Today, biomedical researchers have more tools than ever to fuel their investigations. These tools are facilitating progress at an astounding rate. They are also pushing researchers to delve deeper into specific, population-based issues, such as women’s health or aging.
One company working to tackle some of the diseases associated with aging is Gordian Biotechnology in San Francisco. The founders behind Gordian recognized that many of the diseases with the highest morbidities are age-dependent. Further, many of these conditions lack effective treatments. Diseases related to age involve the complex interplay between cells and physiological environment, yet much of the effects of such an environment remain poorly understood.
To address this issue, Gordian has created a unique discovery platform. It is based on gene therapies, many of which have the potential to address complex diseases related to aging. Most people associate gene therapy with the treatment of rare monogenic diseases. These diseases are generally present from birth and treatment occurs shortly thereafter.
However, gene therapy has the potential to be used to treat chronic and complex diseases that develop over the course of decades. Developing therapies for these conditions is currently extremely difficult. As we age, our physiological functions declines. This shift in physiologic function can interfere with the mechanisms of action of various drugs.
For example, some drugs become less effective as we age and lose the receptors necessary to process them. In addition, aging affects the ability of individual organs to deal with the side effects of drugs, so these side effects can become exaggerated.
The Difficulties in Treating Age-Related Chronic Disease Processes
While these individual hurdles are able to be addressed in clinical trials, the unfortunate truth is that aging is heterogenous, and creating effective studies is nearly impossible. To study the effects of a therapy, trial candidates would need to be 65 or older and in general good health with the exception of the one chronic disease being targeted.
In reality, the majority of individuals 65 or older have two or more chronic diseases and these conditions are often distinct. Sometimes, treating one disease can worsen another, or even cause a secondary problem. For these reasons, treating chronic diseases related to aging is exceedingly difficult.
At the same time, treating these chronic diseases is becoming even more important. The global population continues to age and the number of individuals 65 or older is increasing. Treating chronic health issues is key to reducing burden on health systems and minimizing morbidity.
Also, drugs are getting introduced to the market at an increasing rate. Understanding interactions is important for populations made up of individuals who are older and who are therefore at risk of significant side effects. Preventative care can eliminate the need for some drugs to reduce side effects and keep people healthier overall.
The key to prevention may lie in gene therapy. Interventions can be targeted to specific issues that arise in distinct parts of the body. This approach is precision medicine that targets the genes most likely to cause chronic issues or those that have already initiated chronic processes.
Much work remains to be done when it comes to using gene therapy for chronic diseases related to aging. While targeting the specific genes is easier than ever before, many questions need to be answered, such as how much of the therapy needs to be delivered and which technique is most efficacious. The therapies need to be effective without completely eliminating the function of a particular gene.
Questions also remain around dosing frequency and how gene therapies for multiple illnesses will interact with each other. In addition, researchers will need to scale production of these therapies so that they are affordable and accessible. Work is being done to address all of these issues, and Gordian is playing a big role in finding solutions. Gordian is helping target specific dysfunctions within the body to restore health.
Gordian’s Scalable Research Platform Drive Development
The work being completed by Gordian has already generated considerable excitement regarding potential future applications. Last year, the company won the Astellas Venture Management Gold Ticket Competition, which supports early-stage companies focusing on pre-clinical scientific discovery. The unique Gordian platform focuses on in vivo therapeutic testing as the first step in pre-clinical development rather than the last.
As mentioned above, physiological responses change as individuals age, so the industry’s approach to therapeutic testing should also change. In vivo testing means that efficacy data is available in real-time and is directly applicable to patient health. With this approach, the company can have efficacy data on thousands of different genetic therapies in a matter of months.
The Gordian platform is scalable, which means the development of novel therapeutic targets can happen very quickly. Fast-tracking the pre-clinical development of these therapies ultimately means getting solutions to patients who need them. Currently, the company is partnering with other startups that can use the platform to generate additional data and move closer to answering these key questions and developing effective therapies for the chronic diseases of aging.
