From curiosity to audacity

The promise of cell and gene therapy gets closer

Human cells under the microscope

Bayer is building bold partnerships to get closer to the promise of cell and gene therapy.

Remarkably, as drug makers race to stop a life-threatening virus, other viruses are leading to life-saving therapies. Though it may sound futuristic, non-pathogenic adeno-associated viruses (AAV) can shuttle functional genes into the body to treat, and potentially cure, a wide variety of genetic diseases. Bayer’s acquisition of Asklepios BioPharmaceutical (AskBio) in October solidified our goal to explore this exciting frontier, build a leading position in Cell and Gene Therapy (CGT), and lay the foundation for future partnerships in one of the most cutting-edge spaces of medical research today. 

 

The concept of AAV-mediated gene delivery was first discovered in 1965, and much of its early study was solely for scientific curiosity. It wasn’t until the 1980s when groundbreaking research led by AskBio’s Chief Scientific Officer and Co-founder, Dr. Jude Samulski, brought them closer to the “audacious goal” of erasing genetic diseases. Their gene therapy platform represents one of the most advanced in the industry today, with drug candidates being investigated for the treatment of neuromuscular, central nervous system, cardiovascular, and metabolic diseases. For patients with genetic disorders like Pompe disease, this approach holds the promise of surpassing symptom management and delivering regenerative, and even curative, therapies.

 

In acquiring AskBio, Bayer did not just purchase a company; we became stewards of Dr. Samulski’s life work, and that of CEO Sheila Mikhail and her team of gene-therapy pioneers. By preserving AskBio’s entrepreneurial culture and operational independence, these innovators can keep doing what they do best. With our global reach and translational expertise, Bayer can accelerate the development of urgently needed gene therapies and be a frontrunner in this field. This arms-length partnership model is truly unique, and we are proud of it. We took the same approach last year when Bayer acquired BlueRock Therapeutics, a trailblazing biotech company which uses induced pluripotent stem cells (iPSC) as a platform to manufacture and replace lost or damaged cells that the body needs to repair itself due to neurological, cardiovascular or immune disorders. 

 

BlueRock Therapeutics is also driven by an ambitious goal: to reverse the symptoms of devastating conditions like Parkinson’s disease – the fastest growing neurodegenerative disorder in the world. The company is an output of Leaps by Bayer, a venture capital springboard that invests in paradigm-shifting advances in the life sciences – breakthroughs that could change the world for the better. This acquisition was a major stride toward holding a leading position in cell therapy, and a testament to our passion for innovation, whether born in our own labs or others’. 

 

Finding cures requires enormous curiosity, audacity and teamwork. This is why we follow the science when we recognize opportunities to address diseases where no treatment or cure exist to make a true impact for patients. With every bold step, we move closer to this goal.   
 

Author
Dr. Marianne De Backer
Dr. Marianne De Backer
Head of Strategy and BD&L, Pharmaceuticals Division of Bayer AG