapy Catapult featured in a supplement in The Guardian today for Rare Disease Day 2024, with our Chief Executive, Matthew Durdy, discussing the current pioneering #cell and #gene therapies for rare diseases and the potential to offer treatments for more common conditions in the future. Learn more: https://buff.ly/3uJ4u6K #CellTherapy #GeneTherapy #ATMPs #AdvancedTherapies #RareDiseases24

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

Join us in the webinar as we dive into how to conduct efficient trials in ophthalmology.

Blood Transfusions for Thalassemia: A Lifesaving Therapy Blood transfusions are a crucial part of managing thalassemia, a genetic blood disorder. In this video, we'll delve into the reasons why thalassemia patients need blood transfusions, how often they are typically needed, the process involved, and the potential risks and benefits. We'll also discuss alternatives like iron chelation therapy and emerging treatments like gene therapy. Our Channel Name] is committed to providing comprehensive and accurate health information. Join us as we explore the role of blood transfusions in thalassemia management and empower you with knowledge. Blood transfusions play a vital role in managing thalassemia. Watch our full video to understand why they are needed, how they work, and the potential risks and benefits for patients. If you or someone you know has thalassemia, understanding blood transfusions is essential. Watch the full video on our YouTube channel and share it with others to spread awareness. #ThalassemiaAwareness #Thalassemia #BloodTransfusion #ThalassemiaTreatment #BloodDisorder #GeneticDisorder #ThalassemiaAwareness #HealthEducation #MedicalTreatment

As we approach Rare Disease Awareness Day, Odylia honors our Brydge Solutions partnership with The Usher 2020 Foundation (Susie Trotochaud) and the FAUN Foundation. Through this partnership and the group of collaborators we have brought together we are developing a gene therapy to treat vision loss in patients with Usher Syndrome Type 1 caused by mutations in the USH1C gene. Patients have profound hearing loss usually from birth, vestibular dysfunction, and vision loss beginning in early adolescence. We are honored to work with these incredible foundations towards their goal of treating vision loss in USH1C patients. #rarediseaseawareness #raredisease #visionloss #genetherapy #drugdevelopment #rarediseaseawareness2024 #leapday #brydgesolutions #USH1C #UsherSyndrome

Our CEO Jean-Philippe Combal had a fantastic time at the 10th Annual LSX World Congress where he joined a panel of key industry leaders for a captivating discussion on investing in #CGT clinical strategy to guarantee funding success.   The remainder of the day was spent enjoying productive #networking opportunities, catching up with familiar faces, and making new connections.   Find out more about #Vivet and its novel and long-lasting #rAAV based gene therapies for rare inherited liver metabolic disorders including #WilsonDisease and #CTX in the link in the first comment below!   #AAVGeneTherapy #GenomicMedicines #GeneTherapy #GenomeEditing 

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

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

ADVANCE 2024's packed agenda is starts today at 10:00 a.m. ET. Don't miss out on all of the Sanfilippo-specific presentations and networking. You can still register for this free, global, community-wide conference at www.ADVANCESanfilippo.com. Day 1's agenda is packed with sessions about: - Immune responses, ethics of dose escalation, and community/patient perspectives on gene therapy risk/benefit & long-term follow up - Affected individuals and families share their personal viewpoints and experiences - Nutrition to support a healthy body and brain - The Eyes Have It: Impact on the retina and monitoring strategies in Sanfilippo disease - The advantages of qualitative interviews in clinical trials