NEW YORK (TheStreet) -- Two members of the Biotech IPO Class of 2013 -- Bluebird Bio (BLUE) and Agios Pharmaceuticals (AGIO) -- will deliver important pipeline updates at the European Hematology Association (EHA) Congress on Sat. June 14. Let's preview both presentations, starting with Bluebird and its LentiGlobin gene therapy for beta-thalassemia.
Lenti what for beta who?
LentiGlobin is gene therapy, meaning it insertsa fully functional gene for human beta-globin into the patient's own hematopoietic stem cells. The theory behind gene therapy is relatively simple: For diseases caused by single, malfunctioning or missing gene, effective treatment or even a complete cure can be achieved (theoretically) by replacing the defective gene with one that is fully functional. Gene therapy sounds simple but the execution is obviously a lot more complicated.
I won't get into the weeds explaining the process by which BlueBird produces and delivers LentiGlobin except to say a working gene for human beta-globin would allows beta-thalassemia (B-Thal) patients to produce functional beta-globin -- the largest component of hemoglobin, which carries oxygen in red blood cells. B-Thal patients suffer from anemia and iron overload. There is no currently approved cure or effective treatment, so patients require regular blood transfusions to combat the anemia.
Bluebird went public last year partly on the back of some encouraging proof of concept data showing a first-generation gene therapy approach was feasible for B-thal patients. LentiGlobin is a second-generation gene therapy designed to be more potent -- and hopefully more effective.
Next Saturday, researchers will present data on two B-thal patients treated with LentiGlobin in an ongoing phase I/II study. Key data to look for include the engraftment success rate i.e. how many "gene-therapy treated" stem cells are populating the bone marrow; improvements in hemoglobin levels; achievement of transfusion independence; and if so, how quickly from the gene therapy.
Agios will be presenting a clinical update from an early-stage study of experimental cancer metabolism drugAG-221 last reported at the American Association of Cancer Research annual meeting in April.
Agios is developing AG-221 under a collaboration with Celgene (CELG). The companies are also working together on AG-120, a similar drug designed to block another mutated cancer-growing protein known a IDH1.
At the EHA meeting on Sat. June 14, researchers will be presented updated study results from additional patients treated with AG-221, some given higher doses of the drug.
In April at AACR, six of 10 patients with advanced, treatment-refractory acute myeloid leukemia carrying the IDH2 mutation had objective tumor responses, including three complete remissions and two complete remissions with incomplete platelet recovery. A single patient achieved a partial response.
Here is the original post:
Bluebird, Agios: Preview of Next Week's Important Pipeline Updates
- Gene Therapy: Ethical Issues: Information from Answers.com - February 6th, 2014 [February 6th, 2014]
- Gene, immune therapy help cancer war - April 10th, 2014 [April 10th, 2014]
- Gene Therapy - Genetics Home Reference - Your guide to ... - April 10th, 2014 [April 10th, 2014]
- What is gene therapy? - Genetics Home Reference - April 10th, 2014 [April 10th, 2014]
- Gene therapy - Wikipedia, the free encyclopedia - April 10th, 2014 [April 10th, 2014]
- Gene Therapy PSA Dan Lesser Period 2 - Video - April 15th, 2014 [April 15th, 2014]
- Gene therapy basics - Video - April 15th, 2014 [April 15th, 2014]
- In vivo gene therapy - Video - April 15th, 2014 [April 15th, 2014]
- Ex vivo gene therapy - Video - April 15th, 2014 [April 15th, 2014]
- Broad Institute Gets Patent on Revolutionary Gene-Editing Method - April 17th, 2014 [April 17th, 2014]
- Gene Therapy - Video - April 17th, 2014 [April 17th, 2014]
- Mark Hamalainen: Gene Therapy - Video - April 17th, 2014 [April 17th, 2014]
- JFK - Gene Therapy for Brain Tumors - Video - April 18th, 2014 [April 18th, 2014]
- Fixing the Liver with New Gene Therapy (Brainstorm Ep178) - Video - April 18th, 2014 [April 18th, 2014]
- Gene Therapy | Buzzle.com - April 22nd, 2014 [April 22nd, 2014]
- Gene Therapy I - RCN Corporation - April 22nd, 2014 [April 22nd, 2014]
- Ricky Ashby Microbiology Podcast - Video - April 22nd, 2014 [April 22nd, 2014]
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- Pronounce Medical Words Gene Therapy - Video - April 24th, 2014 [April 24th, 2014]
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- Cochlear Implant Also Uses Gene Therapy to Improve Hearing - April 25th, 2014 [April 25th, 2014]
- Gene Therapy May Enhance Cochlear Implants, Animal Study Finds - April 25th, 2014 [April 25th, 2014]
- Uniqure Gene Therapy Info. - Video - April 25th, 2014 [April 25th, 2014]
- Jean Bennett on gene therapy as a treatment for blindness - Video - April 25th, 2014 [April 25th, 2014]
- mice-science-AFPrelax-250414.jpg - April 26th, 2014 [April 26th, 2014]
- Cochlear implant enhances patient experience through gene therapy - April 28th, 2014 [April 28th, 2014]
- Gene therapy may help hearing - April 28th, 2014 [April 28th, 2014]
- Ethics of Gene therapy - Video - April 30th, 2014 [April 30th, 2014]
- Novel Financing For Gene Therapy Company - May 1st, 2014 [May 1st, 2014]
- Gene Therapy To Enhance Hearing - ABC Austrlia News - 2014 - - Video - May 1st, 2014 [May 1st, 2014]
- Biotechnology - GMO's & Gene Therapy - Video - May 1st, 2014 [May 1st, 2014]
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- Gene therapy for haemophilia - Professor Amit Nathwani - Video - May 4th, 2014 [May 4th, 2014]
- Retrovirus in Gene Therapy - Video - May 4th, 2014 [May 4th, 2014]
- Regeneron, Avalanche To Develop Novel Gene Therapy Products In Ophthalmology - May 5th, 2014 [May 5th, 2014]
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- SCID gene therapy class - Video - May 6th, 2014 [May 6th, 2014]
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- gene therapy video - Video - May 9th, 2014 [May 9th, 2014]
- Chronic Granulomatous Disease (CGD) - Video - May 12th, 2014 [May 12th, 2014]
- Gene Therapy (Breast Cancer) - Video - May 12th, 2014 [May 12th, 2014]
- Suicide gene therapy - Video - May 12th, 2014 [May 12th, 2014]
- Employment of gene therapy in depression (psychiatric disorder) - Video - May 12th, 2014 [May 12th, 2014]
- Gene therapy for blindness (2012) - Video - May 12th, 2014 [May 12th, 2014]
- AGRC1020 Video Presentation Gene Therapy restores colour vision in monkeys - Video - May 12th, 2014 [May 12th, 2014]
- gene therapy~3 - Video - May 12th, 2014 [May 12th, 2014]
- Gene Delivery System (In-vivo and Ex-vivo) HD - Video - May 12th, 2014 [May 12th, 2014]
- Spark Therapeutics to open headquarters in West Philadelphia - May 14th, 2014 [May 14th, 2014]
- Gene Therapy - Nature - May 14th, 2014 [May 14th, 2014]
- Gene therapy for RTT syndrome benefits and drawbacks - Video - May 16th, 2014 [May 16th, 2014]
- Gene Therapy (Using Biolistic gene gun for gene delivery) - Video - May 16th, 2014 [May 16th, 2014]
- Germline Gene Therapy - Video - May 16th, 2014 [May 16th, 2014]
- Gene Therapy Biology Project for Swavely - Video - May 16th, 2014 [May 16th, 2014]
- Gene therapy extends survival in an animal model of spinal muscular atrophy - May 22nd, 2014 [May 22nd, 2014]
- Novel RNAi therapy silences mutated Huntington's disease gene and reduces symptoms - May 22nd, 2014 [May 22nd, 2014]
- Global Gene Therapy Market: Analysis, Size, Share, Growth, Trends and Forecast 2013 - 2019 - May 22nd, 2014 [May 22nd, 2014]
- Gene therapy hits blood cancers hard - May 22nd, 2014 [May 22nd, 2014]
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