Forge Biologics Announces Regulatory Updates from FDA and EMA, Accelerating Manufacturing and Clinical Trial Momentum

Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced that it has received additional designations and regulatory feedback from the European Medicines Agency (EMA) and the U.S. Food & Drug Administration (FDA), advancing the manufacturing capabilities for its own and clients' AAV gene therapies.

The EMA has granted orphan designation for the company's clinical-stage gene therapy FBX-101 for the treatment of patients with Krabbe disease. FBX-101 is a first-in-human potential treatment utilizing a systemically administered adeno-associated virus (AAV) to deliver the correct version of the faulty gene responsible for an abnormality in patients with Krabbe disease, a rare and fatal genetic disorder. FBX-101 previously received Orphan Drug and Rare Pediatric Disease Designations from the FDA earlier this year.

"FBX-101 is the culmination of over 20 years of scientific research to understand the challenges faced by patients with progressive disease after hematopoietic stem cell transplantation, the current standard of care," said Maria Escolar, M.D., Forge's Chief Medical Officer. "We are currently recruiting patients for our RESKUE clinical trial to administer FBX-101 and we are pleased by the EMA's designation so that patients in Europe may also have access to this potentially transformative therapeutic approach."

The company also announced that the FDA has provided positive feedback on Forge's AAV manufacturing processes, including use of its exclusive production plasmids and master cell bank for cGMP manufacturing of gene therapies.

Forge presented information to the FDA on its proprietary HEK293 suspension cell line, Ignition Cellstm, and adenovirus helper plasmid, pEMBRtm, and received FDA alignment that these technologies are suitable for cGMP manufacturing of clinical drug products.

"We are excited that the tools we have developed at Forge will help accelerate and scale cGMP production of AAV gene therapies for patients. We are thankful to have a positive dialogue with the EMA and for gaining alignment with the FDA on the suitability of our key production materials for our own and our clients' use," said Christopher Shilling, Vice President of Regulatory Affairs and Quality. "These new technologies can help mitigate current safety concerns surrounding critical raw materials used in AAV scale-up and production processes for programs moving from research to clinical stage."

Forge offers "concept to clinic" AAV manufacturing and development services to accelerate gene therapy programs from preclinical stage through clinical with technology platforms enabling fully traceable cell line history for potential commercialization.

About Krabbe Disease

Krabbe disease is a rare, pediatric leukodystrophy 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 galactosylceramidase (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. 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 age 2-4 years, while later onset or "Late Infantile" cases have a more variable course of progressive decline. There is currently no approved treatment for Krabbe disease.

About FBX-101

Forge developed FBX-101 to treat patients with Infantile Krabbe disease. FBX-101 is an adeno-associated viral (AAV) 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 in animal models, 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.

About Orphan Designation

EMA orphan designation is designed to encourage the development of new treatments for life-threatening or chronically debilitating conditions that are rare (affecting not more than five in 10,000 people in the European Union). Medicines like FBX-101 that meet the EMA's orphan designation criteria qualify for several incentives, including extended market exclusivity, protocol assistance, and potentially reduced fees for regulatory activities. The orphan designation is issued by the EMA Committee for Orphan Medicinal Products (COMP) for use in the European Union.

About Forge Biologics

Forge Biologics is a hybrid gene therapy contract manufacturing and therapeutics development company. Forge's mission is to enable access to life changing gene therapies and help bring them from idea into reality. Forge has a 175,000 square foot facility in Columbus, Ohio, The Hearth, to serve as its headquarters. The Hearth is a custom-designed cGMP facility dedicated to AAV vector manufacturing and will 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.

For more information, please visit https://www.forgebiologics.com.