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#Pfizer reported results from a phase 3 trial, #CIFFREO, evaluating an investigational recombinant adeno-associated virus #genetherapy known as fordadistrogene movaparvovec among ambulatory boys ages 4 to 7 years with #Duchenne muscular dystrophy. CIFFREO failed to hit its primary goal of improving #motorfunction when compared to placebo at one year of treatment, as assessed by a movement scale known as the #NorthStarAmbulatoryAssessment. Key secondary endpoints, such as 10-meter run/walk velocity and #timetorise, also failed to demonstrate a significant difference between fordadistrogene movaparvovec and placebo. These results come a little more than a month after Pfizer reported the death of a young boy participating in a related phase 2 trial called DAYLIGHT, which included boys with DMD ages 2 to 3 years. The boy had received the gene therapy in early 2023 and died from cardiac arrest on May 3 of this year. It will be interesting to review more detailed results from both of these studies to better understand the reasons why the trial failed and more importantly, whether the death of the young boy was related to the treatment. These learnings will be critical in improving future #clinicalresearch and #development of gene therapies for DMD. #DMD #AAVgenetherapy #musculardystrophy #adverseevent #drugdevelopment #challenges

Sad news from Pfizer DMD clinical trial!Pfizer reported a tragic incident during a Phase 2 clinical trial for a potential gene therapy aimed at treating Duchenne muscular dystrophy (DMD), a severe genetic disorder that causes muscle degeneration. A young participant tragically passed away due to cardiac arrest after being treated with the experimental therapy, fordadistrogene movaparvovec. This occurred within the DAYLIGHT study, which involves young boys aged between 2 and 3 years, all suffering from DMD—a condition affecting approximately one in 3,500 male births globally.The incident was disclosed in a community letter from Pfizer's DMD gene therapy team, which was shared by a non-profit advocacy group. The trial, which began in August 2022, is set to continue monitoring participants for five years post-treatment, aiming for completion in early 2029.Following this event, Pfizer and an independent external data monitoring committee have commenced a detailed review of the data to determine the possible cause of the adverse event. Additionally, Pfizer has temporarily halted dosing in the cross-over part of another late-stage study, CIFFREO, although this pause does not affect other ongoing studies where dosing is already complete.Despite this setback, Pfizer remains optimistic about the progress of the CIFFREO trial and expects to begin primary analysis soon, with preliminary results anticipated in the near future. #pfizer #DMD #genetherapy https://lnkd.in/d2bq4gvu

🔬 Atsena Therapeutics Completes Part A Dosing in XLRS Gene Therapy Trial  Atsena Therapeutics has reached a significant milestone in its Phase I/II LIGHTHOUSE study evaluating ATSN-201 for X-linked retinoschisis (XLRS). ATSN-201 utilizes Atsena’s novel AAV.SPR spreading capsid, designed to achieve therapeutic gene expression in the central retina without the risks of foveal detachment. 👁️ XLRS, a monogenic retinal disease primarily affecting males, causes abnormal retinal layer splitting, leading to vision loss. With no approved treatments available, ATSN-201 offers new hope to approximately 30,000 affected individuals in the U.S. and EU. 🔗 Read the full story and learn about this breakthrough here: https://lnkd.in/dfTcAtb3 #GeneTherapy #ClinicalTrials #EyeHealth #Innovation #InheritedRetinalDiseases #XLRS

I see 4DMT has streamlined its ocular pipeline by ditching their other assets including their gene-therapy delivered shortened CFH programme in Geographic Atrophy. 4dMT were one of a number of companies looking to introduce optimised forms of FH into the eye as a treatment for dry AMD. Now focusing purely on their late-stage gene therapy asset in neovascular AMD. 4DMT tosses 2 programs, halts funds for 3 others as it zeroes in on latest-stage clinical gene therapy

🚀 Strategic Alliance Sends Regenxbio Soaring: $110M Upfront & $700M Milestone Potential 🌟 REGENXBIO and Nippon Shinyaku ’s strategic partnership is shaking up gene therapy innovation with a transformative deal. 💡 Key Highlights: Financial Impact: $110M upfront, up to $700M in milestones, plus double-digit royalties. Strategic Growth: Access to U.S. and Asian markets to deliver cutting-edge treatments. Therapeutic Impact: Advancing promising gene therapies for rare metabolic disorders, including Hunter syndrome (RGX-121) and mucopolysaccharidosis type I (RGX-111). #GeneTherapy #RareDiseases #advancedtherapies #BiotechInnovation #HealthcareInnovation #PrecisionMedicine #StrategicPartnerships #LifeSciencesInvesting #BiotechDeals #HealthcareInvestments #BiotechBusiness #biotech #lifesciences

"Zolgensma as an intravenous infusion has been approved in the U.S. for children under 2 years with SMA since 2019. The intrathecal version injects the gene therapy directly into the spinal cord, thereby allowing for a smaller dose needed to have enough therapeutic payload reaching the motor neurons, Daniel Grant, VP & global program head of some of Novartis’ gene therapy projects, explained in an interview with Fierce Pharma."

Elpida Therapeutics to start Phase III gene therapy trial for SGP50: Aman who set up a rare disease biotech in a bid to cure his son’s disease will soon be initiating a Phase III trial of the gene therapy in the US. Elpida Therapeutics has been conducting a #Phase I/II trial (NCT05518188) of its gene therapy candidate Melpida for patients with spastic paraplegia type 50 (SPG50). The US Food and Drug Administration (FDA) has now given the rare disease biotech the green light to initiate a Phase III study which will dose eight children and begin in August 2024. Terry Pirovolakis started Elpida Therapeutics, a not-for-profit biotech, after his son Michael was diagnosed with the rare disease at five months old. https://lnkd.in/e-xGMa8a #RegulatoryStrategy #RegulatoryCompliance

What's new in #cellandgenetherapy? This week, the sector showed promising early-stage clinical updates. 🧬1st patient dosed in gene therapy trial to treat Fabry disease 🧫Gamma-delta T-cell therapy has promising effectiveness in treating AML 🔬Cell therapies for multiple sclerosis and progressive myasthenia gravis enter clinical trials Read the full stories below and subscribe to our Sector Vector newsletter for more weekly news on cell and gene therapy clinical, business, and manufacturing. 📩 https://lnkd.in/eD7ZNaU uniQure announced this week that the first patient has been dosed in a Phase I/IIa clinical trial of its gene therapy, AMT-191, for the treatment of Fabry disease, a rare, inherited genetic disease. https://lnkd.in/eMiznUxe IN8bio announced early clinical data that showed 100% of treated AML patients were in complete remission. The company plans to submit an IND application in 2025. https://lnkd.in/eZid5Y3z Precision BioSciences, Inc. and TG Therapeutics, Inc. announced that the FDA has approved an IND application for azer-cel to be used in human clinical trials for the treatment of progressive forms of multiple sclerosis. https://lnkd.in/eR_Rfgcd Kyverna Therapeutics announced that the FDA granted the Regenerative Medicine Advanced Therapy (RMAT) for its autologous, fully human CD19 CAR-T cell product candidate, which will be used to treat patients with progressive myasthenia gravis. https://lnkd.in/eCs5Rryc

Breaking: The FDA granted approval of expansion to the label indication for Sarepta’s Elevidys (delandistrogene moxeparvovec), for individuals with Duchenne muscular dystrophy (DMD) who are at least four years of age and have mutations in a specific DMD gene. The FDA grated traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients contingent upon verification of clinical benefit in a confirmatory trial. Elevidys is the first gene therapy for Duchenne muscular dystrophy - a genetic disorder that affects around 1 in 3,500 boys. Children with DMD are unable to make a protein called dystrophin, resulting in progressive muscle degeneration and death around the age of 30 due to heart or respiratory failure. The price for Elevidys is $3.2M (manufacturers invoice price for the one-time infusion) not including administration costs. The Cell & Gene therapy landscape is a significant focus for our health care system as an unprecedented number of treatments for rare and devastating diseases enter the market. Payers face volume uncertainty, clinical effectiveness, and concern for the extraordinary costs. With numerous therapies costing $4M+ not including administration charges, payers need to explore affordable solutions. Sequoia provides specialty financial and clinical resources needed to assess and manage this novel exposure. Contact us to learn how. https://lnkd.in/g3hGHAPM

🎯 𝐔𝐧𝐜𝐨𝐯𝐞𝐫 𝐭𝐡𝐞 𝐬𝐭𝐫𝐚𝐭𝐞𝐠𝐢𝐜 𝐦𝐨𝐯𝐞𝐬 𝐝𝐫𝐢𝐯𝐢𝐧𝐠 𝐢𝗻𝐧𝐨𝐯𝐚𝐭𝐢𝗼𝗻 𝐢𝐧 𝐭𝐡𝐞 𝐅𝐫𝐞𝐧𝐜𝐡 𝐛𝐢𝗼𝗺𝗮𝗻𝘂𝗳𝗮𝗰𝘁𝘂𝗿𝗶𝗻𝗴 𝐬𝐞𝐜𝐭𝐨𝐫 👇 #09JAN25 1️⃣ Coave Therapeutics secures €32M to advance its ALIGATER™ genetic medicines platform. 🔎 𝗣𝗼𝘀𝗶𝘁𝗶𝗼𝗻𝗶𝗻𝗴: Coave focuses on precision genetic medicines for CNS, neuromuscular, and eye diseases. For example, the retinal gene therapy market alone is expected to reach $2.6 billion by 2030, reflecting strong growth in targeted delivery solutions. 👉 𝗦𝘁𝗿𝗮𝘁𝗲𝗴𝗶𝗰 𝗜𝗺𝗽𝗹𝗶𝗰𝗮𝘁𝗶𝗼𝗻𝘀: This funding strengthens Coave’s platform and pipeline, enabling the progression of two lead candidates to IND-enabling studies by 2026, and enhancing its appeal for collaborations. 2️⃣ MaaT Pharma achieves positive Phase 3 results for MaaT013 in GI-aGvHD. 🔎 𝗣𝗼𝘀𝗶𝘁𝗶𝗼𝗻𝗶𝗻𝗴: GI-aGvHD impacts approximately 3,000 third-line patients annually across Europe and the U.S., with a dire 15% one-year survival rate in refractory cases. MaaT Pharma’s MaaT013 could be the first approved therapy for this indication. 👉 𝗦𝘁𝗿𝗮𝘁𝗲𝗴𝗶𝗰 𝗜𝗺𝗽𝗹𝗶𝗰𝗮𝘁𝗶𝗼𝗻𝘀: With its groundbreaking Phase 3 data, MaaT013 is poised to revolutionize treatment for GI-aGvHD. EMA submission is expected mid-2025, accelerating access to this life-saving therapy. 3️⃣ Sensorion finalizes recruitment for the Phase 1/2 Audiogene gene therapy trial. 🔎 𝗣𝗼𝘀𝗶𝘁𝗶𝗼𝗻𝗶𝗻𝗴: Sensorion targets congenital hearing loss due to OTOF mutations, a condition affecting approximately 1 in 25,000 newborns globally, with no approved treatments currently available. 👉 𝗦𝘁𝗿𝗮𝘁𝗲𝗴𝗶𝗰 𝗜𝗺𝗽𝗹𝗶𝗰𝗮𝘁𝗶𝗼𝗻𝘀: Successfully completing the first cohort in this trial validates Sensorion’s ability to address this unmet need. Their 2025 webinar could provide critical data to accelerate recognition and development of SENS-501. 💡 Which of these milestones will have the greatest impact on French biomanufacturing? Share your thoughts below! 👇