Vertex ramps up spending to support gene therapy manufacturing

By Nick Taylor

- Last updated on GMT

(Image: Getty/Iamnoonmai)
(Image: Getty/Iamnoonmai)

Related tags Vertex Gene therapy Stem cell Type 1 diabetes

Vertex Pharmaceuticals’ move into genetic therapies triggers a planned escalation of its manufacturing and research spending.

Across 2019, Vertex has struck deals intended to yield a new generation of breakthrough medicines.

In June, Vertex agreed to pay $245m (€220m) upfront to acquire Exonics Therapeutics​ for its gene editing technology and pipeline of programs targeting diseases including Duchenne muscular dystrophy (DMD). Months later, Vertex put up another $950m​ to buy Semma Therapeutics and its cell therapy treatment for type I diabetes.

The acquisitions moved Vertex, which started out in small molecules, into new areas, and building out capabilities in those areas will cost money.

In recent years, Vertex has grown its annual operating expenses by 10% to 14%. Talking on a recent quarterly results conference call, Vertex CFO Charles Wagner warned investors to expect costs to rise faster in 2020.

Wagner said, “Our current expectation is that the rate of growth will be somewhat higher in 2020 as we invest in research and preclinical manufacturing for selling genetic therapies in support of our programs in type I diabetes, DMD and other diseases​.”

The move into type I diabetes also takes Vertex into territory that, to some observers, looks different than the areas the company has targeted historically.

Asked by an analyst about the shift in focus, Vertex CEO Jeff Leiden downplayed the differences, noting that type I diabetes is treated in the US “in a relatively small number of centers​” that can be targeted by a speciality sales force.

Researchers have achieved positive, long-term outcomes by transplanting cadaveric islets into patients but two barriers have stopped companies from industrialising that approach.

Firstly, there are too few cadaveric islets to treat all type I diabetics. Secondly, immunosuppression is needed to stop patients from rejecting the transplanted cells.

Semma is trying to tackle the problems by differentiating stem cells and using a device to protect them from the immune system. Vertex thinks these technologies are the breakthroughs the field needs to industrialize the concept.

Leiden said, “We were watching companies who are addressing those two problems for the last two, three years. And over the last six to eight months, we were convinced that Semma has actually solved both of those problems​.”

Vertex reached that conclusion on the strength of preclinical data. Now, Vertex is set to invest to find out whether the idea works in the clinic.

Related news

Show more

Related products

Trends in Biopharmaceutical Raw Material Selection

Trends in Biopharmaceutical Raw Material Selection

Actylis – The Partner of Choice | 31-Jan-2023 | Business Advice

Join us as our in-house experts, along with Cecile Bellamy from Pfizer, discuss current and future trends in biopharmaceutical raw material selection.

Liposomal and Nanoparticle Technology

Liposomal and Nanoparticle Technology

Pfizer CentreOne | 18-Nov-2022 | Technical / White Paper

Medical science is continuously searching for better ways to administer drugs into the body and to maximize the therapeutic effectiveness of the drugs...

The Reagents Behind Much of Molecular Biology

The Reagents Behind Much of Molecular Biology

Thermo Fisher Scientific | 25-Jul-2022 | Case Study

The ability to amplify, modify and fabricate DNA is fundamental to modern molecular biology. Why do so many researchers take their constituent parts for...

Related suppliers

Follow us


View more