SOD1

LEXINGTON, MA, USA and AMSTERDAM, The Netherlands I October 15, 2024 I uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the first patient has been dosed in the Phase I/II clinical trial of AMT-162 for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1), a rare, inherited and progressive motor neuron disease. EPISOD1 is a Phase I/II multi-center, open-label trial being conducted in the United States with three dose-escalating cohorts assessing the safety, tolerability and exploratory signs of efficacy of AMT-162 in individuals with SOD1-ALS. “We are pleased to announce the first patient dosing of AMT-162, our investigational gene therapy for the treatment of SOD1-ALS, a debilitating, degenerative and fatal disease,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure . “The start of this trial marks the advancement of our third gene therapy program into the clinic with this trial design, continuing our goal of rapidly generating proof-of-concept data using well-established biomarkers in order to bring treatments to patients as quickly as possible. We believe our novel AAV-based gene therapy candidate can deliver on the convenience of one-time dosing with the potential for a differentiated efficacy profile that is needed for such a devasting disease.” AMT-162 is an investigational AAVrh10-based gene therapy that expresses a miRNA designed to knock down the expression of the mutated SOD1 protein. Patients with SOD1-ALS express a misfolded SOD1 protein. This misfolded protein is toxic to motor neurons causing degeneration which, over time leads to muscle weakness, loss of function and eventually death. AMT-162 may offer a novel one-time, intrathecally administered approach to slowing or halting the progression of SOD1-ALS. AMT-162 has been granted both Orphan Drug status and Fast Track designation by the U.S. Food and Drug Administration. The EPISOD1 Phase I/II clinical trial of AMT-162 will be conducted in the United States. The multicenter, open-label trial consists of three cohorts with up to four patients each receiving a short course of immunosuppression prior to and after an intrathecal infusion of AMT-162. The trial will explore the safety and tolerability of AMT-162 and will assess exploratory signs of efficacy by measuring neurofilament light chain, a biomarker of neuronal damage, and SOD1 protein. There are currently four active sites in the U.S. with plans to activate seven additional sites by the first quarter of 2025. Additional details are available on www.clinicaltrials.gov (NCT06100276). About Amyotrophic Lateral Sclerosis (ALS) caused by mutations in Superoxide Dismutase 1 (SOD1) SOD1-ALS is a rare, progressive and fatal neurodegenerative disease that results in the loss of motor neurons in the brain and spinal cord. As ALS progresses, individuals experience muscle weakness and atrophy causing them to lose the ability to move their arms and legs, speak, swallow and eventually breathe, causing respiratory failure. Based on a 2021 study in Neuroepidemiology, there are an estimated ~170,000 individuals with ALS globally with SOD1 mutations representing 2% within that population. According to the National Institute of Neurological Disorders and Stroke, the average life expectancy for those diagnosed with ALS is three to five years from onset of symptoms. 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. www.uniQure.com SOURCE: uniQure

LEXINGTON, Mass. and AMSTERDAM, Oct. 15, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the first patient has been dosed in the Phase I/II clinical trial of AMT-162 for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1), a rare, inherited and progressive motor neuron disease. EPISOD1 is a Phase I/II multi-center, open-label trial being conducted in the United States with three dose-escalating cohorts assessing the safety, tolerability and exploratory signs of efficacy of AMT-162 in individuals with SOD1-ALS. “We are pleased to announce the first patient dosing of AMT-162, our investigational gene therapy for the treatment of SOD1-ALS, a debilitating, degenerative and fatal disease,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “The start of this trial marks the advancement of our third gene therapy program into the clinic with this trial design, continuing our goal of rapidly generating proof-of-concept data using well-established biomarkers in order to bring treatments to patients as quickly as possible. We believe our novel AAV-based gene therapy candidate can deliver on the convenience of one-time dosing with the potential for a differentiated efficacy profile that is needed for such a devasting disease.” AMT-162 is an investigational AAVrh10-based gene therapy that expresses a miRNA designed to knock down the expression of the mutated SOD1 protein. Patients with SOD1-ALS express a misfolded SOD1 protein. This misfolded protein is toxic to motor neurons causing degeneration which, over time leads to muscle weakness, loss of function and eventually death. AMT-162 may offer a novel one-time, intrathecally administered approach to slowing or halting the progression of SOD1-ALS. AMT-162 has been granted both Orphan Drug status and Fast Track designation by the U.S. Food and Drug Administration. The EPISOD1 Phase I/II clinical trial of AMT-162 will be conducted in the United States. The multicenter, open-label trial consists of three cohorts with up to four patients each receiving a short course of immunosuppression prior to and after an intrathecal infusion of AMT-162. The trial will explore the safety and tolerability of AMT-162 and will assess exploratory signs of efficacy by measuring neurofilament light chain, a biomarker of neuronal damage, and SOD1 protein. There are currently four active sites in the U.S. with plans to activate seven additional sites by the first quarter of 2025. Additional details are available on www.clinicaltrials.gov (NCT06100276). About Amyotrophic Lateral Sclerosis (ALS) caused by mutations in Superoxide Dismutase 1 (SOD1)SOD1-ALS is a rare, progressive and fatal neurodegenerative disease that results in the loss of motor neurons in the brain and spinal cord. As ALS progresses, individuals experience muscle weakness and atrophy causing them to lose the ability to move their arms and legs, speak, swallow and eventually breathe, causing respiratory failure. Based on a 2021 study in Neuroepidemiology, there are an estimated ~170,000 individuals with ALS globally with SOD1 mutations representing 2% within that population. According to the National Institute of Neurological Disorders and Stroke, the average life expectancy for those diagnosed with ALS is three to five years from onset of symptoms. 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. www.uniQure.com uniQure Forward-Looking Statements This press release contains forward-looking statements. 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. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements regarding the availability of proof-of-concept data in the Company’s open-label U.S. Phase I/II clinical trial for SOD1-ALS; the effectiveness of the EPISOD1 trial design; and the potential efficacy profile of AMT-162 through one-time administration with the ability to slow or halt the progression of SOD1-ALS. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, without limitation, risks associated with the clinical results and the development and timing of the Company’s programs; the Company’s interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to approval; the Company’s ability to continue to build and maintain the company infrastructure and personnel needed to achieve its goals; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; 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. 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 February 28, 2024, its Quarterly Reports on Form 10-Q filed May 7, 2024 and August 1, 2024, 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 the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future. uniQure Contacts: FOR INVESTORS: FOR MEDIA: Chiara RussoTom MaloneDirect: 617-306-9137 Direct: 339-970-7558Mobile: 617-306-9137Mobile: 339-223-8541c.russo@uniQure.comt.malone@uniQure.com This press release was published by a CLEAR® Verified individual.

REDDING, Calif., Oct. 2, 2024 /PRNewswire/ -- According to a new market research report titled, ' Regenerative Medicine Market Size, Share, Forecast, & Trends Analysis by Product (Gene Therapy, Stem Cell, Tissue Engineering), Application (Musculoskeletal, Cancer, Cardiovascular, Immunology, Dermatology, Ophthalmology), End User—Global Forecast to 2031. The regenerative medicine market is projected to reach $37.27 billion by 2031, at a CAGR of 21.5% from 2024 to 2031. Download FREE PDF Brochure Of Regenerative Medicine Market Size, Share, Forecast, & Trends Analysis - The growth of the regenerative medicine market is driven by the growing number of new regenerative medicine approvals, consistent advancements in the regenerative medicine space, the rising incidence of chronic diseases, the availability of funding for regenerative medicine development, and the increasing utilization of regenerative medicines in diverse therapeutic areas. However, high treatment costs and ethical issues related to stem cells restrain the growth of this market. Furthermore, the rising demand for personalized medicines, increasing organ transplant procedures, and the strong product pipeline for regenerative medicines are expected to generate market growth opportunities. However, unfavorable reimbursement policies, complexities in the manufacture of regenerative medicines, and the lack of standardized frameworks for the safety & efficacy of regenerative medicines are major challenges in the regenerative medicine market. Increasing Funding for Regenerative Medicine Development Expected to Drive Market Growth During the Forecast Period The field of regenerative medicine has gained traction in recent years owing to increasing applications in oncology, cardiology, orthopedics, and other medical domains. The key players operating in this market are raising funds for regenerative therapy development. Additionally, governments around the globe are offering grants and funding to advance regenerative medicine research. Some of the recent developments in this space are as follows: In April 2024, Century Therapeutics, Inc. (U.S.) acquired Clade Therapeutics (U.S.) and raised $60 million for the testing of natural killer cell therapy in certain blood cancers. Bain Capital Life Sciences (U.S.) provided the funding. With this acquisition, the company is likely to benefit from three preclinical programs and technology, allowing it to better engineer stem cell-derived medicines. In March 2024, Pandorum Technologies Pvt. Ltd. (India), a tissue engineering and regenerative medicine company, raised $11 million in a pre-Series B funding round. The funding was raised for the clinical phase study of 'Liquid Cornea,' a treatment for corneal blindness and advancing the company's Tunable technology platform. In November 2023, Siren Biotechnology (U.S.) received a $4 million grant from the California Institute for Regenerative Medicine (CIRM). The grant was awarded to aid the development of novel AAV immunogene therapies for cancer. Regenerative Medicine Market Analysis: Key Findings By Product: In 2024, the cell therapy segment is anticipated to dominate the regenerative medicine market with a share of 51.2%. The increasing public and private funding for stem cell therapy development, government efforts to streamline approval procedures, and collaborations among key players to advance stem cell therapy are factors contributing to the segment's dominant position in the regenerative medicine market. By Application: The oncology segment is slated to register the highest CAGR of 26.5% during the forecast period. The high global burden of cancer, increasing research on regenerative medicine for cancer treatment, increasing approvals for regenerative medicine products for oncology applications, the growing number of oncology clinical trials, and increasing public & private funding dedicated to advancing oncology treatments are factors expected to support the segment's high growth. In March 2023, researchers at the University of California received a grant of $5.7 million from the California Institute of Regenerative Medicine to support advancements in stem cell therapy for cancer treatment. By End user: In 2024, the hospitals & clinics segment is expected to account for the larger share of the regenerative medicine market. The segment is also slated to register the higher CAGR of 22% during the forecast period. Explore the Key Market Segments Driving Growth (Download Free Sample) - Geographic Analysis: Based on geography, the regenerative medicine market is segmented into North America, Europe, Asia-Pacific, Latin America, and the Middle East & Africa. In 2024, North America is expected to account for the largest share of 41.8% of the regenerative medicine market. This market is projected to reach $14.35 billion by 2031. North America's dominant position in the regenerative medicine market is attributed to the rising prevalence of chronic conditions such as cancer and cardiovascular diseases, increasing funding for the development of regenerative medicine, the presence of key market players in the region, and the rising number of clinical trials in the region. In 2024, the U.S. is expected to account for the major share of 89.0% of the regenerative medicine market in North America. The country's large market share is mainly attributed to the presence of key players involved in the development of regenerative medicine and rising investments from public and private players in the development of novel therapies. The number of clinical trials for regenerative medicine is also increasing in the country, further driving market growth. The number of key players involved in the development of regenerative medicine is also gradually increasing in the U.S. For instance, the number of developers reached 625 in 2022, a 2% increase over 2021 (source: Alliance for Regenerative Medicine). Asia-Pacific: The Fastest-growing Regional Market Asia-Pacific is projected to register the highest CAGR of 25.0% during the forecast period. In 2024, Japan is expected to account for the largest share of the regenerative medicine market in Asia-Pacific. The Asia-Pacific region is becoming an attractive market for healthcare product manufacturers due to ongoing improvements in healthcare infrastructure, the increasing number of hospitals, and growing government investments in the healthcare sector. Rapid economic growth in many countries within the Asia-Pacific region has increased government focus on healthcare, with rising investments aimed at enhancing access to healthcare facilities and building better healthcare infrastructure. The Asia-Pacific region is one of the fastest-growing markets for regenerative medicine, driven by rising healthcare expenditures, a growing geriatric population, and an increasing burden of target diseases. Additionally, conferences promoting the use of regenerative medicine and active research initiatives are expected to further boost awareness and adoption of these therapies in the region. Have specific research needs? Request a customized research report - Germany Continues to Dominate the Regenerative Medicine Market in Europe In 2024, Germany is expected to account for the largest share of 26.6% of the regenerative medicine market in Europe. Germany is the largest healthcare market in Europe by market volume, healthcare providers, number of patients, and medical technology manufacturers (source: Germany Trade & Invest). In 2022, the gross value added to the healthcare industry was $463.2 billion (€439.6 billion) (source: International Trade Administration). The high number of fatalities in Germany is marked by the rising incidence of chronic diseases such as ischemic heart disease, stroke, Chronic Obstructive Pulmonary Disease (COPD), and cancer. Cardiovascular diseases account for 37% of all deaths in Germany. According to Eurostat, ischemic heart disease is responsible for over one in ten deaths in the country. Furthermore, lung cancer is the most common cancer, accounting for 20% of all cancer-related deaths. Thus, the rising prevalence of chronic diseases, high healthcare expenditure, and well-developed healthcare infrastructure are the factors supporting Germany's large market share. Regenerative Medicine Market: Competition Analysis This report offers a competitive analysis based on an extensive assessment of the leading players' product portfolios, geographic presence, and key growth strategies adopted over the past 3–4 years. Major companies in the regenerative medicine market have implemented various strategies to expand their product offerings and global footprints and augment their market shares. The key strategies followed by leading companies in the regenerative medicine market include product launches, expansions, mergers & acquisitions, agreements, collaborations, and partnerships. The key players operating in the regenerative medicine market include Novartis AG (Switzerland), Biogen Inc. (U.S.), Kite Pharma, Inc. (U.S.), Spark Therapeutics, Inc. (U.S.), Integra LifeSciences Corporation (U.S.), Sarepta Therapeutics, Inc. (U.S.), Takeda Pharmaceutical Company Limited (Japan), Amgen Inc. (U.S.), CORESTEMCHEMON Inc. (South Korea), Smith & Nephew plc (England), Vertex Pharmaceuticals Incorporated (U.S.), CSL Behring, LLC (U.S.), Janssen Global Services, LLC (U.S.), Medtronic plc (Ireland), AbbVie Inc. (U.S.), Bristol-Myers Squibb Company (U.S.), Ferring Pharmaceuticals A/S (Sweden), Pfizer Inc. (U.S.), bluebird bio Inc. (U.S.), and Vericel Corporation (U.S.). Regenerative Medicine Industry Overview: Latest Developments from Key Industry Players In January 2024, Smith & Nephew plc (U.K.) acquired CartiHeal Ltd. (Israel), a developer of Agili-C, an innovative sports medicine technology designed for cartilage regeneration in the knee. In May 2024, Amgen Inc. (U.S.) received U.S. Food and Drug Administration (FDA) approval for IMDELLTRA, a T-cell engager therapy for the treatment of extensive-stage small-cell lung cancer. In August 2024, Vericel Corporation (U.S.) received FDA approval for MACI Arthro, a device designed for arthroscopic administration to repair knee cartilage defects. This approval enhanced the company's product portfolio in the MACI segment. In April 2023, Biogen Inc. (U.S.) received the U.S. Food and Drug Administration (FDA) approval for QALSODY (tofersen), a gene therapy used for treating amyotrophic lateral sclerosis (ALS) in people with a mutation in the superoxide dismutase 1 (SOD1) gene. The report provides a competitive dashboard summarizing the market positioning of the 25 profiled market players in four quadrants, namely Industry Leaders, Differentiators, Emerging Companies, and Vanguards. These companies are positioned based on various parameters, including revenue, depth of offerings, brand equity, geographic presence, innovation, and organic & inorganic growth strategies. Novartis AG (Switzerland), Kite Pharma, Inc. (U.S.), Bristol-Myers Squibb Company (U.S.), Janssen Global Services, LLC (U.S.), and Biogen, Inc. (U.S.) are positioned in the Industry Leaders quadrant. IMMEDIATE DELIVERY AVAILABLE | BUY THIS RESEARCH REPORT (Insights, Charts, Tables, Figures and More)- Scope of the Report: Regenerative Medicine Market Assessment—by Product Gene Therapy Cell Therapy Stem Cell Therapy Autologous Therapy Allogenic Therapy Platelet-rich Plasma Therapy Cell-based Immunotherapy Tissue Engineering Regenerative Medicine Market Assessment—by Application Cardiology Ophthalmology Oncology Neurology Immunology & Inflammation Musculoskeletal Dermatology Other Applications Note: Other applications include wound care, orthopedics, and blood disorders. Regenerative Medicine Market Assessment—by End User Hospitals & Clinics Ambulatory Surgical Centers Regenerative Medicine Market Assessment—by Geography North America U.S. Canada Europe Germany France U.K Italy Spain Switzerland Netherlands Sweden Rest of Europe (RoE) Asia-Pacific (APAC) China Japan India South Korea Australia Rest of Asia-Pacific (RoAPAC) Latin America Brazil Mexico Rest of Latin America Middle East & Africa Browse Related Reports: Sports Medicine Market - Sports Medicine Market is set to hit $10.59 billion by 2031, growing at an 8.2% CAGR from 2024. Discover trends and market dynamics driving this growth. Collagen and Gelatin Market - The collagen and gelatin market for regenerative medicine is expected to reach USD 709.9 million by 2022, at a CAGR of 8.5% during the forecast period of 2017 to 2022. Radiation Dose Management Market - Radiation Dose Management Market is projected to reach $749.1 million by 2029, at a CAGR of 13.3%. Molecular cytogenetics market - Molecular cytogenetics market is expected to reach USD 2,266.4 million by 2022, at a CAGR of 11% during the forecast period of 2017 to 2022. Next Generation Sequencing (NGS) Informatics Market - Next Generation Sequencing (NGS) Informatics Market is projected to reach $4.3 billion by 2030, at a CAGR of 15.4% from 2023 to 2030. Regenerative Medicine Market Research Summary: Contact: Mr. Khushal Bombe Meticulous Market Research Pvt. Ltd. 1267 Willis St, Ste 200 Redding, California, 96001, U.S. USA: +1-646-781-8004 Europe: +44-203-868-8738 APAC: +91 744-7780008 Email- [email protected] Visit Our Website: Connect with us on LinkedIn- Content Source: Logo: SOURCE Meticulous Market Research Pvt. Ltd. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED