Sio Gene Therapies ( OTC:SIOX) stock is down 82% in the past 12 months, Generation Bio ( GBIO) is down 69%, and Voyager Therapeutics ( VYGR) stock is down 63% across the past five years. Within five years, the drug becomes more expensive than Zolgensma.ĭeveloping gene therapies is fraught with complex and difficult challenges, however, and most listed companies that have attempted to do so have ended up burning through investors cash in the clinic, without producing a drug that works. Spinraza, administered four times per year, every year (since it is not a "functional cure") costs $750k for the first year of treatment and $350k per annum after that. Compared to Biogen's ( BIIB) Spinraza, also indicated for SMA, however, Zolgensma can be considered cheap. Novartis' ( NVS) Zolgensma, for example, which is indicated to treat Spinal Muscular Atrophy ("SMA") in infants, comes with a >$2m price tag. That is also why approved gene therapies tend to be the most expensive on the market. a "one and done therapy," that after being administered a single time, cures the patient for life. The reason gene therapy is such an exciting field of research is that it offers the possibility of "functional cures" i.e. Despite these breakthrough successes, Bluebird's share price has sunk in value by >90% across the past five years. deals for Horizon include a non-exclusive license agreement with the Broad Institute for the CRISPR/Cas9 gene editing technology, and another non-exclusive license agreement with Stanford University for the AAV (Adeno-Associated Virus) gene delivery technology.Gene therapy is one of the most exciting and innovative fields of drug development research, although for biotech investors it's also one of the most risky places to park your money.Ĭonsider Bluebird Bio ( BLUE) for example, a company that won not one, but two approvals for gene therapies - Skysona, approved for the rare disease cerebral adrenoleukodystrophy ("CALD"), and Zynteglo, for the blood disorder beta thalassemia. In June, Horizon acquired CombinatoRx service business and assets from Zalicus, in turn adding several valuable services to its pre-clinical discovery portfolio, such as high-throughput screening, drug combination sciences, and scalable bioinformatics expertise. This is Cambridge-headquartered Horizon’s second acquisition of an American company in the past few months. The acquisition is expected to be completed on October 2, 2014. and EU sales forces, fortify its IP portfolio, and will provide significant additional revenue. Other assets and capabilities acquired from Sage include a custom engineered in vivo model development service, inventories of ready to use in vivo models, and molecularly annotated patient-derived xenograft (PDx) models, as well as custom development capabilities.ĭisley said the latest acquisition will also strengthen Horizon’s U.S. In acquiring Sage Labs (previously a unit within Sigma Aldrich), Horizon will gain exclusive access to ZFN for in vivo model generation and certain CRISPR in vivo-related intellectual property. The consolidated offering significantly strengthens Horizon commercially, allowing us to offer product, service, and R&D programs to customers engaged at every stage of translational genomics and personalized medicine research from sequence to treatment.” Darrin M Disley, CEO of Horizon Discovery Group, said, “The acquisition positions Horizon as the world-leader in gene-editing and at the forefront of technological advances in the field of translational genomics. Sage Labs also had total assets of $14.5 million at the beginning of this year and has benefitted from investments of over $25 million.ĭr. The acquisition will result in a fully integrated translational genomics platform, which will significantly expand Horizon’s product, service, and research base. The Sage Labs acquisition is an important one for the Horizon group, as Sage is a world leader in advanced in vivo transgenic disease models for preclinical research applications. Horizon Discovery Group is an international life science company that provides research tools to support gene-editing, translational genomics, and personalized medicines development. With this latest acquisition, Horizon is now the world’s leading gene-editing and translational genomics company. Louis for up to $48 million in cash and shares. U.K.-based Horizon Discovery has agreed to acquire Sage Labs in St.
0 Comments
Leave a Reply. |
AuthorWrite something about yourself. No need to be fancy, just an overview. ArchivesCategories |