More than 150 years after a Swiss chemist accidentally discovered DNA – the basic building block of life – scientists are on the cusp of what has been called a “gold rush of discovery,” with technological advances that could revolutionize human health care.
Experts believe it won’t be long before cell and gene therapies will be broadly available as part of the range of tools needed to treat human disease.
“We have a completely new opportunity to transform medicine,” says Dr. Jost Reinhard, head of the relatively new Cell and Gene Therapy Unit at Bayer. “It’s not just the technological standards; to us, it’s really an opportunity to change patients’ lives.”
Cell and gene science has advanced significantly over the last two to three decades, Dr. Reinhard says. Scientists have a much more complete understanding of the human genome and cell biology.
Combined with advances in interpreting and managing data and complex systems, the biotechnology sector has developed breakthrough technologies and Bayer expects to realize clinical application by the latter half of this decade, adds Dr. Reinhard, a doctor in pharmacy with broad experience in drug discovery.
Last December, Bayer launched a Cell and Gene Therapy platform within its pharmaceutical division, saying the field was growing at an unprecedented pace – and it was the 150-year-old company’s goal to be at the forefront.
Health Canada has already approved five gene therapies and, based on the number of clinical trials in progress, there could be 10 to 20 gene therapy products approved every year for the next few years, according to the Centre for Commercialization of Regenerative Medicine (CCRM).
These predictions underscore the progress made in cell and gene therapy, Dr. Reinhard says, adding that Bayer sees a big opportunity from these new technologies.
“We are moving forward from largely treating the symptoms of diseases towards the underlying issue causing the disease – and maybe, at some point in time, even curing the disease,” he says.
The potential includes rare genetic disorders as well as more common degenerative diseases, cancer and immune diseases.
Over the past couple of years, Bayer has acquired several biotechnology companies with promising therapies in development, including the full acquisition of BlueRock Therapeutics in 2019, which specializes in pluripotent stem cell-based therapies, and the 2020 acquisition of AskBio, with its focus on adeno-associated virus-based gene therapy.
Those companies continue to operate independently, Dr. Reinhard says, while Bayer focuses on developing its platform technologies.
The idea is that these subsidiaries can continue to focus autonomously on their innovations while Bayer brings the expertise and scale of a global organization.
“To further evolve these technologies, we are convinced we should really have them evolving as platforms,” Dr. Reinhard says. “That would allow us to learn across therapeutic areas in order to provide optimal treatment, but also in order to speed up the development.”
Bayer’s focus is across a range of diseases. There is exciting progress in the development of new treatments for Parkinson’s disease, cardiology, as well as monogenetic diseases such as Limb-girdle muscular dystrophy, he says.
Dr. Reinhard believes that these treatments could potentially be available to patients in the second half of this decade.
“A lot will depend, of course, on the technological questions that we come across,” he says. “We’re in early-stage development … but we aim to see the first therapies between 2026 and 2029.”
Dr. Reinhard says standard treatments of today will remain part of the medical arsenal and will continue to be developed, but cell and gene therapy have tremendous potential for new therapies to treat or at least stop the progression of a range of diseases.
“I think it will revolutionize medicine, to the degree that these therapies will become a part of the armamentarium,” he says. “They do provide the opportunity to prevent diseases and treat them completely differently and also to treat currently still non-tractable diseases.”
This new approach to patient care with potentially immeasurable benefits will require a total paradigm shift in the Canadian health care system, from pricing and reimbursement to regulatory review to a streamlined patient access strategy.
Is the entire industry and the various levels of government approval, provincially and nationally, ready to cooperate and are all parts of the system ready to work together to ensure Canadian patients have access to these transformative treatments, should they become available?
Dr. Reinhard believes so. In the future, he believes it could be possible for patients with heart failure to be treated with one of these emerging technologies, instead of being put on a waiting list to receive a heart transplant.
“In an ideal world, if everything comes to what we are dreaming about and what we are trying to bring forward, we could be able to treat patients who have exhausted the current therapeutic options,” he says. “We see these approaches providing hope to patients who had little hope before. It’s simply amazing.”
Regenerative medicine: Where will stem cells take us?
On Nov. 30, The Globe and Mail hosted a virtual event called Regenerative medicine: Where will stem cells take us? Presented by Bayer, the webinar explored the way researchers are working on stem cell advances that could change the future of medicine. Read more here.
Advertising feature produced by Globe Content Studio with Bayer. The Globe’s editorial department was not involved.