August 12, 2019

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Top 10 Companies Leveraging Gene Editing in 2019


Bayer made much of its desire to establish a cell therapy pipeline on August 8 when it announced it would shell out up to $600 million to acquire full control of BlueRock Therapeutics. But the deal is just the latest example of growing interest by biopharma giants in applying gene editing toward new treatments.

In explaining its CELL+GENE™ Platform on its website, BlueRock speaks of the “convergence of state-of-the-art technologies in cell biology and gene editing,” adding that its “advanced gene-editing capabilities further allow us to engineer these authentic cells to enable new therapeutic modalities.”

Among companies furthest along into gene editing-based therapeutics is Vertex Pharmaceuticals, which in June announced a $2 billion expansion of its presence in the specialty. Vertex is acquiring Exonics Therapeutics for approximately $1 billion, a deal expected to close later in the third quarter, and will spend an additional up-to $1.175 billion to expand a 3½-year-old, potentially $2.5-billion-plus collaboration with CRISPR Therapeutics that has resulted in the first clinical trial of a gene-editing therapy candidate sponsored by U.S. companies.

Biopharma’s apparent growing enthusiasm for gene editing is not shared by Wall Street investors, judging by the declines in stock prices shown by all four companies that have traded shares longer than a year. The extent of the drop-offs depends on the company. CRISPR Therapeutics shares have largely stayed flat, dipping just 1.3% year-over-year, to $48.00 at the close of trading on August 7, compared with $48.64 on August 7, 2018. The shares fell as far as $22.73 on December 21, 2018, before more than doubling over the past eight months.

Shares of Sangamo Therapeutics fell 13% (to $12.17) over the 12 months ending August 7, while Editas Medicine shares declined 15% (to $24.50), and Intellia Therapeutics shares tumbled 38% (to $17.42). Editas and Intellia, like CRISPR Therapeutics, reached 12-month lows on December 21, 2018 before bouncing back, while Sangamo hit its price low point of $7.72 on February 8, a day after the company released disappointing interim results from a pair of Phase I/II trials assessing two of its zinc-finger nuclease (ZFN) genome-editing candidates for two types of mucopolysaccharidosis (MPS).

Yet, venture investors and even some other biopharma giants appear much more optimistic about gene editing–based companies, given the year-over-year increases in total capital raised by many such startups, including those highlighted on this A-List.

The top five private companies together raised a combined $1.041 billion in total capital, up 72% from the $605.5 million raised by the same five companies last year. The top five public companies on this list generated a combined $228.992 million in revenues, up 43% from $159.892 by the five companies ranked in the 2018 A-List (one of which has since been supplanted by a newly public company that jumped from last year’s private company list).

Below is a list of the top 10 companies focused on gene developing and/or applying gene-editing technologies—five public companies, and five private companies. The public companies are ranked by their 2018 revenues, whether from sales of products or services, or from collaborations and R&D activity. The private companies are ranked by the total capital they have raised, as disclosed in press statements. Each company is listed with a short explanation of their recent activity.

The most notable company missing from this year’s A-List is CRISPR Therapeutics, since its reported 2018 revenue (all of it from collaborations) only amounted to $3.124 million, well below the $40.997 million reported in 2017, due to a decline in revenue recognized from the Vertex collaboration ($0.6 million versus $36.2 million in 2017). The 2017 revenue figure included $30.3 million from Vertex toward development of hemoglobinopathy targets, as well as R&D service revenue.

 

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[Originally posted by GEN — August 12, 2019]