We are at the precipice of incredible advances in medicine, driven by gene and cell therapies. At this moment in time, Orphina is as much a calling as a company. Our mission to preserve muscle health benefits all of humankind, while our immediate focus benefits our loved ones suffering from severely debilitating orphan disease.
Our clinical development plan paves the path towards discovering safe and effective treatments by enabling more dosing options, while minimizing the risk of dead-ends. No matter what happens in each trial, there is a clear path forward. Our technology will change patients’ lives. The current clinical development of muscle targeted gene therapies are hampered by dose-limiting toxicity, ineffective re-dosing, suboptimal placebo control, and manufacturing uncertainties. Our technology addresses all of these challenges, thereby reducing clinical trial duration and number of patients needed to reach meaningful outcomes. While the trials are ongoing, we will fiercely pursue our work on next generation technologies.
We are ready to begin clinical trials on a severely disabling orphan disease known as GNE myopathy (GNEM). Our technology is designed to increase GNE enzyme activity within the arm and leg muscles, while reducing risk to vital organs. This approach increases the chance for an effective treatment, reduces the risk of unwanted or life-threatening side effects, and further paves the path towards developing curative gene therapies for GNEM and other muscle diseases. The technology is ready for clinical trials, and patients are ready, willing, and able to participate.
When I first started experiencing symptoms of GNEM, formally known as Hereditary Inclusion Body Myopathies (HIBM), I realized that the best way to speed up the research is to dedicate my life to it. From the early days of patient advocacy, to the research and development of therapeutics, we remained tireless and passionate. Our relentless quest has led us to Orphina.
We are building a company that values rapid progress over profit margins. All of our work begins and ends with our patients. We are not only doctors, scientists, and patients, we are also fathers and mothers, brothers and sisters, sons and daughters, friends and classmates, colleagues, and stakeholders. We have witnessed the successes and failures in medicine, and we recognize that significant strides towards developing more effective treatments happen only when we all remain engaged and relentless in our pursuit.
Daniel Darvish, MD