Thorsten Stafforst found his big break at the worst possible time. In 2012, his team at the University of Tübingen in Germany discovered that by linking enzymes to engineered strands of RNA, they could change the sequences of messenger RNA molecules in cells. In essence, they could rewrite the genome’s instructions en route to making proteins.

Awareness days and months occur throughout the calendar year to generate a greater understanding about various diseases and help increase visibility about the unmet needs related to these conditions. For many advocacy groups, these activities drive awareness and highlight rare disease states or disorders with great unmet needs.

Dean Scott could hardly see two years ago, but the Spirit River, Alta., carpenter says an experimental drug helped him regain some of his vision and added colour to his life. Scott, 45, first noticed problems with his eyes when he was a child and lost his peripheral vision.

In August of last year, Alkahest reported the results from a six-month trial that saw 40 patients with mild to moderate Alzheimer’s disease infused with a proprietary human plasma “fraction”. It appeared to arrest their expected mental decline.

Frustrated with the slow pace of drug discovery and desperate to help their loved ones, untrained family members take the work into their own hands by starting biopharma companies. But few such companies have been successful.

Valneva’s vaccine, VLA15, encourages the body’s immune system to make antibodies to defend against the bacteria that cause Lyme disease. The FDA gave VLA15 fast-track designation in 2017, and the vaccine is currently in Phase 2 clinical studies.

In drug development, assets are often aimed at a specific disease target or pathway, similar to a bullet fired from a gun. The idea of going after one precise target though, may not be the best solution for treating the totality of diseases, though.