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New Treatment Options for Hemoglobin-Based Blood Disorders

    Sickle cell disease is a group of inherited disorders that affects hemoglobin, the oxygen-carrying protein in red blood cells (RBCs), and causes these cells to distort in shape at low oxygen levels. This condition affects 1 in 500 Americans of African descent and 1 in 1,000-1,400 of Hispanic descent, as well as millions...

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Zolgensma Launch “On Track,” but Significant Challenges Remain

On July 18, Novartis told analysts its Zolgensma launch was “on track.” Despite the gene therapy’s $2.1 million price tag, the company states the launch is “one of the most successful launches from an access standpoint in rare diseases.” Novartis said it had reached coverage deals with more than 20 commercial insurance plans, covering approximately 40%...

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Breaking Out of the Bubble

X-linked severe combined immunodeficiency (X-SCID, or SCID-X1), is an orphan monogenic disorder popularly known as ‘bubble boy disease’ that affects 1 in 50,000-100,000 live births. The most serious of all primary immunodeficiencies, X-SCID causes severe failure of the immune system, including a lack of T cells, natural killer (NK) cells, or functional B cells. For...

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One-and-Done Pricing Challenges

As more gene and cell therapies come to market, a big challenge facing both drug developers and payers is designing and instituting appropriate pricing models for these one-time, potentially curative treatments. Innovation should be rewarded, and a curative — or at least, very long-lasting — treatment is likely to save significant dollars in the typical...

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Gene Therapy Update Part 2

Last week we wrote about recent news in the field of CRISPR/Cas9 research. But CRISPR is not the only approach to gene editing under development, nor the most advanced.   First Human Gene Editing Data — But Not from CRISPR   Sangamo Therapeutics, Inc. has been developing an approach to gene editing based on zinc...

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FDA Issues Set of Guidelines on Gene Therapy

Back in December, the U.S. Food and Drug Administration (FDA) granted its first approval for a gene therapy product, Spark Therapeutics’ Luxturna. According to the Alliance for Regenerative Medicine, as many as 50 more such treatments — addressing a diverse range of health problems from genetic, autoimmune, and heart diseases, to cancer and HIV/AIDS —...

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First Up For The New FDA RMAT Designation: Gene Therapeutics for Hemophilia

We recently wrote about the U.S. Food and Drug Administration’s (FDA) creation of a Regenerative Medicine Advanced Therapy (RMAT) designation in an effort to speed up the approval process for certain types of treatments, including gene therapies. The agency has specifically chosen gene therapy for hemophilia as its first focus, due to the considerable effort...

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Gene Therapy for Inherited Retinal Disease Achieves a Historic Milestone

This has been an amazing year in the world of drug development, with three new therapeutic classes marking historic gains. We first saw Novartis’ FDA approval for the first CAR-T immunotherapy. Then Alnylam’s RNAi treatment achieved Phase 3 success, the first such for that class of therapeutics. Most recently, Spark Therapeutics received a unanimous recommendation...

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Gene Therapy: Coming of Age Perhaps, but Who Will Pay?

New technologies tend to go through cycles of interest and attention. Initial excitement about the potential of a technological advance too often builds to over-blown hype, followed by a quick reversal of interest in the aftermath of a big disappointment. But if continued research later produces truly encouraging results, interest can build again as time...