Gene and Cell Therapy

Organ-directed gene transfer highlighted in review series
Human Gene Therapy is publishing a series of review articles written by key leaders in the field that each focus on therapeutic gene transfer to particular tissues, organs, and organ systems. Samuel C. Wadsworth, Dimension Therapeutics, and coauthors review the current status and challenges, historic context, and future directions for the development of liver-directed gene transfer in the article, "AAV Gene Therapy for Liver Disease." Ronald Crystal and coauthors have contributed the article, "Genetic Modification of the Lung Directed toward Treatment of Human Disease." Nathalie Cartier and colleagues have coauthored, "Adeno-associated Virus-based Gene Therapy for CNS Diseases." Lolita Petit, Hemant Khanna, and Claudio Punzo are the authors of "Advances in Gene Therapy for Diseases of the Eye."

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ESGCT 2017 session topics announced
The 25th Anniversary Congress of the ESGCT, 17–20 October in Berlin, Germany, will feature Plenary sessions on the following wide-ranging and timely topics: 
- Highlights of clinical progress; 
- Stem cells: biology, manipulation and reprogramming; 
- Cancer immuno-gene therapy; 
- New tools and technology: gene and genome editing and engineering; and
- Gene and cell therapy in the market. 
Parallel sessions will target key areas in biomedical and clinical research including the following: iPS disease modeling; ocular and central nervous system gene and cell therapy; oncolysis; gene editing; cardiovascular, muscle, and pulmonary gene and cell therapy; vector development; regenerative therapies; metabolic and lysosomal storage diseases; cancer predisposition, ageing, and genetic instability syndromes; blood disorders; and cancer gene therapy.

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Sangamo BioSciences advances treatments for hemophilia A and MPS I 
Sangamo Biosciences announced U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug Application (IND) for its SB-525 gene therapy program for the treatment of hemophilia A. SB-525 uses an adeno-associated virus (AAV2/6) cDNA human Factor 8 construct containing a proprietary synthetic liver-specific promoter. The company also received Orphan Drug Designation from the FDA for its genome editing product SB-318 to treat mucopolysaccharidosis type I. SB-318 is intended as a single treatment to provide continuous production of the L-iduronidase (IDUA) gene that is deficient in patients with MPS I. Sangamo also plans to test SB-318 in MPS II and hemophilia B.

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Experts discuss progress, controversies with editing the human genome 
Marianne J. Legato, MD, PhD, Editor-in-Chief of the new journal Gender and the Genome, moderated a roundtable discussion, "Editing the Human Genome: Progress and Controversies" published in the premier issue of the journal. Participants George M. Church, PhD, Harvard University and MIT (Cambridge, MA), Henry T. Greely, JD, Stanford University School of Law (CA), Tetsuya Ishii, PhD, Hokkaido University (Japan), Virginia M. Miller, PhD, Mayo Clinic (Rochester, MN), and Justin P. Killian, Esq. and Sherif Moussa, Esq., both of Foundation for Gender-Specific Medicine (New York, NY) debated a wide range of topics. They discussed the ethics of human embryonic genome editing in general, the impact of the sex of the embryo on genome editing, the possible regulatory landscape, potential safety issues, and the legal implications of parents' rights to manipulate the genome of their unborn child, among other key issues.

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Sarepta Therapeutics to develop gene therapies
Known for its development of exon-skipping drugs to treat Duchenne muscular dystrophy, Sarepta Therapeutics has announced that it is partnering with Nationwide Children's Hospital to develop two gene therapies for patients with DMD. One is a microdystrophin gene therapy and the other is a Galgt2 gene therapy. Jerry Mendell, MD, and Louise Rodino-Klapac, PhD, are the lead principal investigators of the microdystrophin gene therapy program, which is expected to enter clinical testing in late 2017.

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Alexandros Sfakianakis
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