UPDATE FROM ABEONA

September 17, 2019

Abeona Therapeutics announce an open-label clinical trial assessing the safety of one-time gene therapy ABO-102 for patients with middle and advanced phases of MPS IIIA.

The official ClinicalTrials.gov listing for study ABT-003 in Patients With Middle and Advanced Phases of MPS IIIA Disease has been posted: https://bit.ly/2kkp9tA. The corporate website also has patient information about the study: https://bit.ly/2kIp9Ul.

July 9, 2019

Clinical Trials Accepting Eligible Children with Sanfilippo Syndrome type A and type B

Abeona Therapeutics Inc. is developing one-time gene therapies for Sanfilippo syndrome types A and B, also known as MPS IIIA and MPS IIIB. The Company is enrolling eligible children into two studies of these therapies, respectively – The Transpher A Study (for MPS IIIA) and The Transpher B Study (for MPS IIIB).

The best way to determine if your child may be eligible for one of these studies is to take a 6-question survey at AbeonaTrials.com. If the survey indicates your child may be eligible to enroll, you have the option of having the information sent to the nearest study site for evaluation by a study investigator – a doctor overseeing the clinical trial.

Importantly, only they can decide if your child may qualify for additional screening for the study and taking the survey does not mean a child is eligible.

If you agree to share the survey results, the study site will then contact you for more information. Survey results can also be downloaded to share with your child’s physician. Abeona does not have access to any patient information that is shared in this process.

If your child is enrolled in the study, reasonable costs of travel and meals needed by you and your child will be reimbursed, and there is no cost to you or your family to participate in the study.

If you think your child may be eligible or if you have questions about the study, email patients@abeonatherapeutics.com. You can also consult this FAQ.

Information for Your Child’s Doctor

Your child’s doctor can find more information about the studies at Clinicaltrials.gov using identifiers NCT02716246 and NCT03315182. Each study is evaluating the safety and efficacy of a single-dose gene therapy that delivers a functional copy of the defective SGSH or NAGLU gene. The therapies, ABO-102 and ABO-101, are administered through a single intravenous infusion of an AAV9-based vector for correction of the underlying enzymatic defect in each type of the disorder. Comprehensive information about each trial, including the full enrollment criteria, can be found at Clinicaltrials.gov.

 
Facebook Ad Campaign and Survey Aims to Help Enroll More Patients in Clinical Trials

Abeona recently launched a concerted effort to enroll more children into ongoing studies of its one-time gene therapies for MPSIIIA and MPSIIIB. The centerpiece is a Facebook advertising campaign and eligibility survey. The latter is intended to help caregivers determine if their child may be able to participate in one of the studies that are enrolling patients 6 months – 2 years of age. Children over 2 must have a development quotient of 60 or above to qualify. Full enrollment criteria is at Clinicaltrials.gov: MPSIIIA, MPSIIIB.

If the 6-question survey indicates a child may be eligible to enroll, the caregiver has the option of having the information sent to the nearest study site for evaluation by the investigator. The study site then engages the family for more information. Survey results can also be downloaded to share with a physician. Abeona does not have access to any patient information that is shared in this process.

Secondly, to bring more awareness to the studies and make them easier to decipher, Abeona has named them The Transpher A Study and The Transpher B Study. These names acknowledge the gene transfer method used by each therapy and the type of Sanfilippo syndrome they aim to treat.

More information about the therapies can be found on the company’s website or by contacting patients@abeonatherapeutics.com.

 
Abeona Therapeutics continues to show progress and bring hope to families. Its work is continually updated with full press releases here.