A frequent impediment to rare disease research is cost. It’s unfortunate, but it’s the reality of the world we live in. That means every fundraising campaign and every dollar raised is incredibly important. It all works to accelerate our speed for finding cures.
Gene Therapy
Gene therapy is one of the latest developments in the field. Basically, it involves taking the patients cells, modifying them to improve the faulty gene (or replace it), and then putting them back in the patient’s body. In clinical trials, its proved it has incredible potential. Unfortunately, it’s very expensive.
However, Orchard Therapeutics just announced the results of a very successful fundraising campaign. Orchard is a biotechnology company that specializes in gene therapy. In June, they raised over 300 million dollars to invest in their research. Then, in a recent campaign, they raised another 225 million. This is great news for all those who may benefit from gene therapy.
Their Research
Specifically, Orchard focuses on severe combined Iimmunodeficiency (SCID) caused by adenosine deaminase deficiency. Children with this condition have a very compromised immune system and any infection, sickness, or even the common cold can be extremely dangerous for their well-being.
Gene therapy has potential to be life-changing for these types of patients.
Looking Forward
We now have around 5 million more dollars invested toward gene therapy. This is not only exciting for further developments in this field, it shows a commitment from researchers to focus more on rare diseases.
We can only hope that the more commitment we make to research, the faster patients living with rare conditions receive the treatments and therapies that they need. Thankfully, we’re making steps in the right direction. It’s an exciting time for the rare disease community.
You can read more about Orchard Therapeutics campaign and the progress of gene therapy research here.
