Bluebird Bio to Present 6 abstracts at ASH

 Six abstracts accepted for presentation –

– Interim data on LentiGlobin in beta-thalassemia show six of six evaluable non-β⁰/β⁰ patients are transfusion-independent as of July 31^st data cut-off; β⁰/β⁰ patients have achieved varying degrees of transfusion reduction as of data cut-off –

    – Median production of corrected HbA^T87Q globin: 5.2 g/dL among patients of all genotypes followed for at least six months in HGB-204 study, as of July 31^st data cut-off –

    – Patient with sickle cell disease from HGB-205 study producing 51.5% anti-sickling hemoglobin at nine months post-treatment –

– Company will discuss ASH abstract data in a conference call today at 8:30 a.m. ET–

http://finance.yahoo.com/news/bluebird-bio-present-lentiglobin-bb305-130000247.html

CRISPR to be Tested in Humans by 2017 says Editas' CEO

The biotechnology startup Editas Medicine intends to begin tests of a powerful new form of gene-repair in humans within two years.
Speaking this week at the EmTech conference in Cambridge, Massachusetts, Editas CEO Katrine Bosley said the company hopes to start a clinical trial in 2017 to treat a rare form of blindness using CRISPR, a groundbreaking gene-editing technology.
If Editas’s plans move forward, the study would likely be the first to use CRISPR to edit the DNA of a person.
CRISPR technology was invented three years ago but is so precise and cheap to use it has quickly spread through biology laboratories. Already, scientists have used it to generate genetically engineered monkeys, and the technique has stirred debate over whether modified humans are next.
Editas is one of several startups, including Intellia Therapeutics and CRISPR Therapeutics, that have plans to use the technique to correct DNA disorders that affect children and adults. Bosley said that because CRISPR can “repair broken genes” it holds promise for treating several thousand inherited disorders caused by gene mistakes, most of which, like Huntington’s disease and cystic fibrosis, have no cure.

Sangamo Mention:
Although the Editas study could be the first for CRISPR in humans, it wouldn’t be the first clinical study of gene editing. An older method called zinc fingers is already in testing to treat HIV infection by the biotechnology company Sangamo Biosciences. But the versatility and ease with which CRISPR can change DNA means it may outpace earlier approaches.


Read more here:
http://www.technologyreview.com/news/543181/crispr-gene-editing-to-be-tested-on-people-by-2017-says-editas/

Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery

1. Jianbin Wang*,
2. Joshua J. DeClercq,
3. Samuel B. Hayward,
4. Patrick Wai-Lun Li,
5. David A. Shivak,
6. Philip D. Gregory,
7. Gary Lee and
8. Michael C. Holmes

- Author Affiliations

1. Sangamo BioSciences, Inc., Richmond, CA 94804, USA

This Article

1. Nucl. Acids Res. (2015) doi: 10.1093/nar/gkv1121 First published online: November 2, 2015

1. This article is Open AccessOA
3. » AbstractFree
4. Full Text (HTML)Free
5. Full Text (PDF)Free

Abstract

The adoptive transfer of engineered T cells for the treatment of cancer, autoimmunity, and infectious disease is a rapidly growing field that has shown great promise in recent clinical trials. Nuclease-driven genome editing provides a method in which to precisely target genetic changes to further enhance T cell function in vivo. We describe the development of a highly efficient method to genome edit both primary human CD8 and CD4 T cells by homology-directed repair at a pre-defined site of the genome. Two different homology donor templates were evaluated, representing both minor gene editing events (restriction site insertion) to mimic gene correction, or the more significant insertion of a larger gene cassette. By combining zinc finger nuclease mRNA delivery with AAV6 delivery of a homologous donor we could gene correct 41% of CCR5 or 55% of PPP1R12C (AAVS1) alleles in CD8⁺ T cells and gene targeting of a GFP transgene cassette in >40% of CD8⁺ and CD4⁺ T cells at both the CCR5 and AAVS1 safe harbor locus, potentially providing a robust genome editing tool for T cell-based immunotherapy. 

Lysogene Planning Phase 3 Gene Therapy Trial for SanFilippo A in 2017 ( lysosomal storage disease)

20 minute podcast with the founder of Lysogene (also mother of child with SanFilippo) planning on starting gene therapy PIII in 2017

https://globalgenes.org/raredaily/rarecast-gene-therapy-company-born-from-mothers-quest-to-cure-daughter/

Sangamo short Interest

Sangamo Short Interest (SGMO)

Sangamo Short Interest (SGMO)

DATE                                   SHORT INTEREST

10/15/15                              12,821,197    (11.36 days to cover)

9/30/15                                12,619,596

9/15/15                                10,882,795

8/31/15                                10,150,146

8/14/15                                  9,488,474

7/31/15                                  9,207,150

7/15/15                                  9,205,202

6/30/15                                  9,387,951
6/15/15                                  9,393,825
5/29/15                                  8,938,987
5/15/15                                  8,668,559
4/30/15                                  8,198,983
4/15/15                                  8,050,307
3/31/15                                  8,285,803
3/13/15                                  8,441,291
2/27/15                                  8,939,000
2/13/15                                  9,268,065
1/30/15                                  9,082,814
1/15/15                                  9,387,913

Jefferies Initiation on 10/23 (SGMO)

Sangamo Biosciences, Inc. ($6.42 BUY)
Assuming with Buy, Risk/Reward Skewed to Upside
Gena Wang, PhD, CFA
Key Takeaway
With stock trading at ~$300M EV, we believe the ZFN gene-editing technology platform
with initial clinical proof-of-concept established, should support the current valuation for SGMO, while any further clinical validation would provide substantial upside.
Price target $12 to $14

Rebar Presentation in September 2015

Edward Rebar, Sangamo Biosciences presented "Genome Editing with Engineered Zinc Finger Nucleases" at the International Society for Experimental Hematology annual meeting on September 18th 2015. For the record Phil Gregory was the original invited speaker. The beat goes on.