Forge Biologics | Our Focus on Krabbe Disease and Genetic Disorders



Patients & Caregivers

Our Commitment

If someone you love has been diagnosed with Krabbe disease, know that we are working to safely, effectively, and expediently bringing healing to the Krabbe community. We are more than just a company; we are real people that care deeply about connecting with, listening to, and understanding the patients and families we serve. If you’d like to connect with us, simply reach out at medicalaffairs@forgebiologics.com. We’re here to listen, learn, and help however we can.



Patients & Caregivers

About Krabbe Disease


Background

  • Krabbe disease, also called globoid cell leukodystrophy, is a very rare genetic disease. It is estimated that one baby out of 100,000 to 250,000 births is born with Krabbe disease.
  • Krabbe disease affects both the brain and peripheral nerves throughout the body.
  • Healthy nerves have a protective coating called myelin. An important enzyme known as GALC is necessary to make myelin. People with Krabbe disease cannot produce enough GALC, so the myelin coating is gradually destroyed by psychosine, a toxic that accumulates in the absence of GALC, eventually damaging the nerves and brain.

Genetics

  • Krabbe disease is a recessive disorder, which means both parents must be a carrier. 2 faulty copies of the gene that causes Krabbe must be present for a person to have Krabbe disease; having only 1 copy makes the person a carrier. Individuals affected with Krabbe disease inherited 1 faulty gene from each parent.

Symptoms & Subgroups

  • Early symptoms of infantile Krabbe disease can include irritability, difficulty feeding, delayed developmental milestones (such as rolling, crawling, walking, and speech development), difficulty swallowing and loss of muscle tone. As the disease progresses, children may experience cognitive impairment, seizures, vision and hearing loss, and breathing.
  • Krabbe disease is typically divided into four subgroups based on age at symptom onset 8:
    • Infantile (0–12 months)
    • Late Infantile (12-36 months)
    • Juvenile (37 months–16 years)
    • Adult (> 16 years)1

Patients & Caregivers

Current Standard of Care


The current standard of care for Infantile Krabbe Disease and Late Infantile Krabbe Disease is Hematopoietic Stem Cell Transplantation (HSCT), a complex procedure that can prolong survival into the second decade of life. Various studies have shown that patients do better later in life the sooner they undergo transplant. 6,7

Currently, the process of HSCT requires chemotherapy to kill the cells in the bone marrow and create space for the cells given from the transplant donor. The cells are given intravenously. After the transplant, the child will need

immunosuppression, lowering of a patient’s immune system, so that their body is prepared to receive healthy cells.

Transplant physicians will use a combination of strong medications, which can have negative effects on certain organs in the body. Recently, a reduced intensity conditioning approach has been developed*; it uses specific medications at lower doses to ensure that that the transplant is successful but without many of the harmful side-effects.

HSCT has been shown to preserve cognition, speech, hearing, and vision but continued weakness still occurs. Motor skills eventually deteriorate because the transplant does not address disease progression in the peripheral nerves. The continued weakness leads to inability to perform activities of daily living and a dependence on adaptive equipment and a wheelchair.

There is a small window of opportunity to prevent damage caused by psychosine due to the rapid growth of the brain and myelination in the first year of life. FBX101 was designed to be given after the standard of care, to address the treatment of the peripheral nerves that HSCT is unable to correct.


Patients & Caregivers

Our Clinical Trials


FBX-101: RESKUE Trial

Phase 1/2 Clinical Trial

The RESKUE study is researching an investigational* therapy for infants with infantile Krabbe disease. There is currently no cure, but trials like the RESKUE study are being conducted to learn more about the disease and discover new ways to manage it.

The study therapy is designed to provide a working copy of the GALC gene to your child’s body. This is done using something called an adeno-associated virus (AAV) vector. This works as a container for the gene therapy, which is delivered into the body through a one-time intravenous (IV) infusion. Once inside the body, the GALC gene is designed to help with the progression and symptoms of the disease.

Visit our clinicaltrials.gov site here.


RESKUE FAQs


Children who meet the following criteria may qualify:
  • 12 months of age or younger
  • Have a diagnosis of infantile Krabbe disease
  • Are eligible to receive hematopoietic stem cell transplantation
  • Have not received any previous gene therapy
Note: additional criteria apply. To learn more about your child’s eligibility, contact our study clinic (link to: medicalaffairs@forgebiologics.com) or visit our clinicaltrials.gov site here.


Screening

Your child will receive tests to make sure they qualify for the study. These will include a review of their medical history, genetic tests, brain MRI, neurodevelopmental and other testing, and blood and urine tests.


IV Infusion

Your child will be admitted to the hospital to receive an intravenous infusion of study therapy.


Inpatient Follow-up

Your child will remain in the hospital for at least 2 days so the study team can monitor their health and provide care.


Outpatient Follow-up

Once the study team has confirmed your child can be discharged from the hospital, you will continue to visit the study clinic over the next 2 years. There will be 14 visits over this period, during which your child will receive tests and assessments to see how they are responding to the study therapy.


More information on the ongoing “RESKUE” clinical trial can be found online at https://www.clinicaltrials.gov/ct2/show/NCT04693598.

Locations


FBX-101: REKLAIM Trial

Phase 1b Trial

The REKLAIM study is researching the investigational* therapy from FBX 101 RESKUE Trial to treat children with infantile and late infantile Krabbe disease.

The study therapy is designed to provide a working copy of the GALC gene to your child’s body. This is done using something called an adeno-associated virus (AAV) vector. This works as a container for the gene therapy, which is delivered into the body through a one-time intravenous (IV) infusion. Once inside the body, the GALC gene is designed to help with the progression and symptoms of the disease.

Visit our clinicaltrials.gov site here.


REKLAIM FAQs


Children who meet the following criteria may qualify:
  • 12 months of age or younger
  • Have a diagnosis of infantile Krabbe disease
  • Are eligible to receive hematopoietic stem cell transplantation
  • Have not received any previous gene therapy
OR
  • 12 - 47 months of age when they first showed symptoms
  • Have a diagnosis of late infantile Krabbe disease
  • Have received a hematopoietic stem cell transplantation
  • Have not received any previous gene therapy
Note: additional criteria apply. To learn more about your child’s eligibility, contact our study clinic (link to: medicalaffairs@forgebiologics.com) or visit our clinicaltrials.gov site here.


Screening

Your child will receive tests to make sure they qualify for the study. These will include a review of their medical history, genetic tests, brain MRI, neurodevelopmental and other testing, and blood and urine tests.


IV Infusion

Your child will be admitted to the hospital to receive an intravenous infusion of study therapy.


Inpatient Follow-up

Your child will remain in the hospital for at least 2 days so the study team can monitor their health and provide care.


Outpatient Follow-up

Once the study team has confirmed your child can be discharged from the hospital, you will continue to visit the study clinic over the next 2 years. There will be 14 visits over this period, during which your child will receive tests and assessments to see how they are responding to the study therapy.


More information on the ongoing “RESKUE” clinical trial can be found online at https://www.clinicaltrials.gov/ct2/show/NCT04693598.

Locations

*Investigational means the study therapy has not been approved for use by any regulatory agency.


Expanded Access Policy

We recognize the value of the Expanded Access program and are dedicated to patient-focused drug development. At this time, we are unable to commit to expanded access of FBX-101 for the general Krabbe patient population while safety assessments are ongoing. All requests for Expanded Access will be referred to study investigators to determine a patient’s eligibility for a clinical study. In very exceptional circumstances, Expanded Access requests may be considered on a case-by-case basis. These will only be considered in lieu of a patient who does not qualify for an ongoing clinical study and based on the potential benefit/risk profile of the experimental medicine for that specific individual patient based on existing safety and efficacy data.

For more information about our clinical trials or Expanded Access, patients, pediatricians, caregivers, and foundations are encouraged to reach out to . Physicians may also email to refer a patient to our clinical trials. A team member from Forge will respond within two business days.


Patients & Caregivers

Community Resources

To learn more & get connected


U.S. Based Foundations


Hunter’s Hope is committed to giving hope through education and awareness, research, and family care for Leukodystrophies and Newborn Screening for Every Child. Every time. Everywhere

Learn more about Hunter’s Hope here.

Krabbe Connect is dedicated to connecting patients, caregivers, healthcare providers, and researchers with the support and resources needed to optimize health, wellness, and advances in care.

Learn more about KrabbeConnect here.

ULF is focused on providing support to the leukodystrophy community and enable platforms to accelerate, improving patient quality of life, and finding cures.

Learn more about ULF here.


International Foundations


ELA International’s mission is to inform and support families, fund medical research on leukodystrophies, develop internationally by creating branches to bring together as many families as possible, and to raise public awareness. ELA International is present in 9 countries: Belgium, France, Germany, Italy, Luxembourg, Spain, Switzerland, the Indian Ocean, and Japan.

Learn more about ELA here.

Fundacion Lautaro te Necesita’s mission is to contribute to improving the quality of life of all those affected by a leukodystrophy, promoting education and research to optimize diagnosis time and accessibility to treatments.

Learn more about Fundacion Lautaro te Necesita here.

Krabbe UK is a charity registered in the UK, supporting families affected by Krabbe Leukodystrophy. Krabbe UK additionally supports the community by championing research and raising awareness of the disease.

Learn more about Krabbe UK here.

To share your story or learn more about our clinical trial, please reach out to our Manager of Patient Advocacy, Michelle Salvo - medicalaffairs@forgebiologics.com

References

1. Bascou, N., A. DeRenzo, M. D. Poe, and M. L. Escolar. 2018. 'A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life', Orphanet J Rare Dis, 13: 126.

2. Escolar, M. L., M. D. Poe, J. M. Provenzale, K. C. Richards, J. Allison, S. Wood, D. A. Wenger, D. Pietryga, D. Wall, M. Champagne, R. Morse, W. Krivit, and J. Kurtzberg. 2005. 'Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease', N Engl J Med, 352: 2069-81

3. Wright, M. D., M. D. Poe, A. DeRenzo, S. Haldal, and M. L. Escolar. 2017. 'Developmental outcomes of cord blood transplantation for Krabbe disease: A 15-year study', Neurology, 89: 1365-72.

4. Rafi MA, Rao HZ, Luzi P, et al. Long-term improvements in lifespan and pathology in CNS and PNS after BMT plus one intravenous injection of AAVrh10-GALC in twitcher mice. Mol Ther 2015; 23:1681–1690.

5. Yoon IC, Bascou NA, Poe MD, Szabolcs P, Escolar ML. Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease. Blood. 2021 Apr 1;137(13):1719-1730. doi: 10.1182/blood.2020005477. PMID: 33150395; PMCID: PMC8020262.

6. Escolar ML, Poe MD, Provenzale JM, Richards KC, Allison J, Wood S, Wenger DA, Pietryga D, Wall D, Champagne M, Morse R, Krivit W, Kurtzberg J. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med. 2005 May 19;352(20):2069-81. doi: 10.1056/NEJMoa042604. PMID: 15901860.

7. Vander Lugt MT, Chen X, Escolar ML, et al. Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders. Blood Adv. 2020;4(13):3041-3052. Blood Adv. 2020 Aug 11;4(15):3508. doi: 10.1182/bloodadvances.2020002967. Erratum for: Blood Adv. 2020 Jul 14;4(13):3041-3052. PMID: 32750127; PMCID: PMC7422113.

8. Bascou, et al., 2018. Note that exact ages and categories may vary based on definition of symptom onset.

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