Category: Gene therapy

MELBOURNE, AUSTRALIA– – Genetic Technologies Limited today announced it has filed its Quarterly Activities Report and Appendix 4C for period ending December 31, 2011 in accordance with the ASX.Total cash …Source:
http://news.search.yahoo.com/news/rss?ei=UTF-8&p=genetic+therapy&eo=UTF-8

* Shire (Stuttgart: A0MMAG – news) to pay $13 mln upfront, plus milestones

* Sangamo shares rise more than 25 percent (Adds analyst comment, share price)

Feb 1 (Reuters) – Pharmaceutical company Shire said on Wednesday it would collaborate with Sangamo BioSciences (Hamburg: GBY.HM – news) to develop treatments for hemophilia and other inherited diseases based on Sangamo's gene regulation and modification technology.

The news, seen as a vote of confidence in Sangamo's experimental gene-disrupting technique, sent shares of the Richmond (NZSE: RHD.NZ – news) , California-based company up more than 25 percent.

Shire is buying the rights to the U.S. company's proprietary technology to target four genes, which the company said it hoped could lead to therapies for haemophilia A and B. Shire also acquired the right to designate three additional gene targets.

Sylvie Grégoire, president of Shire's Human Genetic Therapies business, said: “While still early in the clinical development process, this DNA-binding protein technology is aligned with our focus of developing new treatments.”

The British company is paying Sangamo $13 million in an upfront payment, followed by development and commercial payments and royalties on product sales.

After the deal was announced, Piper Jaffray (Berlin: PJR.BE – news) upgraded Sangamo to “neutral” from “underweight,” and raised its price target on the shares to $4 from $2.

Shares of Sangamo were up 92 cents at $4.37 in midday trading on Nasdaq (Nasdaq: ^NDX – news) .

Genetic disease are an important therapeutic area for Shire, and the group has found success with drugs to treat the rare Gaucher's and Fabry's diseases. (Reporting by Paul Sandle and Deena Beasley. Editing by Gunna Dickson)

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UPDATE 2-Shire partners Sangamo in gene therapy for hemophilia

01-02-2012 05:22 Specific challenges of avant-garde therapies Authorities/sponsors: being concomitantly on a learning curve Translational research: key success factors Case study: SAF-301

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Entering into the clinic with an intracerebral gene therapy product / Karen Aiach, Project Manager, – Video

26-01-2012 21:26 Short Trailer on the history and applications of Gene Therapy in the modern medical scene. Written and directed by Thenmozhi Soundaranagaran Score by Keith Jackson

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Apes Trailer/Gene Therapy – Video

SAN DIEGO, Jan. 30, 2012 /PRNewswire/ – Cardium Therapeutics (NYSE Amex: CXM) today reported on new insights and confirmatory preclinical study results on the Generx (Ad5FGF-4) angiogenic therapy product …

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Cardium Presents New Generx Findings at 2012 Annual Gene and Cell Therapy Forum

Fibrocell Science, Inc. , a biotechnology company focused on the development of autologous cell therapies for aesthetic, medical and scientific applications, announces that the June 2011 FDA approval of LAVIV™ for the improvement of the appearance of “smile line” wrinkles was named the #3 top story of 2011 in cell and gene therapy and regenerative medicine at The 2012 Cell & Gene Therapy Forum …

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Fibrocell Science, Inc. Announces Recognition for LAVIV™ (azficel-T) at The 2012 Cell & Gene Therapy Forum, Washington …

AMSTERDAM, January 30, 2012 /PRNewswire/ –

Amsterdam Molecular Therapeutics (Euronext:
AMT –
News), a leader in the field of human gene therapy, was
informed on Friday January 27, 2012 after business hours that
the European Commission's Standing Committee of the
European
Parliament discussed on Monday January 22, 2012 the
implementation decision not to grant marketing authorization
for Glybera (alipogene tiparvovec) as recommended by the
Committee for Human Medicinal Products (CHMP). After the
discussion no clear position in favor or against granting a
marketing authorization for Glybera was reached. Instead the
Standing
Committee considered it necessary to request additional
information to the CHMP in the European Medicines Agency (EMA).
A formal vote by the Standing Committee will be made on review
of the additional information. It is currently unclear when a
final decision will be reached.

Glybera is a gene
therapy for patients with the genetic disorder
lipoprotein lipase deficiency (LPLD). Currently no alternative
treatment is available for patients deficient of LPL, who are
suffering from recurrent pancreatitis.

On October 23, 2011, AMT received a non-approvable opinion from
the CHMP (following a re-examination of the marketing approval
dossier originally rejected in June 2011) despite a
recommendation to approve Glybera by the Committee For Advanced
Therapies (CAT). The CAT is an expert body implemented by
the European
Commission to prepare and advise the CHMP on decisions
regarding advanced therapeutics, in particular gene and cell
therapies.

“We are very encouraged by the outcome of the discussion by the
European Parliament's Standing Committee to request further
information from the CHMP. The evaluation of (ultra-) orphan
drugs and advanced therapies is novel territory. The ongoing
debate shows that the authorities are determined to find the
best process of dealing with such innovative treatments and
technologies. We will constructively work with the European
Commission and the EMA to find the best process going forward,
not only for Glybera but also for further advanced therapies on
their way to patients,” said Jörn Aldag, CEO of AMT. “While
gaining clarity for the process involving Glybera, we will
continue to implement our revised business strategy revealed
following the last CHMP decision. This involves moving forward
in partnering discussions for several of our gene therapy
products, including hemophilia B and GDNF.”

AMT's current business plan, organizational structure and
financing have been developed on the basis of the current CHMP
opinion for Glybera's non-approval. It involved a reduction of
its workforce by about 50%. In the event that Glybera could be
commercialized in Europe, the Company will evaluate how the
opportunity arising from passing this important milestone could
be realized most efficiently.

About Glybera®

AMT has developed Glybera as a treatment for patients with the
genetic disorder lipoprotein lipase deficiency. LPLD is an
orphan disease for which no treatment exists today. The disease
is caused by mutations in the LPL gene, resulting in highly
decreased or absent activity of LPL protein in patients. This
protein is needed in order to break down large fat-carrying
particles that circulate in the blood after each meal. When
such particles, called chylomicrons, accumulate in the blood,
they may obstruct small blood vessels. Excess chylomicrons
result in recurrent and severe acute inflammation of the
pancreas, called pancreatitis, the most debilitating and
life-threatening clinical complication of LPLD. Glybera has
orphan drug status in the EU and US.

About Amsterdam Molecular Therapeutics

AMT is a world leader in the development of human gene
based therapies. In addition to Glybera, AMT has a product
pipeline of several gene therapy products in development for
hemophilia B, Duchenne muscular dystrophy, acute intermittent
porphyria, Parkinson's disease and SanfilippoB. Using
adeno-associated viral (AAV) derived vectors as the delivery
vehicle of choice for therapeutic genes, the company has been
able to design and validate probably the world's first stable
and scalable AAV manufacturing platform. This proprietary
platform can be applied to a large number of rare (orphan)
diseases caused by one faulty gene and allows AMT to pursue its
strategy of focusing on this sector of the industry.  AMT
was founded in 1998 and is based in Amsterdam. Further
information can be found at

http://www.amtbiopharma.com.

Certain statements in this press release are
“forward-looking statements” including those that refer to
management's plans and expectations for future operations,
prospects and financial condition. Words such as “strategy,”
“expects,” “plans,” “anticipates,” “believes,” “will,”
“continues,” “estimates,” “intends,” “projects,” “goals,”
“targets” and other words of similar meaning are intended to
identify such forward-looking statements. Such statements are
based on the current expectations of the management of AMT
only. Undue reliance should not be placed on these statements
because, by their nature, they are subject to known and unknown
risks and can be affected by factors that are beyond the
control of AMT. Actual results could differ materially from
current expectations due to a number of factors and
uncertainties affecting AMT's business. AMT expressly disclaims
any intent or obligation to update any forward-looking
statements herein except as required by law. 

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Amsterdam Molecular Therapeutics Announces European Commission Delays Decision on Glybera® Marketing Authorisation and …

Putting genetic testing at the heart of the NHS could herald a "revolution" in diagnosing, treating and preventing disease, according to the government's genetics adviser.Source:
http://news.search.yahoo.com/news/rss?ei=UTF-8&p=genetic+therapy&eo=UTF-8

The time for commercial development of gene therapy has come. Patients with diseases treatable and curable with gene therapy deserve access to the technology, which has demonstrated both its effectiveness and feasibility, says James Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy in a provocative commentary and accompanying videocast. Human Gene Therapy and Human Gene Therapy (HGT …Source:
http://news.search.yahoo.com/news/rss?ei=UTF-8&p=gene+therapy&eo=UTF-8

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)Source:
http://www.medworm.com/rss/medicalfeeds/therapies/Gene-Therapy.xml

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)Source:
http://www.medworm.com/rss/medicalfeeds/therapies/Gene-Therapy.xml

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)Source:
http://www.medworm.com/rss/medicalfeeds/therapies/Gene-Therapy.xml

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)Source:
http://www.medworm.com/rss/medicalfeeds/therapies/Gene-Therapy.xml

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)

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http://www.medworm.com/rss/medicalfeeds/therapies/Gene-Therapy.xml

Adoptive immunotherapy using TCR gene modified T cells may allow separation of beneficial Graft versus tumour responses from harmful GvHD. Improvements to this include methods to generate high avidity or high affinity TCR, improvements in vector design and reduction in mispairing. Following adoptive transfer, TCR transduced T cells must be able to survive and persist in vivo to give most effective antitumour responses. Central memory or naive T cells have both been shown to be more effective than effector cells at expanding and persisting in vivo. Lymphodepletion may enhance persistence of transferred T cell populations. TCR gene transfer can be used to redirect CD4 helper T cells, and these could be used in combination with CD8+ tumour specific T cells to provide help for the antitumour r…Source:
http://www.medworm.com/rss/medicalfeeds/therapies/Gene-Therapy.xml

Research sheds light on Joubert syndrome, which causes physical and mental disabilities

Source: HealthDay
Related MedlinePlus Pages: Cerebellar Disorders, Genes and Gene Therapy (Source: MedlinePlus Health News)Source:
http://www.medworm.com/rss/medicalfeeds/therapies/Gene-Therapy.xml

Researchers zero in on 13 new genetic clues

Source: HealthDay
Related MedlinePlus Pages: Genes and Gene Therapy, Menopause (Source: MedlinePlus Health News)Source:
http://www.medworm.com/rss/medicalfeeds/therapies/Gene-Therapy.xml

Mutations in two genes found in tumors appear to fuel their growth

Source: HealthDay
Related MedlinePlus Pages: Bile Duct Cancer, Genes and Gene Therapy (Source: MedlinePlus Health News)Source:
http://www.medworm.com/rss/medicalfeeds/therapies/Gene-Therapy.xml

Human Gene Therapy , Vol. 0, No. 0. (Source: Human Gene Therapy)

Source:
http://www.medworm.com/rss/medicalfeeds/therapies/Gene-Therapy.xml

23-01-2012 19:59 For Bio Honors

Link:
Gene Therapy: Dr. Zehhr – Video