News

Gene therapy back on track
Enlarge image

BusinessNetherlands

Gene therapy back on track

23.07.2012 - A ray of hope for Dutch gene therapy specialist uniQure B.V.: EMA's CHMP
recommended approval of its product Glybera to treat lipoprotein lipase (LPL) deficiency in patients with severe or multiple pancreatitis attacks.

EMA said Glybera (alipogene tiparvovec) is the first gene therapy drug to receive a positive CHMP recommendation. The news is a relief for Amsterdam-based uniQure, after having received three negative opinions before. CHMP said it reversed its prior negative opinions following a draft positive opinion in June from the Committee for Advanced Therapies (CAT),which conducted additional analyses of the data for the adeno-associated virus (AAV) vector encoding LPL gene. CHMP said Glybera should be approved under EMA's exceptional circumstances clause, and that the company be required to establish a patient registry to monitor outcomes. uniQure expects a final decision from the European Commission within three months.

UniQure's predecessor, Amsterdam Molecular Therapeutics Holding N.V., first applied for European approval of the gene therapy in December 2009 for all patients with LPL deficiency. CHMP subsequently issued three negative opinions for Glybera, the most recent of which came in April following arequest by the EC to consider the product in a restricted patient population with severe or multiple pancreatitis attacks. Amsterdam Molecular dissolved into uniQure this February.

LPL disease is a very rare, inherited disease which affects 1-2 people per million. Patients with the disease are unable to handle fat particles in their blood plasma, which leads to recurring severe abdominal pain andpancreatitis. "After today's positive recommendation, Glybera is poised tobecome the first in a class of gene therapy products approved in Europe to treat orphan diseases, rare conditions with a very high unmet medical need", Jörn Aldag, CEO of uniQure, said.

http://www.european-biotechnology-news.com/news/news/2012-03/gene-therapy-back-on-track.html

Stock MarketsFranceEU

27.03.2015 Europe is once more proving that it can be a good bet for biotech companies going public. A newly established EU-wide lobbying initiative could help the IPO boom on its way.

FinancingPoland

24.03.2015 Poland has announced the closing of a new venture capital fund at US$42m (€32m). The fund, which is the first of its kind in Poland, is dedicated to the Life Sciences with a strong connection to Central and Eastern Europe.

BioeconomyFranceSwedenGermanyNetherlandsFinland

23.03.2015 A BASF-led consortium aims to decrease bio-based production costs using industrial biotechnology. The Horizon2020 funded consortium is the sixth project of the SPIRE Public Private Partnership and has a budget of €14m.

Stock MarketsFranceSwedenUKNorwayIreland

20.03.2015 There are no signs of inertia creeping into the European biotech industry this spring. A total of eight IPOs are set to go public by the end of the year’s first quarter.

FinancingUKEU

17.03.2015 Pharma giants GlaxoSmithKline (GSK) and Johnson & Johnson (J&J) will be key collaborators in a new global fund for dementia research to address the rising threat posed by dementia and Alzheimer’s disease.

FinancingFranceDenmarkSwedenGermany

19.03.2015 French venture capital firm Seventure Partners has raised a further €25m for its microbiome-focused Health for Life Capital Fund. It now stands at €100m.

EventsFranceEUSwitzerlandUKGermany

12.03.2015 With around 1,400 companies and 2,400 participants, the springtime counterpart of BIO-Europe enjoyed a successful premiere in France this year. The overall mood of the European biotech industry was positive, and the French hosts in particular reported new initiatives.

M&ABelgium

11.03.2015 Belgian cell-based therapeutics developer and CMO MaSTherCell SA has been acquired by US biotech Orgenesis Inc. for €23m.

FinancingGermany

10.03.2015 The Gates Foundation has dipped into its purse to bring infectious diseases vaccines to the poorest countries. German vaccine specialist CureVac is set to receive US$52m for their mRNA platform technology.

Stock MarketsIrelandUK

05.03.2015 The IPO wave in the biotech sector shows no signs of slowing down. An Irish life sciences fund set up by former Elan executives plans to raise €325m with its IPO, whilst Summit Therapeutics has announced its IPO pricing of US$34m on Nasdaq.

Events

All Events

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • 4SC1.28 EUR52.38%
  • THERAMETRICS0.08 CHF14.29%
  • WILEX2.96 EUR1.37%

FLOP

  • MOLOGEN5.68 EUR-28.01%
  • MORPHOSYS54.71 EUR-22.45%
  • MEDIGENE11.65 EUR-13.70%

TOP

  • CYTOS1.40 CHF311.8%
  • MEDIGENE11.65 EUR174.1%
  • 4SC1.28 EUR64.1%

FLOP

  • MORPHOSYS54.71 EUR-26.8%
  • WILEX2.96 EUR-13.7%
  • BASILEA106.70 CHF-12.7%

TOP

  • SANTHERA92.00 CHF2259.0%
  • WILEX2.96 EUR393.3%
  • FORMYCON23.15 EUR232.6%

FLOP

  • CYTOS1.40 CHF-54.2%
  • MOLOGEN5.68 EUR-49.9%
  • PAION2.35 EUR-29.6%

No liability assumed, Date: 26.03.2015