Wave Is Developing Two Drugs That Target a Common ALS Mutation

Wave Is Developing Two Drugs That Target a Common ALS Mutation

Wave Life Sciences is developing two ALS and frontotemporal dementia therapies that target a gene mutation common in both.

The therapies target mutations of the C9orf72 gene, Wave said in a business update.

“I am excited to announce our next development program targeting C9orf72 mutations in ALS and FTD,” Dr. Paul Bolno, the company’s president and CEO, said in a press release. “With the progression of our pipeline and advancement in our [therapy] platform capabilities, we are closer to potentially bringing new hope and precision medicines to patients living with serious, genetically defined diseases.”

C9orf72 mutations are involved in 30 to 40 percent of inherited ALS cases of amyotrophic lateral sclerosis, and are risk factors in non-inherited forms. Previous studies have also linked them to earlier onset of ALS and more aggressive disease progression.

The mutation of the gene stems from a DNA problem known as the GGGGCC six-letter string expansion. While healthy people have just 20 to 30 copies of this sequence in their genes, those with the mutation can have hundreds.

This leads to the production of abnormal RNA molecules and proteins that contribute to neurological toxicity in both ALS and frontotemporal dementia, or FTD. RNA plays an important role in transmitting gene information from DNA.

Wave can use its proprietary development platform to design nucleic acid-based therapies that target molecular mechanisms or specific genetic sequences.

The company hopes to start clinical trials of its new therapies’ ability to combat ALS by the end of 2018.

Wave is also working on treatments for other genetic-based diseases, including Huntington’s and Duchenne muscular dystrophy. And it is collaborating with Pfizer on therapies for liver diseases.

“Within our discovery engine, we continue to generate compelling data for new modalities [therapy approaches] such as single-stranded RNAi [RNA interference], as well as splice correction,” Bolno said. “We recently achieved two major milestones: transitioning to a clinical stage company, with the commencement of our two Phase 1b/2a trials in Huntington’s disease, PRECISION-HD1 [NCT03225833] and PRECISION-HD2 [NCT03225846]; and the opening of our new cGMP [current good manufacturing practice] manufacturing facility.”

15 comments

  1. Charlie says:

    ‘The company hopes to start clinical trials of its new therapies’ ability to combat ALS by the end of 2018.’

    Oh yeah ?

  2. Charlie says:

    If clinical trials can only start end-2018 then this announcement has more to do with elevation of share price levels than elevation of hope levels.

  3. Diane Riefenhauser says:

    I have been caring for my mother with ALS . Been to the best hospitals . Trust me there is absolutely is NO medical advocacy for ALS patients. Especially in the USA These emails mean nothing. USA is doing nothing to treat ALS patients. Doctors right you off and send you home with a wheel chair to die.
    All they do is say come in every 3mobths for an emg so we can tell you your legs don’t work anymore and charge Medicare thousands of dollars for UNECCESARY tests that lead to “Sorry we have NO treatment” .Where is the money and When is a mandatory compassionate release of trial treatments going to occur .People are dying. If ALS patients don’t qualify for expanded release of experimental treatments , then who the hell does!!!!Where are the politicians to stop doctors and organizations from sucking Medicare dry for not offering any treatment. Despicable!!

    • Steven says:

      I agree. It’s like how many ice bucket challenges do we have to do to make people rich and then they say” we have found a cure”

    • Nondier says:

      I agree,I lost my mom after 4 years fighting against ALS and that is very sad because you can’t do anything ,just tell her how much you love her.
      Sorry I know how do you feel

    • Butch says:

      So very true…going to the clinic every 3 months is very demoralizing…problem is not enough of us for the people with money to care about…..look what 100 million from ice bucket has opened up…just need more on research…the new treatments are only given to about 3 out of 100 of us.

    • Charlie says:

      The thing about Radicava is that it only helps those very recently diagnosed.
      If you have a sulphite allergy you have no chance of getting it prescribed.
      More acutely, if you have had symptoms for more than about 15 months, Radicava , like Riluzole, has no beneficial effect.
      Notwithstanding, Radicava is going to be prescribed by doctors for people who have had symptoms for well over a year. This will make the pharma and the doctor very well-off.However it offers false hope to those in the symptom population who have had ALS for some time.

      This is perhaps the cruellest false trail in that MT are saying they are responding to the whole PALS population for ‘something/anything.’

      Of course the patient who has been very recently diagnosed will take Radicava and may well feel they benefit greatly, but in many cases they will be PALS with slow-progression, so it will be hard to tell if Radicava helps them in any way at all.

  4. Charlie says:

    Radicava looked originally like it was going to be a wonder drug. Then… the small print later emerged which showed that it had a very limited usage and benefit.

    • Steven says:

      When do you think nurown, masitinib, or those Cytokinetic drugs to be approved by the FDA and available for PALS Charlie?

  5. Diane Rief says:

    Hello out there in ALS land . It’s time to call out the doctors and FDA for the hold out on treatments for a dying populations . The aids patients did it so can we.
    Instead of having walks and ice bucket challenges that have done nothing but made big pharma and doctors rich and patients running on the perpetual treadmill to nowhere but the grave. Maybe it’s time to stop the flow of money . Maybe that will get there attention. Maybe it’s time to roll out there on the White House lawn and refuse to leave until expanded access has been achieved from the FDA to all treatments still in trial. its time for a new campaign . People only react to what they see everyday.! That’s how advertising manipulates our behavior to react and respond.
    I for one don’t mind being the crazy lady of the day.

      • Diane Rief says:

        Working on it. Will respond soon . Collecting some much needed data, that’s not in the mainstream first to create a functional plan of action.
        Expecting an answer to an important question any day. Have filed a freedom of information act application. Expecting an answer any day in the mail before moving forward, because you are correct we do need a functional campaign for action now .

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