Business Wire

OH-FORGE-BIOLOGICS

Share
Forge Biologics Reports Positive Clinical Data on Brain Development and Motor Function from the RESKUE Novel Phase 1/2 Gene Therapy Trial in Patients with Krabbe Disease at the SSIEM Annual Symposium

Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced that Maria Escolar, M.D., Chief Medical Officer, will present preliminary clinical data on the treatment of its first patient in the RESKUE Phase 1/2 clinical trial for FBX-101, the Company’s novel gene therapy for the treatment of patients with Krabbe disease. The data will be presented on August 31, 2022, at the annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) in Freiburg, Germany.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220830005417/en/

To view this piece of content from mms.businesswire.com, please give your consent at the top of this page.

Maria Escolar, M.D., Chief Medical Officer at Forge Biologics (Photo: Business Wire)

RESKUE is the first-in-human clinical trial where subjects are administered FBX-101, an Adeno-Associated Virus (AAV) systemic gene replacement strategy, after full myeloablation and umbilical cord blood transplantation. Clinical data support preclinical observations that this approach may lessen many of the immune challenges previously observed with systemic AAV gene delivery and can create a novel approach for extending the delivery of gene replacement strategies to target metabolic diseases amenable to UCBT. The first patient’s data from the RESKUE clinical trial demonstrate that intravenous FBX-101 following UCBT has been safe and well tolerated through Day 180. Notably, the data demonstrate an absence of humoral immune response against the vector and significantly increased GALC enzyme activity. Through Day 180, the patient exhibited improved motor activity and normal brain development compared to previously reported transplanted patients with Krabbe disease.

“Krabbe disease is a rapidly fatal neurodegenerative disorder. While early transplant can help patients, they inevitably develop motor disease. FBX-101, a systemic AAV therapy administered after hematopoietic stem cell transplantation, was well tolerated in the first patient and the initial clinical data support normal brain development during this period of rapid myelination, and normal gross motor function," stated Dr. Escolar. "The increased GALC levels and ablated immune response achieved in this transplant environment may represent a paradigm shift in the treatment of patients with Krabbe disease and for the field of gene therapy. We are excited by results thus far and we look forward to continuing this trial.”

“We are thrilled with Forge’s commitment to get this much-needed therapy into a clinical trial,” said Stacy Pike-Langenfeld, Co-Founder and President of KrabbeConnect.

“We're deeply grateful for this progress toward better treatments for patients suffering from Krabbe disease thanks to the dedication of the team at Forge,” said Anna Grantham, Leukodystrophy Care Network Director of Hunter’s Hope Foundation.

Dr. Escolar’s poster, “Intravenous FBX-101 (AAVrh10.hGALC) following hematopoietic stem cell transplantation increases GALC activity, improves gross motor function, and maintains brain development in Krabbe patients in the RESKUE Phase 1/2 Clinical Trial,” will be available virtually for all symposium attendees and displayed in the main poster hall throughout the course of the conference. The poster walk will take place on Wednesday, August 31, 2022, from 18:45-20:15 CET. For more details on the symposium, please visit: https://www.ssiem.org/

About Krabbe Disease

Krabbe disease is a rare, neurodegenerative disease affecting about 1-2.5 in 100,000 people in the U.S. and is inherited in an autosomal recessive manner. Krabbe disease is caused by loss-of-function mutations in the galactocerebrosidase (GALC) gene, a lysosomal enzyme responsible for the breakdown of certain types of lipids such as psychosine. Without functional GALC, psychosine accumulates to toxic levels in cells. The psychosine toxicity is most severe in the myelin cells surrounding the nerves in the brain and in the peripheral nervous system, eventually leading to the death of these cells and loss of motor function. The disease initially manifests as physical delays in development, muscle weakness and irritability and advances rapidly to difficulty swallowing, breathing and cognitive problems, and vision and hearing loss. Early onset or “Infantile” Krabbe disease cases usually results in death of patients by the age of two years, while later onset or “Late Infantile” patients usually die by the age of six. Although bone marrow transplant is the current standard of care for treating the central nervous system in patients with Krabbe disease, patients continue to develop peripheral nervous system disease that eventually results in death. Currently, 10 states in the USA are approved to try and identify babies with Krabbe disease as part of newborn screening programs.

About FBX-101

Forge developed FBX-101 to treat patients with Infantile Krabbe disease. FBX-101 is an adeno-associated viral serotype rh10 (AAVrh10) gene therapy that is typically delivered after a hematopoietic stem cell transplant. FBX-101 delivers a functional copy of the GALC gene to cells in both the central and peripheral nervous system. FBX-101 has been shown to functionally correct the central and peripheral neuropathy and correct the behavioral impairments associated with Krabbe disease, as well as to drastically improve lifespan in animal models of the disease. This approach has the potential to overcome some of the immunological safety challenges observed in traditional AAV gene therapies and extend the duration of gene transfer.

About Forge Biologics

Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company. Forge’s mission is to enable access to life changing gene therapies and help bring them from idea to reality. Forge’s 200,000 square foot facility utilizes 20 cGMP suites in Columbus, Ohio, the Hearth, to serve as its headquarters. The Hearth is a custom-designed cGMP facility focused on AAV manufacturing and can host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing. By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most. To learn more, visit www.forgebiologics.com.

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220830005417/en/

About Business Wire

Business Wire
Business Wire
101 California Street, 20th Floor
CA 94111 San Francisco

http://businesswire.com
DK

Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

Year-old European startup Maisa named alongside Google and Amazon in elite list of leading AI agent vendors in top global US research reports by Gartner3.7.2025 01:01:00 CEST | Press release

- First time a Spanish startup has made the list, thanks to its industry-first hallucination resistant ‘digital workers’ - AI startup is one of two European AI agent vendors in Gartner’s Hype Cycle report for Artificial Intelligence and Hype Cycle for the Future of Work lists Maisa, a rising star of enterprise AI, has been named by leading global research and advisory firm Gartner in its list of leading vendors for developing reliable AI agents. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250702640749/en/ Maisa founders David Villalón and Manuel Romero Inclusion in Gartner’s 2025 Hype Cycle for AI and Hype Cycle for the Future of Work marks the first time a Spanish startup has been mentioned in these influential reports. The company, which is barely a year old and made its first raise of $5m+ from leading US investors last year, now finds itself named alongside global giants Amazon Web Services, Google, Salesforce and Lan

Hoffmann Green Announces a Historic Increase in Production Over the First Half of 2025, Already Outperforming the Annual Volumes Realized in 20242.7.2025 19:05:00 CEST | Press release

Record activity levels: +151% increase in production volumes in H1 2025 vs. H1 2024More than 130 construction sites supplied with more than 60,000 m³ of 0% clinker concrete delivered by 10,000 truck mixers across the countryAcceleration driven by a strengthened partnership network and the successful diversification of targeted marketsSolid outlook for the second half of 2025, in line with the strategic roadmap Hoffmann Green Cement Technologies (ISIN: FR0013451044, Ticker: ALHGR) (“Hoffmann Green Cement” or the “Company”), an industrial player committed to the decarbonation of the construction sector that designs and markets innovative clinker-free cements, today announces a strong acceleration of production over the first half of 2025, validating the relevance of its industrial and commercial business model. Production volumes reached 19,640 tonnes over the first half of 2025, compared with 7,833 tonnes in the first half of 2024, representing a 2.5-fold increase. This very strong leve

Institutional Real Estate, Inc. Announces Acquisition of U.K.-based Lyndon Publishing 2 Limited2.7.2025 18:44:00 CEST | Press release

Institutional Real Estate, Inc. (“IREI”) has completed its acquisition of the assets of London-based Lyndon Publishing 2 Ltd (“Lyndon Publishing”), which include The Property ChronicleandThe Green Chronicle.The former Lyndon Publishing 2 will now operate as a division of IREI. The Property Chronicle reaches a global audience of real estate investment professionals, researchers, and academics. Its sister publication, The Green Chronicle, is edited to address the interests and concerns of emerging young talent in these real estate professions. Both publications are produced as weekly news briefings filled with original content and contributed feature articles from industry thought and opinion leaders. In addition, The Property Chronicle is also published as a quarterly, illustrated full-color magazine available in both digital and print formats, while The Green Chronicleis published exclusively in digital format. Together, the two publications reach approximately 50,000 professionals acr

Sinopec Wins Technological Innovation Award at 2nd Sino-European Corporate ESG Best Practice Conference2.7.2025 18:21:00 CEST | Press release

China Petroleum & Chemical Corporation ("Sinopec", HKG: 0386) has won the "Best Scientific and Technological Innovation Practice" award at the 2nd Sino-European Corporate ESG Best Practice Conference (the "Conference") on June 26 in Stuttgart. Sinopec’s case submission of Breakthrough in Depth, Climb to New Heights — Empowering the Energy and Chemical Industry with Technological Innovation stood out among numerous entries. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250702387465/en/ Wang Tao, Sinopec representative, delivered a speech at the conference. The Conference reviews and awards the best ESG application cases in six areas of environmental protection, social responsibility, corporate management, scientific and technological innovation, education, and training to further enhance mutual understanding between Chinese and European businesses, facilitate bilateral economic and trade exchanges, and showcase China’s open,

Xsolla Releases Their Q2 2025 The State of Play Report: Mobile Gaming Is Projected to Hit $126B in 2025 Amid Global App Boom With In-Depth Analysis of Trends, Genres, and Monetization Strategies2.7.2025 18:00:00 CEST | Press release

Key Topics Include In-App Purchase Trends, Genre Performance, Player Demographics, D2C Strategy Adoption, and Hybrid Monetization Models Xsolla, a leading global video game commerce company, proudly announces the release of the Q2 2025 Edition of “The Xsolla Report: The State of Play.” This detailed report provides crucial insights, trends, and opportunities shaping the gaming landscape, empowering industry professionals to adapt and thrive in today’s rapidly changing market. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250702208715/en/ Graphic: Xsolla In 2024, global mobile app usage soared to an astonishing 4.2 trillion hours, an all-time high that powered a massive $150 billion consumer spending across mobile platforms. Mobile gaming is leading this charge, which accounted for more than half of that total. Despite a 6% dip in overall game downloads, falling to 49.6 billion, in-app purchase (IAP) revenue surged, indicati

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom
World GlobeA line styled icon from Orion Icon Library.HiddenA line styled icon from Orion Icon Library.Eye