Forge 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

Forge 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


  • FBX-101 clinical data demonstrating safety and initial efficacy from the RESKUE trial is being presented at the 2022 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium
  • FBX-101 is a systemic AAV gene therapy following unrelated cord blood transplantation (“UCBT”) and has been well tolerated through Day 180
  • FBX-101 significantly increased GALC enzyme activity in leukocytes and patient exhibited improved motor function and normal brain development

COLUMBUS, Ohio – August 30, 2022 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.

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 the 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:

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

Media Inquiries 
Marina Corleto – Forge Biologics
Senior Manager, Marketing and Communications   

Families and Clinician Inquiries
Maria Escolar, M.D. – Forge Biologics
Chief Medical Officer

Investor Relations  
Christina Perry – Forge Biologics
Senior Vice President, Finance and Investor Relations   

Business Development  
Magdalena Tyrpien – Forge Biologics
Senior Vice President, Head of Business Development   


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