Latest news with #gene therapy
Bloomberg
6 days ago
- Business
- Bloomberg
Regenxbio's Gene Therapy Helps Kids with Deadly Muscle Disease
Children with a deadly muscle disorder had more control of their bodies after getting an experimental gene therapy from Regenxbio Inc. in a small study. The results foreshadow a potential rivalry with Sarepta Therapeutics Inc. and a critical test for the Trump administration's new drug regulators. The first five children with Duchenne muscular dystrophy to receive a higher dose of Regenxbio's treatment showed 'consistent benefit' after nine months, with improvements in motor function and the time it took to stand, walk and climb, the company said Thursday. It's conducting a larger study involving about 30 patients that it expects to be in full swing by the end of the year, with the potential to file for approval by mid-2026.
Associated Press
02-06-2025
- Business
- Associated Press
uniQure Provides Regulatory Update on AMT-130 for Huntington's Disease
~ Alignment with FDA continues to support Accelerated Approval pathway ~ ~ BLA submission planned for first quarter of 2026 ~ ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, June 02, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today provided a regulatory update on AMT-130, its investigational gene therapy for the treatment of Huntington's disease. Following recent Type B meetings and further guidance from the U.S. Food and Drug Administration (FDA), the Company has reached alignment with the FDA on several key components of the statistical analysis plan and Chemistry, Manufacturing and Controls (CMC) information that will support a Biologics License Application (BLA) submission expected in the first quarter of 2026. 'We are very pleased with our continued, productive engagement with the FDA and the progress we've made toward a planned BLA submission for AMT-130 in the first quarter of 2026,' said Walid Abi-Saab, M.D., chief medical officer of uniQure. 'We are pursuing an accelerated approval pathway supported by multiple years of clinical data – a rigorous and differentiated approach that reflects the urgent need in Huntington's disease and our commitment to delivering the first disease-modifying treatment for people affected by this devastating disease. We are grateful to the FDA for their continued engagement and look forward to sharing three-year top-line data in the third quarter of 2025.' Statistical Analysis Plan In the second quarter of 2025, the Company held a Type B meeting with the FDA to discuss the proposed use of external control data and the prospectively defined statistical analysis plan (SAP) in support of the planned BLA submission for AMT-130. The FDA continued to support its prior agreement that the composite Unified Huntington's Disease Rating Scale (cUHDRS) may serve as an acceptable registrational, intermediate clinical endpoint for accelerated approval. The FDA agreed that the primary efficacy analysis for the BLA will evaluate the 3-year change in cUHDRS in high-dose AMT-130 patients compared to a propensity score-adjusted external control arm. The Company plans to use propensity score-weighted external control derived from the ENROLL-HD dataset for the primary analysis and to submit certain sensitivity analyses, including one using a propensity score-matched external control, as additional support. The FDA also agreed that ENROLL-HD – a large, prospective, longitudinal, natural history study of patients with Huntington's disease – may be acceptable as the external control dataset for the primary analysis of the trial data along with additional sensitivity analyses using the TRACK-HD/TRACK-ON and PREDICT-HD datasets. To date, approximately 33,000 patients have enrolled in ENROLL-HD. Compared to previously used natural history studies like TRACK-HD and PREDICT-HD, ENROLL-HD offers a substantially larger sample size, lower attrition rates, and longer average patient follow-up. The Company expects the larger sample size to reduce variability in the external control data and enhance the robustness of the SAP. The Company plans to submit an updated SAP consistent with discussions from the recently held Type B meeting to the FDA in the second quarter of 2025. Chemistry, Manufacturing and Controls (CMC) In the first quarter of 2025, the Company held a Type B meeting with the FDA to discuss CMC requirements in support of the planned BLA submission for AMT-130. The FDA agreed that validation of the AMT-130 manufacturing process should be possible using experience and prior knowledge from the etranacogene dezaparvovec-drlb (HEMGENIX®) process, complemented with additional full-scale AMT-130 GMP batches and a single Process Performance Qualification (PPQ) batch. The FDA also agreed with the Company's proposed drug product release testing plan, including the proposed potency assay, pending completion of qualification and specification setting activities. Next Steps for the Planned BLA Submission Based on these recent Type B meetings with the FDA, the Company continues to prepare for a BLA submission for AMT-130 in the treatment of Huntington's disease. Certain key next steps and expected timing include: Investor Conference Call and Webcast Information uniQure management will host an investor conference call and webcast today, Monday, June 2, 2025 at 8:30 a.m. ET. The event will be webcast under the Events & Presentations section of uniQure's website at and following the event a replay will be archived for 90 days. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone. If you are joining the conference call, please dial in 15 minutes before the start time. About the Phase I/II Clinical Program of AMT-130 uniQure is conducting two multi-center, dose-escalating, Phase I/II clinical studies to explore the safety, tolerability, and exploratory efficacy signals of AMT-130 for the treatment of Huntington's disease. In the U.S. study, a total of 26 patients with early manifest Huntington's disease were randomized to treatment (n=6 low dose; n=10 high dose) or an imitation (sham) surgical procedure (n=10). Treated patients received a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). The study consists of a blinded 12-month core study period followed by unblinded long-term follow-up of treated patients for five years. An additional four control patients crossed over to treatment. The European open-label Phase Ib/II study of AMT-130 enrolled 13 patients with early manifest Huntington's disease (n=6 low dose; n=7 high dose). A third cohort enrolled an additional 12 patients across sites in the U.S. and EU. This cohort was randomized to explore both doses of AMT-130 in combination with immunosuppression, using the current, established stereotactic administration procedure. Additional details are available on (NCT0543017, NCT04120493) AMT-130 has been granted the FDA's Regenerative Medicine Advance Therapy (RMAT) designation and Breakthrough Therapy designation, the first therapy for Huntington's disease to receive an RMAT designation. About Huntington's Disease Huntington's disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. According to 2021 study in Neuroepidemiology, approximately 70,000 people have been diagnosed with Huntington's disease in the U.S. and Europe, with hundreds of thousands of others at risk of inheriting the disease. Despite the clear etiology of Huntington's disease, there are currently no approved therapies to delay the onset or to slow the disease's progression. About uniQure uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The approvals of uniQure's gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. uniQure is now advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases. uniQure Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as 'anticipate,' 'believe,' 'could,' 'establish,' 'estimate,' 'expect,' 'goal,' 'intend,' 'look forward to', 'may,' 'plan,' 'potential,' 'predict,' 'project,' 'seek,' 'should,' 'will,' 'would' and similar expressions and the negatives of those terms. Forward-looking statements are based on management's beliefs and assumptions and on information available to management as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning: the availability of accelerated approval pathways and the need for additional pre-approval studies for AMT-130; the Company's anticipated timing of the BLA submission; the Company's plans to submit a revised SAP and CMC information to the FDA; the Company's ability to deliver a potentially life-changing therapy to people living with Huntington's disease and related timeline for doing so; the potential clinical and functional effects of AMT-130; the Company's plans to continue clinical development of AMT-130; the Company's plans to share clinical data of AMT-130 in the third quarter of 2025; and the utility of the ENROLL-HD patient dataset with respect to Phase I/II study. The Company may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements. Furthermore, the Company's actual results could differ materially from the plans, intentions and expectations anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks related to the Company's Phase I/ll clinical trials of AMT-130, including the risk that interim data from the trials may not be predictive of later data readouts that will serve as a basis for further regulatory interactions, may not support BLA submissions or accelerated approvals, may not be satisfactory to the FDA and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; risks related to the Company's current and future interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials, its BLA submission plans and pathways to regulatory approval; risks related to the Company's ability to pursue business development efforts with respect to AMT-130; risks related to the Company's use of propensity-weighted external controls in connection with its statistical analysis of clinical outcomes to date; uncertainties as to the FDA's and other regulatory authorities' interpretation of the data from the Company's Phase I/ll clinical trials of AMT-130 and acceptance of the Company's clinical programs and the regulatory approval process;later developments with the FDA and other regulators that could be inconsistent with the feedback received to date; and whether regulatory authorities will accept the Company's approach as a basis for accelerated approval; risks related to the Company's use of nominal p values as a basis for its statistical analyses; whether the measurements that the Company is evaluating continue to be viewed as robust and sensitive measurements of disease progression; whether RMAT designation or any accelerated pathway, will lead to regulatory approval; the Company's ability to continue to build and maintain the infrastructure and personnel needed to achieve its goals; the Company's effectiveness in managing current and future clinical trials and regulatory processes; the Company's ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the continued development and acceptance of gene therapies; the Company's ability to obtain, maintain and protect its intellectual property; and the Company's ability to fund its operations and to raise additional capital as needed and on acceptable terms. These risks and uncertainties are more fully described under the heading 'Risk Factors' in the Company's periodic filings with the U.S. Securities & Exchange Commission (SEC), including its Annual Report on Form 10-K filed with the SEC on February 27, 2025, its Quarterly Reports on Form 10-Q filed May 9, 2025, and in other filings that the Company makes with the SEC from time to time. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements and, except as required by law, the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.
Globe and Mail
28-05-2025
- Business
- Globe and Mail
Genflow Biosciences and CER Sign Master Service Agreement to Strengthen Long-Term R&D Collaboration
LONDON, UK / ACCESS Newswire / May 28, 2025 / Genflow Biosciences Plc (LSE:GENF)(OTCQB:GENFF) ("Genflow" or "the Company"), the only publicly listed longevity company in Europe, is pleased to announce the signing of a Master Service Agreement (MSA) with CER Groupe (CER), a long-standing partner of the Company. CER is a private Belgian research center offering integrated bioproduction and pre-clinical services within a regulated ISO and GxP-compliant environment. The MSA formalizes the working relationship between Genflow and CER, providing a robust R&D framework to accelerate Genflow's pre-IND gene therapy programs. Under the terms of the MSA, Genflow and CER will: Develop, produce and characterize Genflow's gene therapy candidates; Implement a collaborative project management system to drive efficient execution and timely deliverables supporting Genflow's goals with standardized templates for Task Orders and technical services; and Combine scientific expertise derisks, innovate and ensure relevant scientific outcomes. Said Dr. Eric Leire, CEO of Genflow: " Our partnership with CER has been instrumental in supporting our R&D efforts to date. Formalizing this collaboration through the MSA will enable greater operational alignment and strategic agility as we continue to accelerate our pipeline." Contacts Corporate Brokers Capital Plus Partners Ltd Jon Critchley, +44 0203 821 6168 About Genflow Biosciences Founded in 2020, Genflow Biosciences Plc. (LSE:GENF)(OTCQB:GENFF), a biotechnology company headquartered in the UK with R&D facilities in Belgium, is pioneering gene therapies to decelerate the aging process, with the goal of promoting longer and healthier lives while mitigating the financial, emotional, and social impacts of a fast-growing aging global population. Genflow's lead compound, GF-1002, works through the delivery of a centenarian variant of the SIRT6 gene which has yielded promising preclinical results. Genflow's 12-month proof-of-concept clinical trial evaluating their SIRT6-centenarian gene therapy in aged dogs began in March 2025. Other programs planned for 2025, include a clinical trial that will explore the potential benefits of GF-1002 in treating MASH (Metabolic Dysfunction-Associated Steatohepatitis), the most prevalent chronic liver disease for which there is no effective treatments. Please visit and follow the Company on LinkedIn and X. About CER Groupe CER Groupe is a GLP-certified private Belgian research center supporting European life sciences innovation for more than 45years. CER's 200 colleagues gather cutting-edge expertise ensuring high quality integrated bioproduction and non-clinical services. CER supports companies in progressing their development from idea generation up to IND-enabling studies. CER Groupe favors collaborative and agile approaches accelerating companies' access to clinical stage development. Visit to learn what CER's partners say and follow CER Groupe on LinkedIn to stay up-to-date on CER's latest developments. This information is provided by Reach, the non-regulatory press release distribution service of RNS, part of the London Stock Exchange. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@ or visit View the original press release on ACCESS Newswire
Yahoo
22-05-2025
- Health
- Yahoo
ZEVASKYN Approval Positions Abeona as Emerging Player in Dystrophic Epidermolysis Bullosa Market
Approximately a year after the FDA rejected its previous approval submission, Abeona announced that the agency had approved ZEVASKYN, its autologous cell-based gene therapy for both adult and pediatric patients with RDEB. This approval marks Abeona's entry into the commercial gene therapy market for dystrophic epidermolysis bullosa. LAS VEGAS, May 22, 2025 /PRNewswire/ -- Dystrophic epidermolysis bullosa (DEB) is one of the main subtypes of epidermolysis bullosa, a group of genetic skin disorders characterized by varying degrees of fragility in the skin and mucous membranes. This fragility occurs due to the absence, deficiency, or malfunction of proteins critical to maintaining skin structure. In the 7MM, approximately 6,500 diagnosed prevalent cases of DEB were recorded in 2023, with the United States accounting for about 50% of these cases. Currently, there is no cure for DEB. Management is primarily supportive, focusing on wound care, controlling pain and itching, preventing infections, providing nutritional assistance, and addressing complications that may arise. Learn more about the DEB therapeutic market @ Dystrophic Epidermolysis Bullosa Treatment Market As of now, only four therapies, namely FILSUVEZ (Chiesi Farmaceutici), VYJUVEK (Krystal Biotech), JACE (Japan Tissue Engineering), and recently approved ZEVASKYN (Abeona Therapeutics), have been approved for treating DEB, leaving a significant unmet need for effective and targeted dystrophic epidermolysis bullosa treatments. FILSUVEZ (oleogel-S10), developed by Chiesi Farmaceutici, is a plant-based medicinal product derived from birch bark triterpenes. It was granted EU-wide marketing authorization on June 21, 2022. In December 2023, the FDA approved FILSUVEZ topical gel for the treatment of partial-thickness wounds in patients aged six months and older with Junctional or Dystrophic Epidermolysis Bullosa (JEB and DEB). VYJUVEK (beremagene geperpavec), developed by Krystal Biotech, is a topical, repeat-dose gene therapy intended to deliver two functional copies of the COL7A1 gene directly to DEB wounds. It received FDA approval in May 2023 for use in DEB patients aged six months and older. The Marketing Authorization Application (MAA) for VYJUVEK has been validated by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) and is currently under review. JACE, developed by Japan Tissue Engineering, is a combination product featuring a human autologous epidermal cell sheet created using Green's technique. In December 2018, Japan's Ministry of Health, Labour and Welfare (MHLW) granted additional approval for JACE, expanding its use to include both JEB and DEB. Dive deep into the treatment for dystrophic epidermolysis bullosa @ Dystrophic Epidermolysis Bullosa Drugs Market Recently, in April 2025, Abeona Therapeutics Inc. announced that the FDA had approved ZEVASKYN (pz-cel), marking it as the first and only autologous, gene-modified cell therapy for treating wounds in both adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB)—a severe, inherited skin disorder with no known cure. ZEVASKYN is currently the only FDA-approved therapy for RDEB wounds that works with just a single application. The approval was granted based on results from the Phase III VIITAL study (NCT04227106), a multi-center, randomized, intrapatient-controlled trial. The study successfully met its two main goals, showing significant wound healing (50% or more from baseline) and a reduction in pain, as measured by the Wong-Baker FACES scale, at six months post-treatment. ZEVASKYN is anticipated to become available in Q3 2025 through ZEVASKYN Qualified Treatment Centers (QTCs)—specialized facilities across the U.S. with expertise in treating epidermolysis bullosa and administering cell and gene therapies, ensuring broad patient access. To know more about the treatment for dystrophic epidermolysis bullosa in children, visit @ Dystrophic Epidermolysis Bullosa Drugs Companies across the globe are diligently working toward the development of novel DEB treatment therapies with considerable amount of success over the years. Some of the drugs in the pipeline include Dabocemagene autoficel (Castle Creek Biosciences), ABCB5+ MSCs (RHEACELL), and ALLO-ASC-SHEET (Anterogen), among others. Discover which therapies are expected to grab major DEB therapeutics market share @ Dystrophic Epidermolysis Bullosa Drugs Market D-Fi, also referred to as FCX-007 (dabocemagene autoficel), is an investigational autologous gene therapy designed to treat dystrophic epidermolysis bullosa (DEB). It involves using the patient's own dermal fibroblasts, which are genetically engineered using a self-inactivating (SIN) lentiviral vector carrying the COL7A1 gene to produce type VII collagen (COL7). The therapy is delivered via intradermal injection directly into the superficial papillary dermis of persistent wounds, where COL7 helps form anchoring fibrils essential for skin integrity. ABCB5-positive mesenchymal stem cells (ABCB5+ MSCs) represent a novel and promising therapeutic option for DEB. These cells possess anti-inflammatory properties through interaction with immune cells, triggering their reprogramming. Administered systemically via infusion, ABCB5+ MSCs migrate to wound sites, reduce inflammation, and aid in tissue repair by releasing and depositing type VII collagen. This therapy targets both external and internal wounds and has received Orphan Drug Designation (ODD) from both the FDA and EMA for the treatment of RDEB. RHEACELL is currently running a Phase III clinical trial to evaluate the effectiveness of ABCB5+ MSCs in patients with RDEB. Discover more about DEB drugs in development @ Dystrophic Epidermolysis Bullosa Clinical Trials The anticipated launch of these emerging dystrophic epidermolysis bullosa treatments are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the dystrophic epidermolysis bullosa market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the dystrophic epidermolysis bullosa market size in the 7MM is expected to grow from USD 550 million in 2023 at a significant CAGR by 2034. According to DelveInsight's analysis, the growth of the dystrophic epidermolysis bullosa market is expected to be mainly driven by increasing prevalence, patient awareness, and robust clinical pipeline during the forecast period (2025–2034). DelveInsight's latest published market report titled Dystrophic Epidermolysis Bullosa Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the dystrophic epidermolysis bullosa country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The dystrophic epidermolysis bullosa market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Diagnosed Prevalent Cases of DEB Age-Specific Cases of DEB Type-specific Cases of DEB Severity-specific Cases of DEB The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM dystrophic epidermolysis bullosa market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this dystrophic epidermolysis bullosa market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the dystrophic epidermolysis bullosa market. Also, stay abreast of the mitigating factors to improve your market position in the dystrophic epidermolysis bullosa therapeutic space. Related Reports Recessive Dystrophic Epidermolysis Bullosa Pipeline Recessive Dystrophic Epidermolysis Bullosa Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RDEB companies, including Abeona Therapeutics, Inc., Castle Creek Biosciences, LLC., RHEACELL GmbH & Co. KG, among others. Dystrophic Epidermolysis Bullosa Pipeline Dystrophic Epidermolysis Bullosa Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key DEB companies, including Phoenix Tissue Repair, Castle Creek Biosciences, Phoenicis Therapeutics, Aegle Therapeutics, RHEACELL GmbH & Co. KG, Anterogen Co., Ltd., Abeona Therapeutics, Inc., InMed Pharmaceuticals, BridgeBio Inc., Eloxx Pharmaceuticals, Relief Therapeutics, Quoin Pharmaceuticals, among others. Epidermolysis Bullosa Market Epidermolysis Bullosa Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key epidermolysis bullosa companies including Abeona Therapeutics, Castle Creek Biosciences, RHEACELL, Ishin Pharma, Holostem Terapie Avanzate, BridgeBio (Phoenix Tissue Repair), InMed Pharmaceuticals, Shionogi, Anterogen, among others. Epidermolysis Bullosa Pipeline Epidermolysis Bullosa Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key epidermolysis bullosa companies, including Abeona Therapeutics, InMed Pharmaceuticals, BioMendics, RHEACELL, Castle Creek Biosciences, BPGbio, Eloxx Pharmaceuticals, Phoenicis Therapeutics, Aegle Therapeutics, Amryt Pharma, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact Us Shruti Thakur info@ +14699457679 Logo: View original content: SOURCE DelveInsight Business Research, LLP Sign in to access your portfolio
Yahoo
22-05-2025
- Health
- Yahoo
ZEVASKYN Approval Positions Abeona as Emerging Player in Dystrophic Epidermolysis Bullosa Market
Approximately a year after the FDA rejected its previous approval submission, Abeona announced that the agency had approved ZEVASKYN, its autologous cell-based gene therapy for both adult and pediatric patients with RDEB. This approval marks Abeona's entry into the commercial gene therapy market for dystrophic epidermolysis bullosa. LAS VEGAS, May 22, 2025 /PRNewswire/ -- Dystrophic epidermolysis bullosa (DEB) is one of the main subtypes of epidermolysis bullosa, a group of genetic skin disorders characterized by varying degrees of fragility in the skin and mucous membranes. This fragility occurs due to the absence, deficiency, or malfunction of proteins critical to maintaining skin structure. In the 7MM, approximately 6,500 diagnosed prevalent cases of DEB were recorded in 2023, with the United States accounting for about 50% of these cases. Currently, there is no cure for DEB. Management is primarily supportive, focusing on wound care, controlling pain and itching, preventing infections, providing nutritional assistance, and addressing complications that may arise. Learn more about the DEB therapeutic market @ Dystrophic Epidermolysis Bullosa Treatment Market As of now, only four therapies, namely FILSUVEZ (Chiesi Farmaceutici), VYJUVEK (Krystal Biotech), JACE (Japan Tissue Engineering), and recently approved ZEVASKYN (Abeona Therapeutics), have been approved for treating DEB, leaving a significant unmet need for effective and targeted dystrophic epidermolysis bullosa treatments. FILSUVEZ (oleogel-S10), developed by Chiesi Farmaceutici, is a plant-based medicinal product derived from birch bark triterpenes. It was granted EU-wide marketing authorization on June 21, 2022. In December 2023, the FDA approved FILSUVEZ topical gel for the treatment of partial-thickness wounds in patients aged six months and older with Junctional or Dystrophic Epidermolysis Bullosa (JEB and DEB). VYJUVEK (beremagene geperpavec), developed by Krystal Biotech, is a topical, repeat-dose gene therapy intended to deliver two functional copies of the COL7A1 gene directly to DEB wounds. It received FDA approval in May 2023 for use in DEB patients aged six months and older. The Marketing Authorization Application (MAA) for VYJUVEK has been validated by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) and is currently under review. JACE, developed by Japan Tissue Engineering, is a combination product featuring a human autologous epidermal cell sheet created using Green's technique. In December 2018, Japan's Ministry of Health, Labour and Welfare (MHLW) granted additional approval for JACE, expanding its use to include both JEB and DEB. Dive deep into the treatment for dystrophic epidermolysis bullosa @ Dystrophic Epidermolysis Bullosa Drugs Market Recently, in April 2025, Abeona Therapeutics Inc. announced that the FDA had approved ZEVASKYN (pz-cel), marking it as the first and only autologous, gene-modified cell therapy for treating wounds in both adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB)—a severe, inherited skin disorder with no known cure. ZEVASKYN is currently the only FDA-approved therapy for RDEB wounds that works with just a single application. The approval was granted based on results from the Phase III VIITAL study (NCT04227106), a multi-center, randomized, intrapatient-controlled trial. The study successfully met its two main goals, showing significant wound healing (50% or more from baseline) and a reduction in pain, as measured by the Wong-Baker FACES scale, at six months post-treatment. ZEVASKYN is anticipated to become available in Q3 2025 through ZEVASKYN Qualified Treatment Centers (QTCs)—specialized facilities across the U.S. with expertise in treating epidermolysis bullosa and administering cell and gene therapies, ensuring broad patient access. To know more about the treatment for dystrophic epidermolysis bullosa in children, visit @ Dystrophic Epidermolysis Bullosa Drugs Companies across the globe are diligently working toward the development of novel DEB treatment therapies with considerable amount of success over the years. Some of the drugs in the pipeline include Dabocemagene autoficel (Castle Creek Biosciences), ABCB5+ MSCs (RHEACELL), and ALLO-ASC-SHEET (Anterogen), among others. Discover which therapies are expected to grab major DEB therapeutics market share @ Dystrophic Epidermolysis Bullosa Drugs Market D-Fi, also referred to as FCX-007 (dabocemagene autoficel), is an investigational autologous gene therapy designed to treat dystrophic epidermolysis bullosa (DEB). It involves using the patient's own dermal fibroblasts, which are genetically engineered using a self-inactivating (SIN) lentiviral vector carrying the COL7A1 gene to produce type VII collagen (COL7). The therapy is delivered via intradermal injection directly into the superficial papillary dermis of persistent wounds, where COL7 helps form anchoring fibrils essential for skin integrity. ABCB5-positive mesenchymal stem cells (ABCB5+ MSCs) represent a novel and promising therapeutic option for DEB. These cells possess anti-inflammatory properties through interaction with immune cells, triggering their reprogramming. Administered systemically via infusion, ABCB5+ MSCs migrate to wound sites, reduce inflammation, and aid in tissue repair by releasing and depositing type VII collagen. This therapy targets both external and internal wounds and has received Orphan Drug Designation (ODD) from both the FDA and EMA for the treatment of RDEB. RHEACELL is currently running a Phase III clinical trial to evaluate the effectiveness of ABCB5+ MSCs in patients with RDEB. Discover more about DEB drugs in development @ Dystrophic Epidermolysis Bullosa Clinical Trials The anticipated launch of these emerging dystrophic epidermolysis bullosa treatments are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the dystrophic epidermolysis bullosa market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the dystrophic epidermolysis bullosa market size in the 7MM is expected to grow from USD 550 million in 2023 at a significant CAGR by 2034. According to DelveInsight's analysis, the growth of the dystrophic epidermolysis bullosa market is expected to be mainly driven by increasing prevalence, patient awareness, and robust clinical pipeline during the forecast period (2025–2034). DelveInsight's latest published market report titled Dystrophic Epidermolysis Bullosa Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the dystrophic epidermolysis bullosa country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The dystrophic epidermolysis bullosa market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Diagnosed Prevalent Cases of DEB Age-Specific Cases of DEB Type-specific Cases of DEB Severity-specific Cases of DEB The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM dystrophic epidermolysis bullosa market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this dystrophic epidermolysis bullosa market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the dystrophic epidermolysis bullosa market. Also, stay abreast of the mitigating factors to improve your market position in the dystrophic epidermolysis bullosa therapeutic space. Related Reports Recessive Dystrophic Epidermolysis Bullosa Pipeline Recessive Dystrophic Epidermolysis Bullosa Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RDEB companies, including Abeona Therapeutics, Inc., Castle Creek Biosciences, LLC., RHEACELL GmbH & Co. KG, among others. Dystrophic Epidermolysis Bullosa Pipeline Dystrophic Epidermolysis Bullosa Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key DEB companies, including Phoenix Tissue Repair, Castle Creek Biosciences, Phoenicis Therapeutics, Aegle Therapeutics, RHEACELL GmbH & Co. KG, Anterogen Co., Ltd., Abeona Therapeutics, Inc., InMed Pharmaceuticals, BridgeBio Inc., Eloxx Pharmaceuticals, Relief Therapeutics, Quoin Pharmaceuticals, among others. Epidermolysis Bullosa Market Epidermolysis Bullosa Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key epidermolysis bullosa companies including Abeona Therapeutics, Castle Creek Biosciences, RHEACELL, Ishin Pharma, Holostem Terapie Avanzate, BridgeBio (Phoenix Tissue Repair), InMed Pharmaceuticals, Shionogi, Anterogen, among others. Epidermolysis Bullosa Pipeline Epidermolysis Bullosa Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key epidermolysis bullosa companies, including Abeona Therapeutics, InMed Pharmaceuticals, BioMendics, RHEACELL, Castle Creek Biosciences, BPGbio, Eloxx Pharmaceuticals, Phoenicis Therapeutics, Aegle Therapeutics, Amryt Pharma, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. 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