Innovation in medicine R&D is gearing up rapidly, with over 20 new technologies being added over the years and AI improving timelines, costs, and success rate.
We picked some eye-opening products from our portfolio companies that have been launched recently or delivered great results in clinical trials
Sickle cell disease
Crispr Therapeutics recently launched the one and done treatment, Casgevy, resolving the need for regular blood transfusions or a bone marrow transplant.
Wet macular degeneration
This very common eye disease affects tens of millions of people. It typically strikes when people grow older. With an average of 6 injections per eye per year, the current treatment is far from comfortable. 4D Molecular Therapeutics has shown that its one and done gene therapy resolves the need for these regular injections in 80% of the population.
Children that are born blind
Portfolio company MeiraGtx has shown that its gene therapy can change the course of the lives of children that are born blind. All 11 children treated in a clinical study so far gained sight, with some already being able to read and write.
The company also has a gene therapy in development to treat Parkinson’s disease, which is showing good results, equal to the latest innovation in treating Parkinson’s disease: an implant deep in the brain to stimulate parts of the brain. Meira’s therapy is less invasive and risky.
Brittle bone disease
For those people suffering from very brittle bones that break at a minor impact, Ultragenyx has developed an effective medicine that significantly reduces the number of fractures. This allows these people to lead a more normal life again.
Type-1 diabetes
Each of our portfolio companies, Vertex and Crispr Therapeutics, is developing a treatment to eliminate the regular glucose monitoring and insulin administration needed. After 12 months, people with type-1 diabetes that received the treatment so far do not need any use of insulin any longer. What a relief.
The examples above are just a small selection of the life-changing treatments developed by our portfolio companies. They are working on well over a hundred other diseases, from cardiovascular to cancer and from brain to muscle diseases.
We can’t wait to see these groundbreaking therapies reaching patients worldwide.
