News of Interest

19 December 2017 | FDA Approves Novel Gene Therapy to Treat Patients with a Rare Form of Inhereted Vision Loss

Luxturna is the first gene therapy approved in the U.S. to target a disease caused by mutations in a specific gene...more

7 December 2017 | Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

NEJM, USA, December 7, 2017- The prevention of bleeding with adequately sustained levels of clotting factor, after a single therapeutic intervention...more

27 November 2017 | Gene therapy hits a peculiar roadblock: A virus shortage

New-York Times, USA, November 27, 2017- Eager to speed development of revolutionary treatments, the Food and Drug Administration recently announced that it would expedite approval of experimental gene therapies. But...more

13 July 2017 | Novartis CAR-T cell therapy recommended for approval by FDA advisory committee

Basel, SWITZERLAND, July 13, 2017-Novartis announced today that the US Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) unanimously (10-0) recommended...more

20 March 2017 | Will AAV Vectors Have a Role in Future Novel Gene Therapy Approaches?

New Rochelle, NY, March 20, 2017— Recombinant adeno-associated virus (rAAV) vectors for delivering therapeutic genes have demonstrated their safety in multiple diseases and clinical settings...more

01 August 2016 | Pfizer Acquires Gene Therapy Firm Bamboo for $150 Million

Pfizer Inc. said Monday that it acquired Bamboo Therapeutics Inc., a biotechnology firm developing gene therapies, for $150 million and potential milestone payments in a deal to expand the drug giant’s presence in the experimental field....more

22 July 2016 | Breakthrough status for Pfizer/Spark haemophilia gene therapy

Pfizer and Spark Therapeutics say their investigational haemophilia B drug SPK-9001 has been awarded breakthrough therapy status by the US Food and Drug Administration. SPK-9001 is a novel bio-engineered adeno-associated virus (AAV) capsid expressing a codon-optimised, high-activity human factor IX variant, currently being assessed in an ongoing Phase I/II trial as a potential one-time therapy....more

23 June 2015 | bluebird raises $500M follow-on

bluebird bio Inc.(NASDAQ:BLUE) dipped $4.21 to $170.10 after it raised $500 million through the sale of 2.9 million shares at $170 in a follow-on underwritten by BofA Merrill Lynch; Morgan Stanley; Cowen; SunTrust Robinson Humphrey; Wedbush PacGrow; and Roth Capital Partners. The company had proposed to raise $400 million after market close on Tuesday.

The offering is the biotech's third follow-on in less than a year. bluebird raised $259 million through the sale of about 3 million shares at $85 a share in December 2014, and $117.3 million through the sale of about 3.5 million shares at $34 in July 2014.The company had $334.9 million in cash at March 31.

15 May 2015 | Gene Therapy Comes of Age; Gene Therapy Has Had Its Growing Pains, but It Is Now Coming Into Its Own

Vicki Glaser, It seldom happens that a premature shoot of genius ever arrives at maturity. One such shoot, gene therapy, appears to be one of the exceptions. Gene therapy, which showed early promise as a means of replacing defective or missing genes, is branching out.

And it may yet produce abundant and diverse fruits. For example, gene therapy is being cultivated in cardiovascular applications, which are relevant to large, broad-based patient populations. Gene therapy approaches to cardiovascular and other diseases are being tended in billion-dollar collaborations, and they are being evaluated in late-stage clinical trials.

Current gene therapy products in development utilize a variety of viral vectors. Some of them integrate into the host cell genome to achieve long-term protein expression; others do not, aiming for only transient expression of a therapeutic product.


06 April 2015 | Bristol-Myers Squibb and uniQure Enter into Exclusive Strategic Collaboration to Develop Gene Therapies for Cardiovascular Diseases

Bristol-Myers Squibb Company(NYSE:BMY) and uniQure N.V. (NASDAQ:QURE) announced today an agreement that provides Bristol-Myers Squibb with exclusive access to uniQure’s gene therapy technology platform for multiple targets in cardiovascular diseases.....Under the terms of the agreement, Bristol-Myers Squibb will make near-term payments of approximately $100 million, including an upfront payment of $50 million to be made at the closing of the transaction, a $15 million payment for the selection of three collaboration targets, in addition to S100A1, to be made within three months of the closing and an initial equity investment in uniQure for a number of shares that will equal 4.9% of the total number of shares outstanding following such issuance, at a purchase price of $33.84 per share, or at least $32 million in total...more....

16. Februar 2015 | Für immer frisch - USA lassen genetisch veränderten Apfel zu

Katrin Zinkart, Süddeutsche Zeitung, mehr....

13 February 2015 | Nonbrowning Arctic®Apples To Be Granted Approval

Summerland, B.C., Canada– Today, the U.S. Department of Agriculture's (USDA) Animal and Plant Healthy Inspection Service (APHIS) announced its decision to deregulate the first two nonbrowning apple varieties, Arctic® Golden and Arctic® Granny apples,in the United States. It is expected that APHIS' final environmental assessment (EA) and plant pest risk assessment (PPRA) will be published in the Federal Register soon. According to the USDA's announcement, these reviews have found that Arctic®apples "are unlikely to pose a plant pest risk" and deregulation "is not likely to have a significant impact on the human environment." more....

02 February 2015 | FDA Grants Breakthrough Therapy Designation to LentiGlobin for Treatment of Beta-Thalassemia Major

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb. 2, 2015--bluebird bio, Inc.(Nasdaq:BLUE) a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases, today announced that the U.S. Food and Drug Administration(FDA) has granted Breakthrough Therapy designation to LentiGlobin®BB305 Drug Product for the treatment of transfusion-dependent patients with beta-thalassemia major. more....

26 November 2014 | Exclusive: First gene therapy drug sets million-euro price record

Reuters, By Ludwig Burger and Ben Hirschler November 26, 2014 12:52 PM

FRANKFURT/LONDON (Reuters) - The Western world's first gene therapy drug is set to go on sale in Germany with a 1.1 million euro ($1.4 million) price tag, a new record for a medicine to treat a rare disease. more....

14 August 2014 | Miltenyi Biotec acquires gene therapy assets from Lentigen Corporation

Addition of lentiviral gene delivery capabilities complements strategic partnering portfolio.

BERGISCH GLADBACH, GERMANY, August 14, 2014 — Miltenyi Biotec announced today that it has acquired the lentiviral vector manufacturing business and related assets from US company Lentigen Corporation, a global leader in lentiviral technology for cell and gene therapy applications.

The acquisition further strengthens Miltenyi Biotec’s portfolio in cell and gene therapy. Assets obtained in the transaction include a broad range of intellectual property, process technologies, and cGMP-qualified manufacturing facilities. The Lentigen team will join Miltenyi Biotec, and operate through its newly formed, wholly owned US subsidiary Lentigen Technology, Inc. based in Gaithersburg, MD.bluebird bio, a developer of gene therapies for severe genetic and orphan diseases, said today it acquired Precision Genome Engineering (Pregenen), in a deal that could have the buyer shelling out as much as $139.9 million for the company and its debt. more....

10 August 2014 | Clinical trial to start soon on GSK Ebola vaccine

Credit: Reuters/Tommy Trenchard

LONDON (Reuters) - A clinical trial of an experimental vaccine against the deadly Ebola virus is set to start shortly, according to British drugmaker GlaxoSmithKline, which is co-developing the product with U.S. scientists. ......

The investigational vaccine is based on a chimpanzee adenovirus into which two Ebola genes have been inserted, which means it contains no infectious Ebola virus material. Adenoviruses are best known for causing the common cold.

Once the vaccine enters a cell and delivers its genetic payload, the two gene inserts produce a protein that generates an immune response in the body - but the adenovirus carrying the genes does not replicate further.

GSK acquired the vaccine after buying Swiss-based biotech company Okairos for 250 million euros ($335 million) last year. more....

30 June 2014 | bluebird bio Acquires Pregenen for Up to $139.9M


bluebird bio, a developer of gene therapies for severe genetic and orphan diseases, said today it acquired Precision Genome Engineering (Pregenen), in a deal that could have the buyer shelling out as much as $139.9 million for the company and its debt.

The deal gives bluebird access to Pregenen’s gene editing and cell signaling technologies, which the companies said could have a broad range of potential therapeutic applications in gene therapy as well as cancer immunotherapies.


06 November 2013 | Mit Viren gegen Tumoren

PHARMAZEUTISCHE ZEITUNG online, von Hermann Feldmeier / Die Idee, maligne Tumoren gezielt mit Viren zu bekämpfen, klingt bestechend. Erste Untersuchungen mit gentechnisch modifizierten Viren bestätigen das Konzept. Doch noch sind viele Fragen offen: Wie »erkennen« onkolytische Viren die Tumorzellen, und wie kommen sie überhaupt bis zum Tumor?


30 October 2013 | 'Bubble kid' success puts gene therapy back on track

Five children with a genetic disease that wipes out their immune system have successfully been treated with gene therapy (source: NewScientist)


16 October 2013 | Antibodies plus adenovirus-vectored interferon shows promise against Ebola

First Ebola antibody treatment to halt deadly virus inprimates (source: The Conversation)


10 September 2013 | Die Welt:Viren können bösartige Tumore zerstören, Hermann Feldmeier

Besonders Krebs in einem fortgeschrittenen Stadium kann häufig nicht mehr geheilt werden. Wenn Chemo- und Strahlentherapie nicht mehr wirksam sind, sollen künftig gentechnisch veränderte Viren helfen.


20 August 2013 | First Pre-Clinical Gene Therapy Study to Reverse Rett Symptoms

The concept behind gene therapy is simple: deliver a healthy gene to compensate for one that is mutated. New research published today in theJournal of Neurosciencesuggests this approach may eventually be a feasible option to treat Rett Syndrome, the most disabling of the autism spectrum disorders. Gail Mandel, Ph.D., a Howard Hughes Investigator at Oregon Health and Sciences University, led the study. The Rett Syndrome Research Trust, with generous support from the Rett Syndrome Research Trust UK and Rett Syndrome Research & Treatment Foundation, funded this work through theMECP2 Consortium.


11 July 2013 | Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

Science - 11 July 2013 - Wiskott-Aldrich syndrome (WAS) is an inherited immunodeficiency caused by mutations in the gene encoding WASP, a protein regulating the cytoskeleton. Hematopoietic stem/progenitor cell (HSPC) transplants can be curative but, when matched donors are unavailable, infusion of autologous HSPCs modified ex vivo by gene therapy is an alternative approach. We used a lentiviral vector encoding functional WASP to genetically correct HSPCs from three WAS patients and reinfused the cells after reduced-intensity conditioning regimen. All three patients showed stable engraftment of WASP-expressing cells and improvements in platelet counts, immune functions, and clinical score. Vector integration analyses revealed highly polyclonal and multilineage haematopoiesis resulting from the gene-corrected HSPCs. Lentiviral gene therapy did not induce selection of integrations near oncogenes, and no aberrant clonal expansion was observed after 20 to 32 months. Although extended clinical observation is required to establish long-term safety, lentiviral gene therapy represents a promising treatment for WAS


25 February 2013 | Okairos initiates Phase I clinical trial evaluating vaccine against RSV infections

basel, Switzerland – 25 February 2013 – Okairos today announced the initiation of a Phase I clinical trial evaluating a vaccine against respiratory syncytial virus (RSV), a leading cause of hospital admissions in infants and children. This clinical trial will enroll 40 healthy adult volunteers in the UK, with endpoints measuring the vaccine’s safety and immunogenicity. ....

Okairos' technology platform is centered on the development of new, potent adenovirus vectors to generate a pipeline of T-cell vaccines against a range of infectious diseases for which there is currently no effective vaccine. Okairos and its collaborators have vaccinated over 700 subjects with Okairos’ proprietary vectors, and in all cases have seen a strong immune response and excellent safety profile. The company is also pursuing therapeutic vaccines to treat cancer.


11 Februar 2013 | Onkolytische Viren könnte Leben bei Leberkrebs verlängern|Randomized dose-finding clinical trial of oncolytic immunotherapeutic vaccinia JX-594 in liver cancer

Aerzteblatt & Nature Medicine

San Francisco – Ein genetisch modifiziertes Impfpockenvirus, das Tumorzellen zerstört und eine Immunantwort auf die Krebserkrankung induziert, hat in einer Dosis-Findungsstudie die Überlebenszeit von Patienten mit hepatozellulärem Karzinom verlängert, wie aus einer Studie inNature Medicine (2013; doi: 10.1038/nm.3089)hervorgeht.


21 Januar 2013 | Die Spritze gegen Krebs

Focus Online: Die SPRITZE GEGEN TUMOREN ist kein Wunschtraum mehr. Ärzte setzten sie bereits für Impfung und Immuntherapie gegen bösartige Zellen ein. Die Erfolge machen Hoffnung...

TITEL: Die neue Krebs-Medizin - weiter lesen auf FOCUS

Focus Online....

04 January 2013 | Oral vaccination with an adenovirus-vectored vaccine protects against botulism

El Paso, TX – ...shown that an adenovirus vectored vaccine delivered intramuscularly or intranasally was effective in protection against botulism in a mouse model. The adenoviral vector encodes ......

Elsevier at ScienceDirect....

17 December 2012 | Virus programmiert Muskelzelle zum Herzschrittmacher

Los Angeles – Durch die Injektion eines einzelnen Steuergens können Herzmuskelzellen in Schrittmacher der kardialen Reizleitung verwandelt werden. Die tierexperimentellen Studien in Nature Biotechnology (2012; doi: 10.1038/nbt.2465) wecken Hoffnungen auf eine Gentherapie von Herzrhythmusstörungen.

06 December 2012 | Gentherapie soll Amputationen zukünftig verhindern

Wien - Eine Gentherapie mit veränderten Stammzellen aus dem Fettgewebe und Knochenmark könnte eine neue Option für die Behandlung von schweren orthopädischen Verletzungen an den Extremitäten darstellen. Entwickelt wurde diese Therapie.... GmbH 2012

02 November 2012 | Europe approves high-price gene therapy

Business & Financial News, Reuters | Ben Hirschler

01 October 2012 | Adenovirus Vectors and Subviral Particles for Protein and Peptide Delivery

Adenovirus vectors belong to the most frequently used vector type in gene therapy approaches. In addition, adenovirus vector particles and adenovirus subviral particles offer a great potential for the direct delivery of proteins into cells. In this review we discuss this potential and the technology of adenovirus as a protein delivery platform for applications ranging from vaccination to gene therapy.

Current Gene Therapy | Vol 12, Number 5, October 2012, pp. 362-373

10 September 2012, Will the floodgates open for gene therapy?

In a matter of days, a momentous event will occur: a gene therapy will, for the first time anywhere in the Western hemisphere, be available commercially with full marketing approval.

Nature Biotechnology 30, 805 (2012), Published online

24 July 2012 ι Gibt es bald eine Impfung gegen HIV?

Wissenschaftsjournalist berichtet von der Welt-AIDS-Konferenz in Washington - Martin Winkelheide im Gespräch mit Jochen Steiner, Deutschlandradio

Press Article in German

22. July 2012 ι Gentherapie vor der Zulassung

Die EU-Gesundheitsbehörde empfiehlt die Zulassung einer Gentherapie. Damit könnten 20 Jahre der Diskussion enden -

Press Article in German

20. July 2012 ι Biotech's gene therapy grabs first regulatory nod in Europe

By Ryan McBride ι Fierce Biotech

Europe could be months away from having its first approved gene therapy. The Dutch biotech uniQure won a positive opinion from the EMA's Committee for Medicinal Products for Human Use (CHMP) for its gene therapy to treat severe cases of the extremely rare disorder lipoprotein lipase deficiency (LPLD), making the first such regulatory backing for a gene therapy in the Western World.

Press Article in English

16 July 2012 ι Eine Folterkammer für Zellen

Christian Müssgens ι FAZ

Beiersdorf will herausfinden, was unsere Haut altern läßt. Der Nivea-Konzern bekommt dafür Steuergeld aus einem Förderprogramm für die Lichtforschung.

Press Article in German