Passage Bio has just announced that they’ve received 115.5 million dollars to help advance their development of gene therapies. They will begin by using the funds to further five gene therapy projects which began at the University of Pennsylvania, one of their close partners. Two of these projects are very far along and Passage believes they will begin human clinical trials by early 2020. These are for a form of frontotemporal dementia and GM1 gangliosidosis. Additionally, with this funding, the company has the option to fund pre-clinical development of seven more gene therapy programs for rare diseases at UPenn. Basically, UPenn is responsible for all of their pre-clinical work. After this work is done, Passage completes the clinical trials and the commercialization.
Hopefully, with this new funding we will see all five of Passage’s current gene therapy investigations move to this commercialization phase in the near future.
The Evolution of Gene Therapy
Gene therapy was once seen as a crazy, far-fetched idea. But at this point numerous successful studies have indicated there is huge potential in this form of treatment. It is now backed by prestigious researchers, the FDA, and other medical authorities worldwide.
In essence, gene therapy modifies a virus to deliver healthy genes to a patient. These genes replace the faulty or missing genes caused by the patients disease. It’s such a unique concept because it targets the root of conditions as opposed to just treating their symptoms. This means it could potentially cure many illnesses, eliminating the need for lifelong therapies, and improving patients quality of life overall.
James Wilson, one of Passage Bio’s co-founders was a key player in the development of the adeno-associated virus (AAV) as a vector for delivering gene therapies. AAV vectors are now part of many gene therapy success stories. They are being used in gene therapy investigations for Spinal Muscular Atrophy and Hemophilia (both of which are nearing FDA approval). They are also currently being utilized in human clinical trials for Parkinson’s disease and Duchenne Muscular Dystrophy. As a result of the many successes like these, the FDA has actually had to specifically discuss how they are going to handle the influx of gene therapy applications.
Ultimately, the successful development of gene therapies could substantially change the lives of patients. It is an especially momentous investigation for those living with rare diseases as many of these individuals have few treatment options. The need is high, and companies like Passage are working to fill this gap in care as quickly as possible.
Of course none of it would be possible without collaboration and funds. Thankfully, Passage now has both. You can read more on this new funding and Passage’s recent work, specifically in GM1 gangliosidosis and frontotemporal dementia here.