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Home / Business / Roche gladly reports to buy Spark Therapeutics, biotechnology behind the first FDA gene therapy

Roche gladly reports to buy Spark Therapeutics, biotechnology behind the first FDA gene therapy



Swiss drug giant, Roche, lives up to buying a biotech company behind the first federally approved gene therapy and the most expensive medicine in the United States, the Wall Street Journal reported on Saturday.

Called Spark Therapeutics, the Philadelphia-based biotechnology created a one-time treatment called Luxturna for a rare form of blindness. It currently costs $ 425,000 per eye, or $ 850,000 in total. That price tag makes it the most pricy medicine in North America.

The Roche-Spark deal could be announced as early as Monday, WSJ said at a sticker price of nearly $ 5 billion. From Friday's proximity, Spark had a market value of less than half of the amount.

Founded in 201

3, Spark pioneers research on a new class of treatments for a rare, genetic form of blindness called Leber's inborn amaurosis at Children's Hospital of Pennsylvania. In 2017, the US Food and Drug Administration approved Spark's one-time treatment for the condition, making it the first of such gene therapies to gain regulatory approval.

But gene therapies are tough to develop and even tougher to receive.

And although Spark announced earlier this month that it had sent 75 vials of its new fabric and generated $ 27 million in sales, the company generated less than $ 65 million in revenue last year, the WSJ reported, and reported a net loss of close. to $ 79 million.

For Roche, the deal is expected to be part of the Swiss pharma giant's expansion into treatments for hemophilia, a rare disorder where some blood does not stick as it should because it lacks blood clot proteins. Spark works on treatments for disorder, which is also a major potential source of growth for Roche.

Gene therapies are buzzy, but costly and difficult to obtain

Despite years of being spied on as having the potential to cure dozens of diseases, gene therapy remains difficult.
AP Photo / Eric Risberg

Spark's blindness treatment falls into a larger category of potentially revolutionary new drugs known as gene therapies. Despite decades being spied on as having the potential to cure dozens of diseases, the treatments are difficult to access. Few have been approved by federal regulators; Those who have can cost about $ 1 million to get and even more to develop.

Earlier this month, Spark said it had sent 75 vials of its new drug and generated $ 27 million in sales, according to the Philadelphia Business Journal. Nevertheless, the company generated less than $ 65 million in revenue last year, the WSJ reported, and sent a net loss of nearly $ 79 million.

The approach behind Spark's treatment – and all other gene therapies – involves modifying a person's DNA to solve the underlying cause of a hereditary disease. Doctors take a sample of some diseased cells, correct the errors in the code and return the corrected cells to the person's body. Over time, the fresh cells surpass the sick, and the disease disappears too well, thinking goes.

Read more: This Silicon Valley startup requires a future where gene therapies are as accessible as vaccines.

But developing the therapies and getting them to patients has proven a steep challenge. In addition to targeting rare diseases, patients must pay attention to the time taken to ensure that they work. In addition, they are expensive and are offered only on a small number of accredited facilities, according to a recent analysis by the IQVIA Institute of Human Data Science and Arm Foundation for Cell and Gene Medicine.

Having launched its gene therapy Luxturna, Spark released three different payment models to attract more patients to use it, Business Insider previously reported. They included paying for the treatment based on how well it works and paying for it over time.

Sparks also work on gene therapy for hemophilia, a lucrative growing area for Roche. Two years ago, Roche won the FDA approval for Hemlibra, treating a type of hemophilia and a substance expected to generate billions of dollars in annual sales.


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