We are at the dawn of a golden age in medicine, driven by therapeutic biologics, including gene and cell therapies. At this 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 a severe rare disease.
Our clinical development plan paves the path towards discovering safe and effective treatments by enabling more options, while minimizing risk of dead ends — no matter what happens in each trial, there is a clear path forward. The current clinical development of muscle targeted gene therapies are hampered by dose-limiting toxicity, ineffective re-dosing, sub-optimal placebo control, and manufacturing uncertainties. Our technology addresses 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 will change patients’ lives.
We are ready to begin human trials on a disabling rare disease known as GNE myopathy (GNEM) by H001 gene therapy, designed to increase GNE enzyme activity within the arm and leg muscles. This approach increases the chance for effective treatment, reduces the risk of life-threatening side effects, and further paves the path towards developing curative gene therapies for GNEM and other muscle diseases. The patients are ready, willing, and able to participate.
When I first experienced symptoms of GNEM, formerly known as Hereditary Inclusion Body Myopathy (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 above all. 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