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Gene Therapy Production Company Opens, Raises $40M

Adeno-associated virus

Adeno-associated virus diagram (Lawrence Berkeley National Lab)

21 July 2020. A new enterprise developing and manufacturing gene therapies, both its own and for others, is underway, raising $40 million in venture funds. Forge Biologics in Columbus, Ohio is also commercializing research at University of Pittsburgh on the rare children’s disorder Krabbe disease as its first gene therapy.

Forge Biologics provides specialized lab space and manufacturing facilities for gene therapies designed to treat inherited diseases. The company devotes its development and production efforts to adeno-associated viruses for delivering healthy genes to replace missing or defective genes in patients. The adeno-associated virus or AAV is a benign and naturally occurring microbe that infects cells, but does not integrate with the cell’s genome or cause disease, other than at most mild reactions. As a result, the virus has become a workhorse for delivering modified genes to treat inherited diseases. As reported by Science & Enterprise in January, FDA began issuing new regulatory guidance on gene therapies, including for manufacturing.

At present, Forge Biologics provides lab and production facilities for research-scale work on AAVs and gene therapies. By mid-2021, however, the company plans to scale up its manufacturing capabilities to provide full-scale services for biotechnology and pharmaceutical companies, from preclinical research to commercial production. The new facility, called The Hearth, is expected to have 175,000 square feet of manufacturing space for gene therapies that meet current pharmaceutical industry standards regulated by FDA.

“As gene therapy programs continue to demonstrate clinical success,” says Forge Biologics’ co-founder and CEO Timothy Miller in a company statement released through BusinessWire, “the need for AAV manufacturing has never been greater. Our mission is to enable access to potentially life-saving therapies and help bring them from idea into reality.”

In addition to its contract gene therapy services, Forge Biologics is developing its own treatments for rare diseases. The company’s lead product, code-named FBX-101, is a gene therapy for Krabbe disease, a severe genetic neurological condition mainly affecting infants. Krabbe disease results from mutations in the galactosylceramidase or GALC gene that provides instructions for enzymes to break down certain fats in the body, including those that regulate production of myelin, the protective coating on neurons or nerve cells in the brain and elsewhere in the body.

Without these enzymes, the fats turn toxic and prevent myelin from forming around neurons, causing damage to nerve cells. Infants under the age of one year with Krabbe disease first show irritability, muscle weakness, and feeding difficulties, and later exhibit difficulty moving, chewing, swallowing, and breathing, as well as vision loss and seizures. FBX-101 is based on research by University of Pittsburgh pediatric neurologist Maria Escolar that combines gene therapy with an umbilical cord bone marrow transplant to address central and peripheral nerve degeneration associated with Krabbe disease. Escolar is joining Forge Biologics as chief medical officer.

Forge Biologics is raising $40 million in its first venture funding round, led by the Perceptive Xontogeny Venture Fund, with Drive Capital also taking part. The Perceptive Xontogeny Venture Fund finances early-stage companies incubated by Xontogeny, a life science incubator in Boston. Perceptive Advisors, a life science venture investor in New York, provides financing for the fund.

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