One of the most tragic inherited red blood cell disorders, to which mostly children are vulnerable, is sickle cell anemia — a condition in which there aren’t enough healthy red blood cells to carry adequate oxygen throughout your body (Mayo Clinic). With an increase in collaboration in drug research, the odds are increasing to bringing more therapeutic drugs to market. With this disease, the shape of blood cells’ changes to that of a “sickle,” an immune basis for malaria protection, as firmly established through a number of clinical field studies from different parts of Africa.
On Sunday, October 16, 2016, an article appeared in the Los Angeles Times – A new path toward curing sickle cell disease” lauding research on a “revolutionary” gene-editing technology that is beginning to “be realized in experiments aimed at curing sickle cell disease.”
The article explained the process of identifying the disease’s effects, the drawbacks to such present therapies as bone marrow transplant, both painful and dangerous, and it traced the progression of research trials.
It was revealed at the end of the article, however, that it will be years of “very careful and rigorous study to ensure that this gene-editing technology is totally and completely safe before we even contemplate an actual clinical trial,” according to Mark DeWitt, a postdoctoral fellow at UC Berkeley’s Innovative Genomics Initiative.
Here’s the thing: Instead of isolated research groups studying sickle cell disease, what if thousands more research scientists around the world were motivated to come together to research and develop therapies for this and other lethal diseases? It would certainly make sense that with more people working on the problem, solutions could come faster.
This is exactly what Open Therapeutics’ vision is for its platform, debuting in 2017, that will bring scientists together from around the globe to research and develop new drugs and therapies for both common and rare diseases. Open Therapeutics is providing an open environment where biotechnology researchers freely adopt and submit pharma techs; and crowd-sourced and collaboratively developed manuscripts. It’s no mystery as this why this is such an appealing concept.
For one thing, scientists will no longer have to work in a “silo” environment, confined by non-disclosure agreements they’ve had to sign. Instead, they’ll be able to share research and take credit for their work. Open Therapeutics’ platform concept emulates the Red Hat business model, which provides the freedom to see the code, to learn from it, to ask questions and offer improvements. Open source refers to something people can modify and share because its design is publicly accessible.
Treating Biologics using the same business model, Open Therapeutics’ platform will enable bio researchers around the world to openly exchange information on the platform without charge and based upon open sourcing, that can lead to acceleration of research results. The platform will permit researchers to publish their findings in prestigious publications around the world. The concept of open sourcing in the biotech industry has recently been supported by Bill gates – Gates Foundation – and separately by Vice President Joe Biden – Moonshot.
This is collaboration at its best and by the very numbers of scientists and researchers who can come together in an open source community, the possibility is more expedient research and development of prescription drugs and therapies for some of the world’s most formidable diseases and disorders.