Four pharma companies are providing half of the investment required for ‘world’s largest genetics project’, in return for access to the genomic data generated.
Already in 2019, there have been a number of deals to pick up vector and plasmid manufacturers, such acquisitions are a sign of a rapidly developing market, one report suggests.
Vertex is set to acquire Semma, which is developing a stem cell curative treatment for type 1 diabetes and expects the deal to ‘rapidly advance’ its potential therapy.
Pfizer invests half a billion dollars to expand its gene therapy manufacturing facility in North Carolina and enhance its position to compete in the booming market.
AveXis and BIA Separations announce an agreement for the commercial purification process of SMA gene therapy drug Zolgensma and further gene therapy candidates.
CGT Catapult reports that employment has increased substantially, and production capacity has grown by 60% in the UK cell and gene therapy manufacturing industry.
Catalent Biologics works with Novartis to develop and manufacture its SMA gene therapy treatment and provide manufacturing capacity, as the treatment enters the market.
Vertex agrees to acquire Exonics and to expand its collaboration with CRISPR Therapeutics, which sees it invest $420m in upfront payments to develop its position in gene therapy.
Thermo Fisher and Scinogy partner to improve scalability and cost-effectiveness of cell and gene therapy manufacture through the addition of the former’s centrifuge.
The private equity firm Ampersand Capital has acquired Vibalogics with plans to increase development and manufacturing capabilities for complex viral products.
SGS’ completed the ₤8.7m expansion of its large molecule testing facility in Glasgow and made room to increase its existing biosafety service and PCR platform capabilities.
Novartis receives US FDA approval for its gene therapy, Zolgensma, which is a treatment for spinal muscular atrophy, and prices the drug at $2.1m per patient.
Novartis investigates the death of an infant in a gene therapy clinical trial to determine whether it was related to the treatment but does not anticipate an impact on FDA approval.
Bluebird bio opens its first wholly owned manufacturing facility to produce lentiviral vectors for the company’s investigational gene and cell therapies.
Thermo Fisher Scientific bolsters its gene therapy capabilities by acquiring Brammer Bio for $1.7bn, as the market becomes an ‘increasing focus’ for customers.
As technology has advanced, rare diseases have been met with therapies that target the cause of the condition, enabling the global market for orphan drugs to reach an expected $262bn by 2024.
Biogen agrees Nightstar acquisition for $800m to take on its pipeline of AAV treatments for retinal disorders, diversifyingy its pipeline in the rare disease market.
In addition to the $55m previously invested, Novartis will spend a further $60m in expanding AveXis’ site in North Carolina enabling the manufacture of multiple gene therapies simultaneously.
Lonza Pharma & Biotech established itself in Israel, with the opening of a collaborative hub aiming to advance manufacturing technology for cell and gene therapy commercialization.
A study published by graduate students at MIT showed that a synthetic mRNA-based ‘programming language’ can control protein expression in gene therapy.
Thermo Fisher opened a new business center and biorepository along with an expanded distribution center, to help support mounting demand for clinical trial supply chain logistics.
Sarepta Therapeutics signed a long-term, strategic manufacturing partnership with Paragon Bioservices to produce micro-dystrophin to grow its gene therapy program.
PTC Therapeutics will acquire Agilis Biotherapeutics to continue work on gene therapy for rare monogenic diseases affecting the central nervous system.
The US FDA has released draft guidance documents on gene therapy programmes, which “should help sponsors avoid expending time and resources on unproductive drug development efforts,” says expert.