MDA Invests 550K into San Diego Therapy Company

MDA Awards Locana $550K to Advance Potential RNA-targeting Therapy for Myotonic Dystrophy

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MDA Awards Locana $550K to Advance Potential RNA-targeting Therapy for Myotonic Dystrophy
 

The Muscular Dystrophy Association (MDA) has awarded Locana $550,000 through its MDA Venture Philanthropy (MVP) program to help advance the biotech company’s therapy platform for myotonic dystrophy (DM).

Specifically, the grant to the San Diego, California-based gene therapy company is aimed at furthering the development of its RNA-targeting technology.

“We’ve witnessed incredible innovation with the development of the first FDA-approved gene therapy for a neuromuscular disease,” Lynn O’Connor Vos, the MDA’s president and chief executive officer, said in a news release. She was speaking in reference to Zolgensma (AveXis and Novartis), approved May 24 by the U.S. Food and Drug Administration to treat spinal muscular atrophy.

“This gives us hope that novel approaches — such as Locana’s for designing highly specific RNA-targeting candidates — for treatment of myotonic dystrophy can address the significant unmet needs for patients who live with this genetic disease.”

The project’s chief investigator will be Ranjan Batra, Locana’s vice president of research and development.

“We appreciate the support of MDA to address this devastating disease,” said Jeffrey M. Ostrove, PhD, Locana chief executive officer. “DM is caused by expression of dysfunctional, repetitive RNA in diseased tissues, where application of Locana’s core RNA-targeting technology has been shown to have  potential for single-dose benefit, and could provide a long-lasting approach for patients.”

Estimated to affect one in 8,000 individuals globally, myotonic dystrophy is the most common adult-onset form of muscular dystrophy (MD) and has two types, both caused by genetic mutations. DM1 results from an abnormal expansion in a region of the DMPK gene. DM2 is caused by an expansion in the CNBP gene.

Locana is using its gene-editing CRISPR/Cas9 technology to target disorders, including DM and Huntington’s disease, caused by abnormally repeated genetic sequences.

With this monetary support, the MDA is hoping to build upon work detailed in proof-of-concept studies published the journal Cell in 2017. Those efforts involved researchers, including founding officers of Locana, that used CRISPR to edit RNA originating from mutations responsible for myotonic dystrophy. Scientists were able to successfully edit the faulty RNA in muscle cells of those with DM and related diseases, as well as reverse hallmark features of disease.

Producing RNA-targeting molecules could lead to enduring effects, the company said. That means the therapy might need to be administered just once.

The strategy is distinct from DNA-targeted approaches, as well as from nucleic acid-based RNA targeting. Its overarching goal is to produce a portfolio of treatments that address the primary cause of genetic diseases caused by the actions of dysfunctional RNA.

“Mutations in our DNA can cause disease, and our RNA-targeting technology platform allows us to precisely address these mutations,” Locana states on its website. “By targeting RNA, our approach avoids the risk of off-target effects in DNA.”

MVP, the MDA’s drug development program, is solely focused on funding the discovery and clinical application of treatments and cures for neuromuscular diseases.

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Myotonic Dystrophy Researcher Receives Award

https://news.illinois.edu/view/6367/787310

  • Images

    • The Barry M. Goldwater Scholarship and Excellence in Education Program was established by Congress in 1986 to honor Goldwater, who served 30 years in the U.S. Senate.

      The Barry M. Goldwater Scholarship and Excellence in Education Program was established by Congress in 1986 to honor Goldwater, who served 30 years in the U.S. Senate.

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  • Editor’s note: For more information, contact David Schug, National and International Scholarships Program director, 217-333-4710, email topscholars@illinois.edu

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New Info about the AMO Myotonic Dystrophy Drug

Here is some information from the Muscular Dystrophy Group in the UK about the AMO drug that will be tested in the USA. I may be difficult to read if so click here for the link to the full report and look on page 9-10 https://issuu.com/musculardystrophycampaign/docs/mduk_campaign_april_2019

 

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Cydan, Inc is using new business model to accelerate Myotonic Dystrophy drug

Developing a new drug to treat myotonic dystrophy is very expensive. It can take tens millions of dollars to make and test a new drug. Cydan a comapny has a new novel approach that spins off a new company and aquires capital n a novel way to help reduce the risk of this new drug development. For example Ionis Pharmaceuticals spents millions developing a drug that in fact they did not bring to market. A big loss this new framework might lead to more drug development for us! Below is a presentation on this technique

De-risking-Rare-Disease-Projects-Chris-Adams-Cydan-Development

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New drug, Tideglusib, approach moving to clinical trial shortly

There is a new compound that is being tested to see if it can help with congenital myotonic dystrophy. It is being tested for a number of applications including tooth repair and Alzheimer’s  and just might help with the congenital form of myotonic dsytrophy. this is a molecule being developed by AMO pharma.

Tideglusib (NP-12NP031112) is a potent, selective and irreversible[1] small molecule non-ATP-competitive glycogen synthase kinase 3 (GSK-3) inhibitor.

Potential applications[edit]

Tideglusib is under investigation for multiple applications:

  • Alzheimer’s disease and progressive supranuclear palsy. As of 2017 it was undergoing Phase IIa[2] and IIb clinical trials.[3][4][5][6] The first trial to be published (in English) was Phase IIand demonstrated that tideglusib was well tolerated, except for some moderate, asymptomatic, fully reversible increases in liver enzymes.[4]
  • Tooth repair mechanisms that promotes dentine reinforcement of a sponge structure until the sponge biodegrades, leaving a solid dentine structure. In 2016, the results of animal studies were reported in which 0.14 mm holes in mouse teeth were permanently filled.[7]
  • Tideglusib is being studied in Phase II clinical trials as a treatment for congenital/juvenile-onset myotonic muscular dystrophy type I.[8]

There is a clinical study that will be starting shortly and you might be able to participate when this trial opens. Click here for more information.

https://clinicaltrials.gov/ct2/show/NCT03692312?cond=Myotonic+Dystrophy%2C+Congenital&rank=1

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

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