Annalisa Jenkins joined Dimension Therapeutics, a small biotech based in Cambridge, MA, as CEO after leading global research and development at Merck Serono, a large German pharmaceutical company. This jump from big pharma culture to niche biotech gives Jenkins a unique perspective on the state of drug development. Dimension focuses primarily on gene therapies for rare diseases, which Jenkins says is in line with what she views as an evolution in the biopharma space towards patient-centric drug development.
This evolution towards personalized medicine requires understanding of the biology of individual patients and their disease to link to available therapies, and Jenkins believes that to enable this evolving approach, the voice of the patient must be brought into all aspects of drug development.
“As we’re thinking about understanding the biology of specific diseases, improving the way we understand disease epidemiology, all the way to how we design our clinical experiments, we need to make sure that patients have a voice in this process,” Jenkins said.
Jenkins points to the rare disease space — Dimension’s therapeutic focus — as an area where the patient’s voice can drive the development of new therapies. As companies seek therapeutics that match the unique needs of patients with rare disorders, the understanding of biology increased through novel technologies will lead to the evolution of how drugs are developed, Jenkins says.
The business model for rare disease development cannot rely on the peak sales of blockbuster products. Rather, Jenkins stresses the importance of building a portfolio of products that is supported by a platform that can optimize what is known about the disease, the therapeutic target and the technology used to attack these targets.
“This platform approach offers the opportunity, particularly in the rare disease space, to expand [a company’s portfolio] into different diseases and address the never-ending requirement to deliver growth,” Jenkins said.
For Dimension, the technology that underpins its business is an adeno-associated virus (AAV) platform, technology that has been investigated by the academic community over the last 30 years. AAV relies on the natural biology of a virus to carry the payload — a copy of a healthy gene to replace a mutated gene. Dimension recently announced drug development programs for two rare diseases — ornithine transcarbamylase deficiency (a genetic defect of the liver) and glycogen storage disease type Ia, which can lead to renal failure.
“I believe that gene therapy is really entering its second or third phase of evolution. … I think our understanding of the genome and the basic biology that underpins so many of these rare genetic disorders has unlocked enormous potential for new therapeutics,” Jenkins said.
The optimism of gene therapy ebbed and flowed since lab discoveries in the 1980s, but Jenkins notes that setbacks are an inevitable part of the biopharma industry. Now, Jenkins is optimistic about gene therapy’s potential for disorders of the liver, brain, eye and a number of other target organs.
Jenkins points to two main issues that will impact gene therapy’s ability to advance to the next step to deliver high-quality therapeutics.
First, and most importantly, companies will need to be able to prove they can deliver high-quality therapeutics on a consistent basis in a commercially viable model. Companies will need to demonstrate they are able to deliver therapeutics to target organs in a consistent way that results in predictable clinical outcomes. “I think a lot of progress in the last five to six years has really enabled acceleration in this space.”
Second, the clinical development profile and regulatory framework for gene therapies will require close collaboration between clinicians, patients and regulatory agencies.
“To a certain extent we’re all learnings together as we go,” Jenkins said, adding that stakeholders need to “enable everyone to feel comfortable that gene therapy truly will give us opportunities to address very severe diseases.”
