CureDuchenne’s Post

Cell and gene therapies hold enormous potential. I'm hugely optimistic about their future. But we do ourselves a disservice if we don't acknowledge this fact: They don't always work. Lots of work still needs to be done. We owe it to little guys like Ben. https://lnkd.in/ggUj-zRb #genetherapy #cellandgenetherapy #biotechnology #innnovation

🎯 Topical gene therapy 🖍 FDA approved first-ever "Topical Gene Therapy" for treating wounds in patients with Dystrophic Epidermolysis Bullosa (DEB). https://lnkd.in/ggfRbps4 ✅ This application received Orphan Drug and Fast Track designations. ✅ In a placebo-controlled clinical trial including 31 subjects with DEB, 65% of topical gene-treated wounds completely closed, while only 26% of placebo-treated wounds closed completely. #topical #gene #fda #orphandrug #fasttrack #2023year

Helping our customers to better understand and manage the risks they face is a top priority. Roctavian is a groundbreaking gene therapy for the treatment of Hemophilia A. While it holds great potential, it also comes with a hefty $2.9 million price tag, which can create challenges for companies with self-funded health plans. Learn more about Roctavian in our current state of the market report: https://ow.ly/Enaj50PXkmJ If you’re a Berkley Accident and Health customer, we can offer cell and gene therapy insights and payment integrity services through our relationship with Emerging Therapy Solutions (ETS). #celltherapy #genetherapy #riskmanagement #stoploss #groupcaptives

#Estimation #Estimatecostofgenetherapy Creating and implementing a novel financial model that estimates the future number of gene therapy approvals across all therapeutic classes, the size of their targeted patient populations, and their prices. We use advanced simulation methods to conduct the analysis. The use of simulation, rather than purely deterministic methods, allows us to capture the inherent risks and uncertainty in costs, revenues, and other parameters of this new therapeutic class

Gene therapy represents a trans­for­ma­tive approach to medicine, but a few obstacles stand in the way of widespread adoption. DCVC Bio-backed Ascend is actively addressing them. Learn how from CEO Mike Stella. 👇 https://lnkd.in/gThSfEg3

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> FDA's Peter Marks single-handedly approved Sarepta’s gene therapy — what does that mean for everyone else?: Is Sarepta special? Or has the FDA set a new precedent for how it approaches treatments for devastating diseases with few options? On Thursday, the FDA announced it was expanding the allowed use of Sarepta’s gene therapy for Duchenne muscular dystrophy to a far wider group of patients. The treatment, Elevidys, will now be available to those ages 4 and older, an expansion that’s expected to make it available to 80% of patients with the disease, according to the company. But the move by the FDA, in which CBER Director Peter Marks overruled his own staff, is prompting new questions about just how the agency will approach similar drugs in the future. On Monday, ahead of the decision on Sarepta’s gene therapy, Endpoints News asked Marks about disagreement within the FDA. He said there’s a “spectrum” of views within the agency, but “our job as leaders is to help people feel like we’re going to lead them in a place where we get more products across the finish line.” “I’d like to move it from the baseline further towards a little bit of willingness to take risks or a moderate amount more toward taking more risks and hopefully people will come along,” Marks said. “That’s where we’re hoping to head in the coming months to years.” Staff overruled Memos accompanying Thursday’s action showed how Marks overruled two top FDA officials, including his deputy Nicole Verdun — the director of the Office of Therapeutic Products which the agency established last year under Marks to deal with a flood of new cell and gene therapies. Verdun’s lieutenant Lola Fashoyin-Aje recommended rejecting the therapy and running a new clinical trial. Fashoyin-Aje said there wasn’t enough evidence to show benefit in the patient populations for which Sarepta asked for approval, including the initial accelerated approval population of boys ages 4 and 5. Verdun, who like Marks has spoken about working more closely with patient groups, agreed with Fashoyin-Aje’s recommendation. Elevidys failed to meet the primary endpoint last year in a confirmatory study, but Sarepta cited key secondary endpoints suggesting the therapy may improve boys’ ability to stand and walk. In his memo, Marks called the secondary and exploratory data points “compelling,” and called the benefit-to-risk ratio “favorable, taking into consideration the severe unmet need and existing uncertainties.” “I’m just stunned that one person can override the decision of multiple career staff at the FDA with varied expertise,” said Reshma Ramachandran, an assistant professor of medicine at Yale University. “That is troubling to see that sort of power can be wielded this way.” But for patient advocates, Marks’ action… #lucidquest #genetherapy #celltherapy

https://lnkd.in/ge3fSBDn "Without big changes to the cost of gene therapies, they will never go global, Marks argued. Improving access to gene therapy is above all the most important thing that can be done to reduce prices." #genetherapy #aav #aavgenetherapy

📈🔬 Check out the results from the HOPE-B trial comparing Etranacogene Dezaparvovec (EDZ) gene therapy to extended half-life Factor IX therapies. 🧬 In a 24-month study, #EDZ demonstrated superior efficacy in reducing bleeds compared to rIX-FP, rFIXFc, and N9-GP. This novel gene therapy offers a potential shift from severe to mild #haemophilia or even complete amelioration with a single dose, eliminating the lifetime injection burden associated with traditional FIX therapies. 💉 Read more 🔗 https://lnkd.in/eEqGnm26 🔓 Open access #HaemophiliaB #genetherapy

🔬 Unveiling FDA's New Guidance on Cell and Gene Therapy Potency 🌟 At Revio, we're thrilled to highlight the FDA's latest initiative: their new draft guidance on potency assurance for cell and gene therapy products. 🧬 This guidance is crucial for professionals and companies in the field, providing science- and risk-based strategies to ensure the efficacy of these groundbreaking treatments. 💡 Discover key insights from this guidance, including potency assurance strategies, risk management, and more. 📆 The guidance is open for comments until March 27th, making now the perfect time to stay informed! 📖 Read our complete analysis on the Revio blog and join the conversation. Have questions or thoughts? The Revio team is here to assist! 🔗 Find the full analysis and more information at Revio blog link. #FDA #CellAndGeneTherapy #MedicalInnovation #Revio"

Great to read that this years #cellandgenetherapy GMP and Skills Demand market assessments from Cell and Gene Therapy Catapult report a sustained and continued growth in the UK’s advanced therapy and bioprocessing industries. The increase in both manufacturing capacity as well as the size of the advanced therapy skilled workforce makes the UK an attractive environment for advanced therapy set up and manufacture and provides a strong pipeline for future growth in the UK despite a challenging global economic landscape. #cellandgenetherapy #advancedtherapies #ATMPs