A Long-term Follow-up Study to Evaluate Safety and Efficacy of Krabbe Patients From Gene Therapy Clinical Trials Involving the Administration of FBX-101 (AAVrh.10-hGALC)

This is an observational study that will enroll any patients with Krabbe disease that have participated in prior interventional clinical trials involving the administration of FBX-101.

Start Date06 Aug 2024

Sponsor / Collaborator

Forge Biologics, Inc.Startup

NCT05739643 / RecruitingPhase 1/2

A Phase 1/2 Clinical Study of Intravenous AAVrh10 Vector Expressing GALC in Krabbe Subjects Who Previously Received Hematopoietic Stem Cell Transplantation (REKLAIM)

This is a non-blinded, non-randomized dose escalation study of intravenous FBX-101 in which subjects will receive a single infusion of an adeno-associated virus gene therapy product, after more than 21 days of the HSCT (UCBT preferred HSCT source). Data from previously transplanted patients with infantile and late infantile Krabbe disease will be used as a comparator group.

Start Date03 Feb 2023

Sponsor / Collaborator

Forge Biologics, Inc.Startup

NCT04693598 / Active, not recruitingPhase 1/2

A Phase 1/2 Clinical Study of Intravenous Gene Transfer With an AAVrh10 Vector Expressing GALC in Krabbe Subjects Receiving Hematopoietic Stem Cell Transplantation (RESKUE)

This is a nonblinded, non-randomized dose escalation study of intravenous AAVrh10 after hematopoietic stem cell transplantation (HSCT) in which subjects will receive standard of care hematopoietic cell transplantation for Krabbe disease, followed by a single infusion of an adeno-associated virus gene therapy product. Extensive natural history subjects will be used to compare as control group.

Start Date05 Nov 2021

Sponsor / Collaborator

Forge Biologics, Inc.Startup

100 Clinical Results associated with FBX-101

100 Translational Medicine associated with FBX-101

100 Patents (Medical) associated with FBX-101

Literatures (Medical) associated with FBX-101

26 Feb 2021·BioImpactsQ3 · ENGINEERING & TECHNOLOGY

Can early treatment of twitcher mice with high dose AAVrh10-GALC eliminate the need for BMT?

Q3 · ENGINEERING & TECHNOLOGY

ArticleOA

Author: Luzi, Paola ; Rafi, Mohammad A ; Wenger, David A

Introduction: Krabbe disease (KD) is an autosomal recessive disorder caused by mutations in the galactocerebrosidase (GALC) gene resulting in neuro-inflammation and defective myelination in the central and peripheral nervous systems. Most infantile patients present with clinical features before six months of age and die before two years of age. The only treatment available for pre-symptomatic or mildly affected individuals is hematopoietic stem cell transplantation (HSCT). In the animal models, combining bone marrow transplantation (BMT) with gene therapy has shown the best results in disease outcome. In this study, we examine the outcome of gene therapy alone. Methods: Twitcher (twi) mice used in the study, have a W339X mutation in the GALC gene. Genotype identification of the mice was performed shortly after birth or post-natal day 1 (PND1), using polymerase chain reaction on the toe clips followed by restriction enzyme digestion and electrophoresis. Eight or nine-day-old affected mice were used for gene therapy treatment alone or combined with BMT. While iv injection of 4 × 1013 gc/kg of body weight of viral vector was used originally, different viral titers were also used without BMT to evaluate their outcomes. Results: When the standard viral dose was increased four- and ten-fold (4X and 10X) without BMT, the lifespans were increased significantly. Without BMT the affected mice were fertile, had the same weight and appearance as wild type mice and had normal strength and gait. The brains showed no staining for CD68, a marker for activated microglia/macrophages, and less astrogliosis than untreated twi mice. Conclusion: Our results demonstrate that, it may be possible to treat human KD patients with high dose AAVrh10 without blood stem cell transplantation which would eliminate the side effects of HSCT.

01 Nov 2015·Molecular TherapyQ1 · MEDICINE

Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice

Q1 · MEDICINE

Article

Author: Luzi, Paola ; Rao, Han Zhi ; Rafi, Mohammad A ; Wenger, David A

Krabbe disease is an autosomal recessive disorder resulting from defects in the lysosomal enzyme galactocerebrosidase (GALC). GALC deficiency leads to severe neurological features. The only treatment for presymptomatic infantile patients and later-onset patients is hematopoietic stem cell transplantation (HSCT). This treatment is less than ideal with most patients eventually developing problems with gait and expressive language. Several naturally occurring animal models are available, including twitcher (twi) mice, which have been used for many treatment trials. Previous studies demonstrated that multiple injections of AAVrh10-GALC into the central nervous system (CNS) of neonatal twi mice resulted in significant improvements. Recently we showed that one i.v. injection of AAVrh10-GALC on PND10 resulted in normal GALC activity in the CNS and high activity in the peripheral nervous system (PNS). In the present study, a single i.v. injection of AAVrh10-GALC was given 1 day after bone marrow transplantation (BMT) on PND10. The mice show greatly extended lifespan and normal behavior with improved CNS and PNS findings. Since HSCT is the standard of care in human patients, adding this single i.v. injection of viral vector may greatly improve the treatment outcome.

01 Mar 2015·Molecular Genetics and MetabolismQ2 · BIOLOGY

Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features

Q2 · BIOLOGY

Article

Author: Curtis, Mark T ; Luzi, Paola ; Luddi, Alice ; Rao, Han Zhi ; Wenger, David A ; Rafi, Mohammad A

Globoid cell leukodystrophy (GLD) or Krabbe disease is an autosomal recessive disorder resulting from the defective lysosomal enzyme galactocerebrosidase (GALC). The lack of GALC enzyme leads to severe neurological symptoms. While most human patients are infants who do not survive beyond 2 years of age, older patients are also diagnosed. In addition to human patients, several naturally occurring animal models, including dog, mouse, and monkey, have also been identified. The mouse model of Krabbe disease, twitcher (twi) mouse has been used for many treatment trials including gene therapy. Using the combination of intracerebroventricular, intracerebellar, and intravenous (iv) injection of the adeno-associated virus serotype rh10 (AAVrh10) expressing mouse GALC in neonate twi mice we previously have demonstrated a significantly extended normal life and exhibition of normal behavior in treated mice. In spite of the prolonged healthy life of these treated mice and improved myelination, it is unlikely that using multiple injection sites for viral administration will be approved for treatment of human patients. In this study, we have explored the outcome of the single iv injection of viral vector at post-natal day 10 (PND10). This has resulted in increased GALC activity in the central nervous system (CNS) and high GALC activity in the peripheral nervous system (PNS). As we have shown previously, an iv injection of AAVrh10 at PND2 results in a small extension of life beyond the typical lifespan of the untreated twi mice (~40 days). In this study, we report that mice receiving a single iv injection at PND10 had no tremor and continued to gain weight until a few weeks before they died. On average, they lived 20-25 days longer than untreated mice. We anticipate that this strategy in combination with other therapeutic options may be beneficial and applicable to treatment of human patients.

News (Medical) associated with FBX-101

02 May 2024

·www.globenewswire.com

Tenaya Therapeutics to Highlight Growing Capabilities in Capsid Engineering, Gene Editing and Manufacturing at the American Society of Gene and Cell Therapy 27th Annual Meeting

Abstracts Demonstrate Tenaya’s Continued Innovation Across Core Competencies Facilitating Current Pipeline and Enabling Next Wave of Precision Heart MedicinesSOUTH SAN FRANCISCO, Calif., May 02, 2024 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced that it will present seven abstracts focused on the company’s growing capabilities for discovering and advancing genetic medicines for heart disease at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, being held May 7-11, 2024, in Baltimore, MD. Tenaya has established integrated internal capabilities to broadly enable modality agnostic target validation and the design and manufacture of adeno associated virus (AAV)-based genetic medicines focused on the treatment of heart diseases. The company’s pipeline includes two clinical-stage gene therapies for cardiomyopathies, as well as earlier-stage research related to gene therapy, gene editing and cardiac cell regeneration, all using adeno-associated virus (AAV) as a delivery vehicle. Tenaya’s seven presentations at this year’s ASGCT will highlight new capsid engineering insights, the company’s emerging gene editing efforts, and manufacturing process optimizations intended to enhance the safety and efficacy profiles of AAV-based gene therapies. Details of the presentations are as follows: Capsid Engineering and PromotersTenaya has established an active capsid engineering effort encompassing the identification and comparison of known and novel capsids, which are then tested across multiple species to characterize transduction and expression in specific heart cells and/or liver detargeting. At ASGCT, new data from murine and non-human primate studies will be presented comparing previously identified AAV-based capsids, AAV9, AAVrh10 and AAVrh74, for cardiac cell tropism and transgene expression. In two other posters, Tenaya scientists will present new data describing a novel approach to creating a library of promoters for cardiac-specific gene expression, as well as detailing efforts to design more compact cardiac-specific promoters to accommodate larger gene therapy or gene editing therapeutics. Wednesday Poster Session, May 8, 2024, at 12 pm ET AAV9, AAVrh.10, and AAVrh.74 Exhibit Different Cell Type Tropisms in the Heart Following Systemic Delivery and AAV9 Mediates Superior Cardiomyocyte Transgene Expression in Murine and NHP (abstract #2662) Lead author: Ze Cheng, Ph.D., Senior Scientist Chimeric and Rationally Designed Compact Promoters for Cardiac-Specific Gene Expression (abstract #464)AAV DNA shuffle library of GH Loop Variable Regions for Directed Evolution of Cardiotropic Capsids (abstract #482) Lead author: Prasad Konkalmatt, Associate Director, AAV Capsid Engineering Gene EditingTenaya scientists will present preclinical results suggesting that gene editing of the R14del variant of the phospholamban (PLN) gene may hold promise as a treatment for PLN-R14del-associated dilated cardiomyopathy. Tenaya has developed a gene editing therapy utilizing a single AAV vector designed to deliver a proprietary self-inactivating CRISPR-Cas9 and PLN- R14del-mutation-specific single guide RNA. Previously, Tenaya shared data from a mouse model showing that the company’s gene editing vector corrected the PLN-R14del mutation while leaving the wild-type PLN gene intact resulting in preserved heart function, reduced fibrosis and extended survival. This year’s presentation will replicate that set of data in the same model of disease using self-inactivating vectors designed to decrease the risk of off-target edits without any diminishment of efficacy. Friday Poster Session, May 10, 2024, at 12 pm ET Precision Editing of PLNR14del Mutation Using a Self-Inactivating, All-in-One AAV Vector to Rescue PLN-R14del-Associated Cardiomyopathy (abstract #1701) Lead author: Huanyu Zhou, Ph.D., Senior Scientist Product Development and Manufacturing Clinical supply of TN-201 and TN-401 gene therapies was manufactured under current Good Manufacturing Practice regulations at Tenaya’s Genetic Medicines Manufacturing Center using the company’s proprietary Sf9 recombinant baculovirus (Sf9/rBV) production process at the 1000L scale. At ASGCT, Tenaya researchers will present abstracts related to increasing yield and scalability associated with Sf9/rBV manufacturing processes. Tenaya has also internalized the HEK293 manufacturing platform up to the 200L scale. Tenaya researchers will present data on a novel small molecule additive that increases the productivity of HEK293-based AAV manufacturing processes, which may have implications for improved scalability, productivity and costs. Friday Poster Session, May 10, 2024, at 12 pm ET Development of a Scalable High Yield HEK293 Expression Platform for AAV Manufacturing (abstract #1531) Lead author: Charles Feathers, Process Development Manager Utilization of Tenaya’s AAV Productivity Boosting Small Molecule (SMB) for Intelligent Design of HEK293 Cell Line to Improve AAV Productivity (abstract #1530)Development of Highly Productive, Rhabdovirus-free Sf9 Insect Cell Line for Large-scale AAV Production for Cardiovascular Gene Therapies (abstract #1533) Lead author: Jackson Leong, Process Development Associate Scientist, To view full event programming, please visit the ASGCT 27th Annual Meeting website. Following the conference, Tenaya’s presentations will be available in the “Our Science” section of the company’s website. About Tenaya TherapeuticsTenaya Therapeutics is a clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Leveraging integrated proprietary core capabilities enabling target identification and validation, design of AAV-based genetic medicines and in-house manufacturing the company is advancing a pipeline of novel therapies with diverse treatment modalities for rare genetic cardiovascular disorders and more prevalent heart conditions. Tenaya’s most advanced candidates include TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301, a small molecule HDAC6 inhibitor being initially developed for heart failure with preserved ejection fraction (HFpEF). Tenaya also has multiple early-stage programs progressing through preclinical development. For more information, visit www.tenayatherapeutics.com. Forward-Looking StatementsThis press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Words such as “will,” “may,” “promise,” and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, the presentation of data covering Tenaya’s capabilities for discovering and advancing genetic medicines; and the therapeutic and commercial potential of Tenaya’s capsid engineering, gene editing and manufacturing process optimizations efforts. The forward-looking statements contained herein are based upon Tenaya’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including but not limited to: availability of data at the referenced times; risks associated with the process of discovering, developing and commercializing therapies that are safe and effective for use as human therapeutics; Tenaya’s ability to develop, initiate or complete preclinical studies and clinical trials, and obtain approvals, for any of its product candidates; Tenaya’s continuing compliance with applicable legal and regulatory requirements; Tenaya’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; Tenaya’s reliance on third parties; Tenaya’s manufacturing, commercialization and marketing capabilities and strategy; the loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenaya’s ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled “Risk Factors” in documents that Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.Contact Michelle CorralVP, Corporate Communications and Investor RelationsTenaya TherapeuticsIR@TenayaThera.com InvestorsAnneMarie FieldsStern IR AnneMarie.Fields@SternIR.com MediaWendy RyanTen Bridge Communicationswendy@tenbridgecommunications.com

Gene TherapyClinical Study

30 Apr 2024

·www.biospace.com

Forge Biologics Announces Nine Presentations at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

COLUMBUS, Ohio--(BUSINESS WIRE)-- Forge Biologics, a member of Ajinomoto Bio-Pharma Services and leading manufacturer of genetic medicines, today announced that it will deliver nine presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27thAnnual Meeting, taking place May 7-11, 2024, in Baltimore, Maryland. The presentations include a late-breaking oral presentation, three technical session presentations, and five scientific posters spanning Forge’s process development, molecular development, integrated regulatory services, and clinical program advancements. “As we continue to advance how genetic medicines are manufactured, we are thrilled to present data showcasing our novel molecular findings, platform process improvements, and regulatory knowledge all centered around efficient, safe, and scalable manufacturing of AAV gene therapies,” said Timothy J. Miller, Ph.D., President and CEO of Forge. “We are also especially encouraged by a late-breaking oral presentation that our Chief Medical Officer, Dr. Maria Escolar, will be delivering that provides a clinical update on FBX-101 where all five patients with Krabbe disease receiving Forge’s gene therapy are walking—a remarkable result. Retention of gross-motor function is one of the most visible and debilitating symptoms seen in patients with Krabbe disease, so this finding is a meaningful clinical outcome.” ORAL PRESENTATION Title: REKLAIM, A Phase 1b Clinical Trial Using a Novel Immune Modulation Strategy for Systemic Administration of FBX-101 (AAVrh10.GALC) After Umbilical Cord Blood Transplantation for the Treatment of Infantile Krabbe Disease Presenter: Maria Escolar, M.D., Chief Medical Officer Date and Time: Friday, May 10, 2024, 8:00-8:15 a.m. ET Room: Ballroom 4 SESSION PRESENTATIONS ASGCT Workshop Title: Preparing for CMC Success: How Early CMC Decision Drives Your Pace and Success to the Clinic Presenter: David Dismuke, Ph.D., Chief Technical Officer Date and Time: Tuesday, May 7, 8:25 a.m. ET Location: Ballroom 3 Tools & Technology Forum Title: Improving Analytical Tools for AAV Characterization Presenter: David Dismuke, Ph.D., Chief Technical Officer Date and Time: Wednesday, May 8, 2:15-2:30 p.m. ET Location: Exhibit Hall Theater Sponsored Symposium Title: Forge’s Discovery and Modification of Genetic Elements for Enhanced Productivity and Efficacy of AAV-based Gene Therapies Presenters: Angela Adsero, Ph.D., Molecular Development Scientist II Frank Agbogbo, Ph.D., VP of Process Development David Dismuke, Ph.D., Chief Technical Officer Date and Time: Thursday, May 9, 1:30-2:00 p.m. ET Location: Room 337-338 POSTER PRESENTATIONS Title: Development of a Scalable Upstream Process for Suspension HEK 293 rAAV Production in Single-Use Bioreactors for Efficient Manufacturing of Gene Therapy Products Presenter: Danielle Sexton, Senior Scientist II, Process Development Date and Time: May 8, 12:00 p.m. ET Poster number: 528 Title: From Benchtop to Cleanroom; Designing an Optimized Platform Purification Process for rAAV Production to Meet the Growing Demand Presenter: Blake Gursky, Scientist II, Process Development Date and Time: May 8, 12:00 p.m. ET Poster number: 527 Title: The Potential of Global Regulatory Harmonization in Gene and Cell Therapy Presenter: Claire Marasco, Associate Director, Regulatory Affairs Date and Time: May 8, 12:00 p.m. ET Poster number: 526 Title: Regulatory Agility: Value of CDMOs Utilizing Platform Technology Presenter: Angela Coy, Ph.D., Senior Manager, Regulatory Affairs Date and Time: May 8, 12:00 p.m. ET Poster number: 905 Title: The Sixth Element: Identification and Evaluation of the Adenovirus L4 22/33K Region as a Requirement for Adeno-Associated Virus Production Presenter: Angela Adsero, Ph.D., Molecular Development Scientist II, Forge Biologics Date and Time: May 9, 12:00 p.m. ET Poster number: 954 Full abstracts are available on the ASGCT Annual Meeting website, and conference participants can also access posters through the ASGCT website. Forge Biologics will be at booth #827 in the Exhibitor Hall. About Forge Biologics Forge Biologics, a member of Ajinomoto Bio-Pharma Services, is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company, enabling access to life-changing gene therapies by bringing them from concept to reality. Forge’s 200,000 square foot facility, the Hearth, is headquartered in Columbus, Ohio, and houses 20 custom-designed cGMP suites with 200,000L of manufacturing capacity. Forge’s end-to-end, scalable plasmid and AAV manufacturing services include research-grade manufacturing, process and analytical development, cGMP manufacturing, fill and finish, and integrated regulatory support to help accelerate the timelines of transformative medicines for patients with genetic diseases. To learn more, visit .

Phase 1Gene Therapy

19 Mar 2024

·www.biospace.com

Forge Biologics’ Novel AAV Gene Therapy FBX-101 for Patients with Krabbe Disease is Granted UK’s Innovation Passport Designation

Designation grants access to enter the Innovative Licensing and Access Pathway (ILAP) Five patients treated with FBX-101 show positive safety and efficacy results, including significant improvements in motor function Forge’s CMO, Dr. Maria Escolar, to present updated FBX-101 clinical data during the Advanced Therapies 2024 conference in London March 19-20 Designation provides opportunities for further global acceptance of Forge’s manufacturing process and facility through additional reviews and inspections COLUMBUS, Ohio--(BUSINESS WIRE)-- Forge Biologics, a member of Ajinomoto Bio-Pharma Services and a leading manufacturer of genetic medicines, today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA), the healthcare regulatory body of the United Kingdom (UK), granted Innovation Passport designation to the Company’s novel AAV gene therapy program, FBX-101, to enter the Innovative Licensing and Access Pathway (ILAP). FBX-101 was created for the treatment of patients with Krabbe disease, a rare neurodegenerative disease that is usually fatal in untreated patients by age two. The ILAP is intended to accelerate regulatory and market access interactions in the UK. Maria Escolar, M.D., Forge’s Chief Medical Officer, will discuss the significance of achieving this regulatory designation and present a poster on updated data from REKLAIM, an early phase clinical trial for FBX-101, during the Advanced Therapies 2024 conference being held in London, March 19-20, 2024. “Considering the rapid progression of Krabbe disease in young patients and the positive results we’ve observed in the REKLAIM trial, we are pleased to receive the Innovation Passport designation which will enable Forge to further accelerate the clinical development of FBX-101,” stated Dr. Escolar. “ILAP was created to provide access to tools to decrease the time to market and facilitate patient access to medicines in the UK for life-threatening or seriously debilitating conditions.” The REKLAIM clinical trial is evaluating the safety and efficacy of FBX-101 in patients with infantile and late infantile Krabbe disease. Children assessed in REKLAIM have received intravenous FBX-101 in addition to the current standard of care, hematopoietic stem cell transplantation (HSCT). Clinical data demonstrate that FBX 101 has been well tolerated and has an excellent safety pro the treatment of five patients. Results show improvements in motor function in all patients, the most meaningful clinical outcome for this disease. FBX-101 has been granted Orphan Drug Designation and Priority Medicines (PRIME) designation by the European Medicines Agency (EMA), allowing Forge to advance and expedite the development of FBX-101 as the leading worldwide gene therapy for patients with Krabbe disease. In the US, the FDA has granted FBX-101 Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation. ILAP is intended to accelerate regulatory and market access interactions in the UK for patient populations with significant need, leading to accelerated patient access to novel therapies. It also provides access to a range of UK development services and tools, including frequent MHRA interactions, accelerated Marketing Authorization Application (MAA) assessment, a Target Development Profile, expanded licensing routes, and more, creating a roadmap for advancement of therapies through regulatory approval. Regulatory review by the MHRA through ILAP provides opportunities for further global acceptance of Forge’s manufacturing process and facility though additional reviews and inspections, including Compliance Readiness Inspections that provide custom and pragmatic supervisory and licensing inspections specific to the GMP guidelines. Through this designation Forge has expanded global regulatory experience to include interactions with the MHRA in the UK. This access to global health authority resources will enable an added level of assurance to clients utilizing Forge’s CDMO services with integrated regulatory support. About Krabbe Disease Krabbe disease is a rare neurodegenerative disease affecting about 1-2.5 in 100,000 people in the US. Krabbe disease is caused by autosomal recessive mutations in the galactocerebrosidase (GALC) gene, an enzyme responsible for the breakdown of certain types of sphingolipids, such as psychosine. Without functional GALC, psychosine accumulates to toxic levels in cells, specifically in cells insulating the nerves in the brain and peripheral nervous system, causing rapid demyelination. Krabbe disease initially manifests in young patients as irritability, developmental delay, and progressive muscle weakness. Symptoms rapidly advance to difficulty swallowing, breathing, and regression of neurodevelopment followed by seizures, vision and hearing loss. Infantile Krabbe disease (0-12 months of age at onset) usually leads to death in untreated patients by two years of age. Late Infantile patients (12-36 months of age at onset) usually die by the age of six. The current standard of care, hematopoietic stem cell transplantation (HSCT), has been shown to stabilize cognitive decline and significantly improve long-term neurological outcomes. However, HSCT does not correct the peripheral neuropathy that is progressive as the patient grows, leading to loss of gross motor skills and eventually death. About FBX-101 FBX-101 was developed to treat children with Krabbe disease. FBX-101 is an adeno-associated viral serotype rh10 (AAVrh10) gene therapy that is delivered intravenously after HSCT infusion. The vector delivers a functional copy of the GALC gene to cells in both the central and peripheral nervous system and has shown to functionally correct the central and peripheral neuropathy, improve myelination and gross motor function, and significantly prolong lifespan in animal models. This approach also 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 the REKLAIM Trial REKLAIM is a nonblinded, non-randomized, early phase dose escalation clinical trial currently enrolling children with asymptomatic infantile and symptomatic late infantile Krabbe disease to investigate the safety and efficacy of a single intravenous infusion of FBX-101 administered more than 21 days after HSCT, the current standard of care. Data from extensive natural history subjects will be used to compare as the control group. More information on the REKLAIM trial can be found online at . About Forge Biologics Forge Biologics, a member of Ajinomoto Bio-Pharma Services, is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company, enabling access to life-changing gene therapies by bringing them from concept to reality. Forge’s 200,000 square foot facility, the Hearth, is headquartered in Columbus, Ohio, and houses 20 custom-designed cGMP suites with 200,000L of manufacturing capacity. Forge’s end-to-end, scalable plasmid and AAV manufacturing services include research-grade manufacturing, process and analytical development, cGMP manufacturing, fill and finish, and integrated regulatory support to help accelerate the timelines of transformative medicines for patients with genetic diseases. To learn more, visit .

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Indication	Highest Phase	Country/Location	Organization	Date
Leukodystrophy, Globoid Cell	Phase 2	US	Forge Biologics, Inc.Startup	05 Nov 2021

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04693598 (RESKUE, BusinessWire) Manual	Phase 1/2	Leukodystrophy, Globoid Cell	-	FBX-101	(iikooemuos) = FBX-101 significantly increased GALC enzyme activity in white blood cells, improved motor function, and normal brain development nfapmzwigq (arosfjawdo )	Positive	30 Aug 2022

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