MedImmune, 4D Molecular Therapeutics to collaborate on gene therapy

AstraZeneca subsidiary and California-based firm to develop AAV-based therapy for chronic lung disease.

Drugmaker AstraZeneca’s biologics subsidiary is partnering with a company specialized in adeno-associated virus (AAV)-based gene therapies to develop a treatment for chronic lung disease.

MedImmune and Emeryville, California-based 4D Molecular Therapeutics announced the development and commercialization partnership Tuesday, using 4DMT’s vector discovery and engineering, optimization and process development technology, while MedImmune conducts product development starting from early clinical stages.

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AAV technology forms the basis of many of the gene therapies currently in development, as well as the first to receive Food and Drug Administration approval, Spark Therapeutics’ Luxturna, for a rare form of blindness. Spark’s other two leading therapies, SPK-8011 and SPK-9001 – respectively for hemophilia A and B – are also AAV-based, as is competitor BioMarin’s hemophilia A treatment, BMN 270. Meanwhile, bluebird bio is developing LentiGlobin, a lentiviral vector-based therapy for sickle cell disease and beta-thalassemia.

In addition to the partnership with MedImmune, 4DMT has a similar partnerships with Pfizer, announced in January 2016, focused on AAV-based gene therapy for heart disease. Another partnership, with Roche and dating back to 2015, involves the therapy 4D-110, under development for choroideremia. Spark is also developing an AAV-based choroideremia gene therapy, SPK-7001, which is in a 15-patient Phase I/II clinical trial.

All of 4DMT’s product candidates remain in preclinical development, with 4D-110 listed on its pipeline page as a candidate for an IND, or Investigational New Drug application with the FDA, which would enable clinical testing to begin. In April, the company said that it and Roche would expand the research agreement into a long-term development and commercialization partnership focused on ophthalmology, noting that IND-enabling studies for 4D-110 are underway.

Gene therapy has become a hot topic in medicine, including being the subject of a panel Wednesday at MedCity’s CONVERGE conference, currently underway in Philadelphia. Investment in gene therapy reached $4.5 billion in 2017, constituting a 164 percent increase over 2016, according to report released in March by the Alliance for Regenerative Medicine. A report in March by Market Reports Center forecast that the global market for gene therapy would reach $363 million by 2022. However, it defined gene therapy broadly to include Novartis’ Kymriah and Gilead Sciences’ Yescarta, which are CAR-T cell therapies for blood cancers, and Amgen’s Imlygic, an oncolytic viral therapy for melanoma.

Photo: Natali_Mis, Getty Images

CORRECTION: An earlier version of this story’s subhead misidentified the disease target as chronic liver disease. The error has been fixed.