Novatos Ultragenyx partners with New Englands Solid Biosciences to fight muscular dystrophy

The day before Muscular dystrophy association Telethon returns to television after a five-year hiatus, a Marin County biopharmaceutical company on Friday announced a critical collaboration with a New England life science company to lead its own attack on the insidious disease.

Novato-based Ultragenyx Pharmaceutical will partner with Solid Biosciences of Cambridge, Massachusetts, to develop and market gene therapies to fight Duchenne muscular dystrophy, one of the nine forms of the disease that targets young people boys.

Muscular dystrophy is a muscle disease caused by mutations in a person’s genes. Over time, muscle weakness decreases mobility, making daily tasks difficult. There is no known cure for Duchenne muscular dystrophy. Treatment aims to control symptoms to improve quality of life. Steroids can slow the loss of muscle strength, according to medlineplus.gov.

The Ultragenyxs He La cell line manufacturing platform will combine with Solid Biosciences’ microdystrophin construct to produce gene therapy. Think of the strategic partnership as the first providing the vehicle in terms of producing an efficient and affordable drug, and the second providing the engine.

We believe this fits well with a marriage of technology and manufacturing to help us develop a superior product, Mardi Dier, Ultragenyx CFO and executive vice president, told The Business Journal.

Terms of the collaboration involve an initial investment of $ 40 million by Ultragenyx in Solid Biosciences and up to $ 255 million in cumulative payments when the Cambridge-based company hits milestones, as well as tiered royalties for product releases. .

Ultragenyx estimates that a new gene therapy is at least a year before entering its clinic and more than five years after its discharge.

It takes years to develop these gene therapies as part of the (Federal Drug Administration) process. Side effects are a safety concern when changing a biological pathway in the body, Dier explained.

Still, Dier insists his company excels in the arena given its ability to manufacture drugs and guide them through the testing and approval process.

The price at this point is an unknown once the final product is launched.

Gene therapy can be effective but also expensive, but if we have a great compound delivered it will be received around the world, Dier said.

In business for a decade, Ultragenyx brings to market products that treat serious rare and ultra-rare genetic diseases. The biotechnology company now has 800 employees working on three continents in North and South America as well as in Europe.

Ultragenyx currently has three AAV gene therapies in clinical development, one involving Bayer with the intention of treating hemophilia A, which is a medical condition in which the blood’s ability to clot is severely reduced.

This is one of a series of deals the company has made, Dier said.

This latest collaboration with Solid Biosciences comes by chance just before the announcement of the third quarterly report on the results of the public company. Its second quarter ended June 30 showed net income of $ 25.3 million, compared to a net loss of $ 99.2 million in 2019.

The 2020 growth evidenced by total revenue of $ 61.7 million comes at a significant time for advancing the treatment of these rare diseases.

We believe we can build on our mutual strengths to develop a high-quality AAV-based treatment alternative for Duchenne (muscular dystrophy), said Emil D. Kakkis, founder and CEO of Ultragenyx. At one point, he worked at BioMarin based in San Rafael.

The commitment and track record can be music to the ears of Iian Ganot, Co-Founder, President and CEO of Solid Biosciences, whose personal journey has led to a lifetime of hard work.

Ganot was not available to comment on the Business Journal but provided a statement.

We believe (Ultragenyx) is the partner of choice for exploring new gene therapy opportunities for Duchenne patients, Ganot said.

The deal also drew applause from the Muscular Dystrophy Association.

This is great news. It solves many of the challenges that Solid (Biosciences) encountered in its approach to gene therapy, said Dr Sharon Hesterlee, director of research at MDA. This puts (the Cambridge company) back in the game.

Much progress has been made in tackling the disease, which is often noted by a teacher who notices that the boy is not jumping and not active like other children around the age of 5.

From 11 to 14 years old, teenage boys most often need a wheelchair. The disease is caused by a genetic mutation and becomes fatal when the diaphragm muscle breaks down, leading to cardiac arrest or respiratory failure. It occurs in one in 3,500 to 5,000 men born worldwide, according to Sarepta Therapeutics, one in two biotech companies with Pfizer that researches the rare disease.

Gene therapy could be a game-changer here, Hesterlee said.

In the meantime, the Hesterlees organization will stage the legendary MDA telethon Saturday live at 5 p.m. on Pluto TV, YouTube and the MDA Facebook page.

Funding raised through the benefit hosted by comedian Kevin Hart supports 150 MDA clinics and benefits research efforts to develop treatments and cures.