- Forge Biologics has received IND, IBC, and IRB
clearance for FBX-101, a gene therapy for patients with Krabbe
disease, a rare and fatal genetic disorder
- FBX-101 is a first-in-human potential treatment
utilizing an adeno-associated virus (AAV) to deliver a functioning
copy of the GALC gene to cells in the CNS and peripheral
organs
- FBX-101 GMP manufacturing is completed, screening for
enrollment commences in January
- The RESKUE trial evaluating FBX-101 is the
culmination of nearly 20 years of Krabbe disease research, led by
Maria Escolar, M.D., Forge Biologics' Chief Medical Officer
COLUMBUS, Ohio, Jan. 4, 2021 /PRNewswire/ -- Forge
Biologics Inc., a gene therapy manufacturing and development
company, today announced that the company has received FDA
clearance of the Investigational New Drug (IND) to initiate a Phase
1/2 clinical trial evaluating its novel, first-in-human AAV gene
therapy, FBX-101, for patients with Krabbe disease. FBX-101 is the
company's first therapeutic program to receive an IND which also
received Institutional Biosafety Committee (IBC) and Institutional
Review Board (IRB) approvals required prior to any patient
enrollment. This marks a major step forward in building out the
company's hybrid model as a gene therapy manufacturing and
development engine.
Krabbe disease is a rare and fatal pediatric leukodystrophy
affecting about 1-2.6 in 100,000 people in the United States. Patients are born with
mutations in the galactosylceramidase (GALC) gene, which
encodes an enzyme that helps break down lipid molecules inside
cells. This results in the toxic buildup of psychosine, a lipid
molecule that can't be degraded in cells, particularly in cells in
the brain and peripheral nerves, and leads to toxic levels that
cause cell death and myelin loss. The disease initially presents as
physical delays in development, muscle tightness and irritability,
and rapidly advances to difficulty swallowing and breathing, loss
of vision and hearing, and increasing cognitive degeneration. Early
onset, or "Infantile", Krabbe disease cases usually results in
death 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 either form of Krabbe
disease.
FBX-101 is an adeno-associated viral (AAV) gene therapy
administered after hematopoietic stem cell transplant (HSCT), that
delivers a functioning copy of the GALC gene, the enzyme needed to
prevent the buildup of psychosine in myelinated cells of both the
central and peripheral nervous system. FBX-101 has been shown to
correct the central and peripheral myelination deficits,
significantly improve the behavioral impairments associated with
Krabbe disease in animal models, and drastically improve the
lifespan of treated animals. The use of transplant and intravenous
AAV gene therapy infusion has the potential to overcome some of the
immunological safety challenges of traditional AAV gene
therapies.
"The ground-breaking treatment approach using HSCT and AAV gene
therapy, initially developed by Dr. Escolar, has safely
demonstrated superior benefits in preclinical animal studies of
Krabbe disease than either treatment method alone," said
Timothy J. Miller, Ph.D., Forge's
CEO, President, and Co-Founder. "We are grateful for the FDA's
engaged review and allowance of the IND, and look forward to
enrolling patients very soon."
FBX-101 is the culmination of nearly 20 years of Krabbe disease
research, led by Maria Escolar, M.D., M.S., Chief Medical Officer
at Forge Biologics and the pioneer for evaluating the natural
history and new treatment approaches for patients with Krabbe
disease. "This combination approach is extremely exciting because
the preclinical data demonstrate significant correction of
survival, behavior and neuromuscular function in animal models
compared to either transplant or AAV treatment alone. This is a
significant milestone for Krabbe disease families suffering from
this deadly disease," said Dr. Escolar.
The initiation of the RESKUE trial in Forge's gene therapy
pipeline continues Forge's momentum within the biotechnology
industry in Columbus, Ohio,
bringing positive impact to both Ohio and the global rare disease
community.
Patients and families can learn more about clinical trials for
FBX-101 by visiting
https://www.forgebiologics.com/science/#krabbe.
About Krabbe disease
Krabbe disease is a rare,
pediatric leukodystrophy affecting about 1-2.6 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 problems, cognitive, vision and hearing loss.
Early onset or "Infantile", Krabbe disease cases usually results in
death 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 is developing FBX-101 to treat
patients with infantile Krabbe disease. FBX-101 is an
adeno-associated viral (AAV) gene therapy that is 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, and to
drastically improve the lifespan of treated animals. This approach
has the potential to overcome some of the immunological safety
challenges observed in traditional AAV gene therapies.
About Forge Biologics
Forge Biologics is a hybrid gene
therapy contract manufacturing and therapeutic 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
ft2 facility in Columbus,
Ohio, "The Hearth", to serve as their headquarters. The
Hearth is the home of a custom-designed cGMP facility dedicated to
AAV viral 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.
Patient, Pediatrician, Genetic Counselors & Family
Inquiries
Dr. Maria Escolar
Chief Medical Officer
Forge Biologics Inc.
medicalaffairs@forgebiologics.com
Media Inquiries:
Dan Salvo
Director of Communications and Community Development
Forge Biologics Inc.
media@forgebiologics.com
Investor Relations and Business Development
Christina Perry
Vice President, Finance and Operations
Forge Biologics Inc.
Investors@forgebiologics.com
Logo -
https://mma.prnewswire.com/media/1393865/Forge_Logo.jpg