Final Tests Planned into Potential Gene Therapy for OPMD, Could Support Trial in Patients
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Three new animal-model tests of a potential gene therapy for oculopharyngeal muscular dystrophy (OPMD), known as BB-301, are planned, Benitec Biopharma announced. Results are expected to support a request for a Phase 1 clinical trial in patients.
OPMD results from a faulty PABPN1 gene, leading to a protein that can form insoluble clumps linked with muscle weakness.
BB-301 uses a DNA-directed RNA interference (ddRNAi) strategy. Delivered via a modified, harmless adeno-associated virus (AAV), the therapy is intended to “silence and replace” the mutant protein. It suppresses its production and provides a source of normal, or wild-type, protein to target cells.
A 2017 study showed that this approach significantly reduced the amount of PABPN1 aggregates, restored muscle strength and eased muscle fibrosis (scarring) in mice. Subsequent experiments in cells from OPMD patients confirmed these results.
The preclinical research, to be conducted in a canine disease model, will help to optimize methods of administration, confirm the efficiency of the AAV vector in introducing a healthy gene into key muscle cells, assess optimal dosing, and further characterize toxicological data necessary for regulatory filings and clinical trial design.
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According to the company, these three planned dog studies will support the submission of an Investigational New Drug (IND) application, an essential step in getting regulatory approval to start the trial. Pre-IND application meetings with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and Health Canada took place in 2017.
“Through our continued focus on … BB-301 for the treatment of [OPMD], our team has an unprecedented opportunity to develop a novel genetic medicine that could facilitate clinically meaningful patient benefit,” Jerel A. Banks, MD, PhD, Benitec’s executive chairman and CEO, said in a press release.
The preclinical studies will be done in partnership with a medicine and surgery team with several decades of experience in OPMD treatment, Benitec added.
The FDA granted orphan drug status to BB-301 as a potential OPMD treatment in January 2018. The EMA awarded it similar status in early 2017.
Rebecca
You have a TYPO in the title , this is about OPMD not OCMD
José Lopes, PhD
Thank you and please apologize, Rebecca. It will be fixed. Best regards.
Carol Forde
Umm...Its OPMD, not OCMD. And this has been FACT CHECKED? Scary!!!!
José Lopes, PhD
Thank you and please apologize, Carol. It will be fixed. Best regards.
Danny Walker
Please correct typo in link. It is not OCMD it is OPMD.
Thanks
José Lopes, PhD
Thank you and please apologize, Danny. It will be fixed. Best regards.
M
The article title includes “OCMD” instead of OPMD. What a perfect representation of how little attention there is on this rare form of MD. Or perhaps I missed something and OCMD is correct?
José Lopes, PhD
Thank you and please apologize, M. It will be fixed. Best regards.
Jennie and Tony Ortiz
My husband, age 79, suffers from OPMD, as do 3 of his sisters. We have 3 children and do not know yet which ones have OPMD.
His symptoms began to manifest themselves when he was about 55-60 years of age. He has had “sling” eye surgery to help him use his forehead muscles to control his eyelids, and we have made many changes to the way food is prepared so that he is able to eat well. In the last 5 years , there has been significant loss of muscle in his legs so that he has much difficulty with balance and walking. He tires so easily because of the lack to muscle when he ambulates.
We are ecstatic about the new developments in gene therapy, not only for him but for our children and grandchildren. We are willing to relocate anywhere temporarily or travel anywhere if we can participate in trials for OPMD treatments. Please let us know if there is any way we can contribute to the research information or any way Tony can participate in clinical trials.
Please DO NOT publish our name, phone number, or address, which we are enclosing. Please delete them before sharing our note.
Jennie Ortiz for Tony Ortiz
Richard Patenaude
This is somewhat amazing. While reviewing and searching for articles relating to OPMD, I read the comments posted on October 19, 2019 by Jeannie and Tony Ortiz. I also am 79 years old and was diagnosed with OPMD In my late 50’s. Like Tony, I am experiencing the same leg muscle loss and balance issues. After several eyelid surgery’s I also had “Sling” surgery which has helped to a limited degree. I had read about BB 301several months ago and attempted to contact someone at Benitec who I believe was named Jay (last name not remembered) possibly in Marketing. I did not receive a response. I had asked
about the possibility of participating in Clinical Trials of BB-301. I had been scheduled to
participate in the Clinical Trials of another Drug at the UCI Medical Center in California but
the trials had conclude at another facility before they began at UCI so our group was cancelled. (Can’t remember the name of the other Drug). All that said, I would like to become a candidate for any type of Clinical Trials of BB-301 if that option is available.
Anna canale for Francesco Losapio
My dad who is 70 started having symptoms in his early 50’s. All 5 of his siblings also have/had OPMD. Since onset he has lost most of his strength in his legs and has most recently been hospitalized for aspiration pneumonia. He is actively attending PT to keep whatever muscle he has to continue functioning. I would love for him to be considered for part of the human clinical trial. I am one of 3 of his children. We have not been tested but would love to see a treatment for all involved.
Samuel Telles
I’ve been living w OPMD for 6 years since age 69 & very disappointing my MDA neurologists never provide any news nor information about clinical trials. Nevertheless the less I don’t give up & continue my own ways of coping including regular PT sessions several times per week.
Please consider me for your clinical trials.
Thank you !!!
Samuel Telles
Continuation of my previous comments.
Due to severe choking when I eat because of my OPMD, this Thursday, (day after tomorrow ) I have surgery to avoid another near death event as I’ve had in recent months
Ive also had 6 surgeries to help keep my eyelids open also due to OPMD
I too was a candidate with BioBlast for the cabelleta clinical trials promised here in San Antonio but at last minute were canceled without any logical explanation.