Big Pharma closing in on Gene Therapy

The blog, Clinical Leader offers a good review of the state of the gene therapy field.
Sangamo BioSciences (SGMO) CEO is quoted with regards to regulatory requirements, but no mention of the clinical programs Sangamo is fielding. This is disappointing.With regards to Biogen the article refers to its Hemophilia work.
http://www.clinicalleader.com/doc/keeping-tabs-on-gene-therapy-big-pharma-is-closing-in-0001

Juno Therapeutics: In a Phase 1 clinical trial, 91 percent of pediatric patients taking Juno’s JCAR017 for relapsed/refractory CD19-positive acute lymphoblastic leukemia (ALL) achieved complete remission. In even more recent news, the company launched a research collaboration with Fate Therapeutics to bolster the therapeutic potential of Juno’s T-cell immunotherapies, as well as acquired Stage Cell Therapeutics. 

Novartis: One researcher from Novartis, in partnership with an expert from GenVec, is exploring a gene therapy treatment for patients with hearing loss, Bloomberg reported in February. These two researchers are attempting to regrow the sound-sensing hair cells within the ear that have been destroyed by excessive loud noise. A trial enrolling 45 U.S. patients received NIH approval and is currently underway, with results expected by 2017. In addition, the company made headlines at the beginning of 2015 for its deals with Intellia Therapeutics and Caribou Biosciences which will provide Novartis with CRISPR  (clustered regularly interspaced short palindromic repeats) technology to edit the genes of targeted cells and boost drug discovery efforts. Novartis is also currently in a collaboration with the University of Pennsylvania, which led to the development of CTL019 for pediatric and adult patients with r/r ALL. This treatment earned a BTD from the FDA last summer for this indication, and, in a clinical trial, led to complete remission of the disease in 36 of the 39 patients receiving the therapy.

Sanofi: In February, Sanofi’s subsidiary Genzyme launched an $845 million partnership with Voyager to collaborate on gene therapy programs in Parkinson’s disease (VY-AADC01), Friedreich’s ataxia (VY-FXN01), and Huntington’s disease (VY-HTT01). In particular, the two are working to accelerate the Parkinson’s candidate through Phase 1, as it promises to be able to treat those who don’t respond to standard-of-care levodopa and carbidopa. Voyager, with Sanofi’s help, is also planning preclinical programs for the other candidates. 

Spark Therapeutics: In January, the gene therapy company expanded its pipeline of inherited retinal dystrophy treatments by initiating a Phase 1/2 clinical trial of its SPK-CHM candidate for choroideremia, an inherited retinal dystrophy (IRD) affecting male children. The company’s lead candidate, SPK-RPE65, is currently in Phase 3 trials for IRDs.

Bristol-Myers Squibb (BMS): In April, BMS signed an agreement with UniQure for the rights to UniQure’s lead heart failure candidate, S100A1. According to the agreement, BMS will also gain the rights to three other UniQure candidates in the upcoming months.

Biogen Idec: Following in GSK’s footsteps, Biogen Idec has joined forces with Fondazione Telethon and Ospedale San Raffaele against hemophilia A and B. The partners will explore liver-targeting lentiviral gene transfer technology. This step followed the company’s decision to hire Olivier Danos as VP of its burgeoning gene therapy operation.

Pfizer: Adding to the pool of companies exploring hemophilia gene therapy treatments, Pfizer jumped aboard with Spark Therapeutics in December 2014 to accelerate development of the AAV vector SPK-FIX and launch Phase 1/2 clinical trials this year.

bluebird bio: In October 2014, the company launched a trial for its primary gene therapy candidate, LentiGlobin BB305, indicated for sickle cell disease. The first patient dosed was enrolled in the Phase 1/2 HGB-205 study being conducted in Paris, while a separate Phase 1 trial, HGB-206, enrolling upwards of eight sickle cell disease patients is currently underway in the U.S. LentiGlobin also recently demonstrated its ability to boost hemoglobin production in patients with beta-thalassemia major in the HGB-204 study — an indication for which the treatment recently was awarded an FDA BTD. The company has also been in a partnership with Celgene since 2013 to explore new and develop existing chimeric antigen receptor T-cell (CART) therapies in cancer.

UniQure: In a Phase 1 dose-escalation clinical trial, the company’s AAV5-PBGD candidate for Acute Intermittent Porphyria, AMT-021, was found to be a safe method of inserting the porphobilinogen deaminase gene (PBGD) into patients liver cells.

Bayer: Last summer, Bayer teamed up with Dimension Therapeutics to come up with a gene therapy treatment for hemophilia A.