The recent acquisitions of Spark Therapeutics and Nightstar Rx signals the race to acquire gene therapy delivery platforms is well underway.
In the aftermath of the purchases, the few remaining validated platforms left on the market are looking like attractive buys. With the leaders of the field quickly being snapped up, the valuations of other gene therapy companies rose on the release of the news.
Ripe for gene therapy acquisition
Biotech companies Bluebird Bio, Uniqure and Sangamo Therapeutics are targets for acquisition, in addition to the growing array of CRISPR-focused companies such as Editas Medicine and CRISPR Therapeutics.
Despite a small rally at the start of the year, the US biotech index still looks relatively cheap compared to the start of 2018.
Gene therapy is in vogue
In vivo gene therapy, the process of delivering genetic material into the body to cure disease has the potential to cure millions suffering from genetic conditions such as cystic fibrosis or muscular dystrophy. But the field is littered with failures: historically, delivery has proved to be the challenging roadblock to successful therapy.
Yet, the pharmaceutical world has been watching recent developments with interest. Renewed faith was rekindled by the Novartis acquisition of gene therapy company AveXis Therapeutics in April 2018 for $8.7 billion.
Suddenly, after years of looking like a risky proposition, gene therapy is now very much in vogue. Large pharmaceutical companies are racing to acquire the few validated delivery platforms under development – and many have a serious fear of missing out.
Gene therapy acquisitions: a potential sign of company value
In February, Swiss-based Roche acquired gene therapy biotech Spark Therapeutics for $4.8 billion. Late stage Haemophilia A and B candidates (SPK-8001 & SPK-9011) from Spark had proven successful in late-stage trials. Roche will use these to expand its offering in haemophilia and is likely to use the Spark core delivery platform for approaches that target the liver.
Success has also arrived for Nightstar Therapeutics, acquired by Biogen for $800 million in the last week. The Nightstar lead candidate, NSR-REP1 for choroideremia, had received Regenerative Medicine Advanced Therapy Designation by the US Food and Drug Administration and the company announced expansion into a Phase 2/3 study in November 2018. Biogen is likely to use the Nightstar platform in the development of other products in ophthalmology.
The winter is almost over – and now gene therapy looks set for spring.