Destroy Duchenne selects DNA Nanobots as Partner to Develop Non-Viral Gene Delivery Solution for Duchenne Muscular Dystrophy

 

 

DMD patient and advocate Elijah Stacy’s non-profit foundation chooses a targeted dystrophin gene therapy approach using DNA nanoparticles to overcome limitations of viral delivery systems

 

NORCO, Calif. & COLUMBUS, Ohio--(BUSINESS WIRE)-- Destroy Duchenne, a nonprofit organization founded by DMD patient, Elijah Stacy that aims to Complete the CureTM for Duchenne Muscular Dystrophy (DMD), and DNA Nanobots, a biotechnology leader in DNA nanoparticles engineered for targeted therapeutics, today announced a partnership to develop new targeted non-viral gene therapy technologies for DMD.

Duchenne Muscular Dystrophy (DMD) is a serious progressive, fatal genetic muscle wasting disease that impacts 1 in 3,500 almost exclusively boys and over 300,000 worldwide. DMD is incurable and caused by mutations in the dystrophin gene that affects the ability to hold muscles together, leading to muscle deterioration, paralysis, heart and lung failure and early death. Current treatment options for DMD include steroid medications to maintain muscle strength, physical and occupational therapy, surgery, and viral gene therapy. Last year, the FDA approved Sarepta Therapeutics’ Elevidys, the first adeno associated virus (AAV) gene therapy for the treatment of DMD for patients ages 4 and 5 as a single, intravenous dose of a shortened segment of the dystrophin gene. However, significant limitations remain including immunogenicity against the AAV vector, off target toxicity, single dose administration, and tremendously high cost (one-time treatment of $3.2 million).

Destroy Duchenne, a 501(c)3 non-profit organization located in Norco, California was founded by DMD patient and advocate, Elijah Stacy in 2017 to cure his own disease. Their mission is to Complete the CureTM for DMD by advancing gene therapy and gene-editing approaches and techniques into the clinic to save the lives of young boys suffering from DMD. Elijah works with several biopharmaceutical companies and was intrigued by the targeted, non-viral full length dystrophin gene delivery approach of DNA Nanobots presented to him by DNA Nanobots Co-Founder and Chief Science Officer, Dr. Christopher Lucas.

“I am extremely encouraged by the promising targeted non-viral gene delivery approach that DNA Nanobots uses. I am thrilled to announce and support our partnership with DNA Nanobots to potentially cure my disease,” said Stacy.

“We are very excited to announce our partnership with Destroy Duchenne and Elijah Stacy, who is tremendously helpful to us in understanding DMD and limitations of current treatment options. It is truly humbling to partner with an individual that is fighting tirelessly to cure his own disease,” Lucas said.

“Our targeted, non-viral gene delivery approach has several key advantages over viral gene delivery systems including cost of manufacturing, particle uniformity, biocompatibility, muscle-specific targeting, minimal immunogenicity allowing for re-dosing, and delivery of full-length dystrophin, a cDNA of about 14 kb, which is too large for AAV systems,” said Lucas. Visit www.dnananobots.com to learn more about our BioPharma Partner Program.

“Their technology is not limited by gene size and so the entire full-length dystrophin coding sequence with multiple antibodies for tissue targeting can be made into a precision gene delivery system. This could be a game changing treatment option to specifically target satellite stem cells to ultimately cure the disease,” said Stacy.

About Destroy Duchenne

Destroy Duchenne is a 501(c)(3) nonprofit organization founded by Elijah Stacy to cure DMD. Its mission is to Complete the CureTM for DMD by advancing gene therapy and gene-editing technologies into human practice. See www.destroyduchenne.org or contact them at email@destroyduchenne.org.

About DNA Nanobots

DNA Nanobots is a platform biotechnology company pioneering customizable DNA nanoparticles to address targeted drug delivery. We engineer custom tissue-targeted solutions for partners to deliver a variety of therapeutics, while developing our own pipeline for cancer and rare diseases. For more information on how DNA Nanobots is revolutionizing therapeutics development and gene delivery, visit https://dnananobots.com. Potential investors should contact investor@dnananobots.com.

Contacts

Jeff Spitzner, CEO
DNA Nanobots
(614) 325-2103
jspitzner@dnananobots.com

 
 

Source: DNA Nanobots

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