Bayer’s gene and cell therapy pipeline aims to transform Parkinson’s, oncology, rare diseases and more

Scientists at Viralgen, a subsidiary of AskBio that pharma major Bayer acquired in 2020, are seeking a cure through cell and gene therapies (CGT) for diseases affecting a large number of people.

While Parkinson’s disease is the most advanced program in Bayer’s CGT pipeline, oncology, cardiovascular and rare diseases are the other priority diseases they are seeking to find a way to cure.

“Like every new field, you get your hits and misses. But I would say that the hits are meaningful. I would not say that a cell and gene remain just a promise that has not been delivered. I would say that it is true that the hits are limited, and you can count them, but I think it also is very clear that there is an upward curve and we will see more and more therapy here, with very significant impact,” said Gustavo Pesquin, CEO of Asklepios Biopharmaceutical Inc (AskBio), Bayer’s wholly owned, independently operated subsidiary.

“We work relentlessly to reshape what’s possible for those impacted by the severe diagnosis,” he said at Bayer’s annual APAC media round table at San Sebastián, Spain.

After the first investments, Bayer’s drug development pipeline in cell and gene therapies has grown and comprises several programs in preclinical, early, and advanced clinical stages. The key research areas include neurology, rare diseases, ophthalmology, and cardiovascular diseases.

Scientists are working in the heart of San Sebastian’s technology park. The manufacturing facility makes Viralgen, the largest gene therapy manufacturer in Europe.“One day we will be helping many patients cure their diseases. This is different. This is impactful. And I really look forward to that day. This is what we want to do, but at the same time, bring it and bring it fast to all the markets and patients as much as possible,” said Ashraf Al-Ouf, head of commercial operations, Asia Pacific Bayer Pharmaceuticals Division.Bayer is advancing progress for patients with Parkinson’s disease, being the first company with both investigational cell as well as gene therapies.

In 2019, a girl named Irai was treated by complex gene therapy operation, the first of its kind in the world. From that moment, her brain has been able to create the neurotransmitters needed to restore the connection between her brain and body. Only 20 months after the surgery, Irai began to walk. This past winter, Irai even managed to go skiing.

According to Pesquin, Parkinson was a very “appealing” disease to generate the gene therapy.

“The probability that gene therapy could create a completely breakthrough therapy is very high. It is a disease that manifests itself in the mid-50s, early 60s. So clearly, aging population increases the prevalence of the disease, and we have seen the prevalence quite consistent across the world. So, we see that the therapeutic value that this can bring for the Asian countries, Australia, and New Zealand would be very high,” he said.

Bayer continues to expand its conventional oncology portfolio, and CGT could ultimately reshape treatment paradigms.

“Of the approximately 7,000 identified rare diseases, only five percent have found a cure. Unfortunately, these rare diseases have affected around 400 million people worldwide. CGT will expand into cardiovascular, oncology, rare diseases and many other therapeutic areas over time,” said Jimmy Vanhove, CEO of Viralgen.

Bayer’s acquisitions of AskBio, BlueRock Therapeutics, Vividion Therapeutics, and other biotech ventures have laid the foundation for the CGT expansion.

Experts are hopeful that one day they will be helping many patients cure their diseases.

“And this is why we do what we do in this company. And this is why I wake up every morning thinking, “I want to help, that one day we will be helping many patients cure their diseases,” added Ashraf.