Pacira BioSciences, Inc.’s Post

Better is possible. A brighter future with non-opioid solutions.

September is National Sickle Cell Awareness Month, a critical time to highlight the challenges faced by over 100,000 Americans living with sickle cell disease (SCD). Patients with SCD often require complex therapies, frequent hospitalizations and specialized medication management to prevent complications like organ damage. Learn more about our partner Children's National Hospital who treated the first patient with new sickle cell gene therapy. 🔗https://ow.ly/mgoE50TgLqf #SickleCellAwareness #SpecialtyPharmacy #ClearwayHealth #PatientOutcomes #MedicationManagement Source: https://ow.ly/B3XE50TgLqg

The gene therapy MCO-010 has been shown to enhance eyesight in patients with retinitis pigmentosa (RP), as seen by a recent clinical experiment in which 60% of participants reported better vision after 76 weeks. The company has shared that future trials could include those with Leber's congenital amaurosis (LCA), extending the therapy's benefits for those with inherited retinal diseases. To learn more about this: https://loom.ly/51Vfpws Image Description: Helix shape DNA stand with a blue background. #BlindCommunity #LowVision #VisuallyImpaired #Inclusion #LCA #LeberCongenitalAmaurosis #HopeInFocus #RP #RetinitisPigmentosa #ClinicalTrials #GeneTherapy #MCO010

For decades, treatment options for sickle cell disease have been limited to blood transfusions, pain management, and medications like hydroxyurea, which can reduce the frequency of sickle cell crises. However, these treatments only address symptoms and do not target the underlying genetic cause of the disease. Enter gene therapy, a groundbreaking approach that offers the potential to cure sickle cell disease by correcting the faulty gene responsible for the condition. Read more here: https://lnkd.in/eW7gKTUw

October is #BlindnessAwarenessMonth— a time to spotlight the challenges of vision loss and the advancements that aim to preserve and restore sight. This month has been especially meaningful for us as we mark a new chapter in the Opus journey to tackle inherited retinal diseases (#IRDs), a leading cause of blindness worldwide.    Opus is committed to developing transformative gene therapies for IRDs, and we are encouraged by the recent positive data from our lead gene therapy program for #LCA5. In a first-in-human, open label, Phase 1/2 dose-escalation trial, multiple assessments demonstrated clear signs of visual improvement in all participants, including in the FST functional test of light sensitivity and in mobility testing, and OPGx-LCA5 was well tolerated with no serious adverse events reported. We look forward to advancing this program, enrolling our first pediatric patients and sharing pediatric data in 2025.    For more details, check out our latest corporate presentation: https://lnkd.in/eBxueB8n   #InheritedRetinalDisease #GeneTherapy #OpusGenetics

Cerba Research is a proud supporter of #rarediseaseday to raise awareness of rare and orphan diseases. For us, this is not just one day, but part of our main goal to transform research and advance health for you! Over the past 6 years, Cerba Research has been part of over 120+ rare diseases clinical trials and over 26,700 patients screened. We also would like to take this time to thank all our employees who are in the front lines of rare diseases and cell and gene therapy research. They give their best every day to help advancing the development of new drugs and therapies. Check our laboratory solutions for your trial: https://lnkd.in/eh3sZttG #RareDiseasesDay #CerbaResearch #TransformingResearch #AdvancingHealth

Join Dr. Craig Kessler and Dr. Guy Young for a 1-hour accredited learning activity on the evolving therapeutic landscape of hemophilia. This activity covers therapies tailored to meet the individual needs of patients with Hemophilia A and Hemophilia B. The faculty share their insights and experiences with clotting factor concentrates, recombinant factors, factor VIII mimetics, rebalancing agents, and gene therapies. They will also discuss the safety and efficacy of late-phase investigational products that have the potential to address unmet needs and further transform patient care. https://lnkd.in/g4G7kvFR

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

Souad Messahel, PhD, Elpida Therapeutics on Clinical Trials in Rare Diseases - Strategies for Resource Management, exploring the development costs for a biotech for a gene therapy. #ASGCT24, within the “Why Do Rare Disease Clinical Trials Often Miss The Mark” workshop

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