8 Biopharma Trends and Top Startups to Follow in 2017

1. Gene editing: CRISPR/Cas9

Being one of the brightest scientific breakthroughs in years, a new targeted genome editing technology based on the CRISPR-Cas9 system, offers a huge promise for the future of medicine.  

CRISPR (clustered regularly interspaced short palindromic repeats) refers to the system consisting of two key molecules: an enzyme Cas9 -- “molecular scissors” able to cut the two strands of DNA at a specific location in the genome; and a piece of pre-designed RNA sequence -- “guiding” RNA (gRNA), within a longer RNA scaffold. The role of the latter is to direct Cas9 “scissors” to the exact location in the DNA of interest and modify it as desired.

While CRISPR technology is still in its infancy with a number of serious issues to be addressed on the way, the discovery of the CRISPR/Cas9 gene editing approach has already transformed the biomedical research field forever. Compared to its “predecessors”, such as zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), the CRISPR-Cas9 system is simpler, preciser, and relatively cheaper, making it an ideal vehicle for “playing with genome”. And it can be done in high-throughput screening formats.

With all the benefits CRISPR technology represents no wonder it has captured the attention of the popular media, the scientific community and top biotech investors. Over the last several years the number of Google search requests and research citations related to CRISPR have skyrocketed almost exponentially.

Some of the most well-established biotech startups in the field of CRISPR include:

Intellia Therapeutics: (IPO in 2016, highest post-money valuation ever for a pre-clinical biotech startup).  Advances programs for in vivo and ex vivo therapies, the latter including HSC transplants and CARTs.

Editas Medicine (IPO in 2016) develops programs to treat eye diseases, uses engineered T-cells (e.g., CARTs) among other technologies.

CRISPR Therapeutics (IPO in 2016) works on a broad range of diseases including blood disorders, congenital heart disease, blindness, and cystic fibrosis.

Eligo Bioscience: The startup’s advanced approach utilizes the idea of delivering CRISPR via phages to attack selectively nocive bacterial strains.

Other emerging players using CRISPR/Cas9 technology include: (neuromuscular diseases), (viral diseases), (multiple myeloma, prostate cancer and beta-thalassemia), , , and .