• Sign In
  • Sign Out
  • My Account
Subscribe
  • BioWorld
  • BioWorld MedTech
  • BioWorld Asia
  • BioWorld Science
  • Data Snapshots
  • Special reports
Clarivate
  • Data Snapshots
  • BioWorld
  • BioWorld MedTech
  • Infographics: Dynamic digital data analysis
  • Index insights
  • Special reports
  • Infographics: Dynamic digital data analysis
  • Trump administration impacts
  • Biopharma M&A scorecard
  • BioWorld 2024 review
  • BioWorld MedTech 2024 review
  • BioWorld Science 2024 review
  • Women's health
  • China's GLP-1 landscape
  • PFA re-energizes afib market
  • China CAR T
  • Alzheimer's disease
  • Coronavirus
  • More reports can be found here

BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld MedTech
  • BioWorld Asia
  • BioWorld Science
  • Data Snapshots
    • BioWorld
    • BioWorld MedTech
    • Infographics: Dynamic digital data analysis
    • Index insights
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • Biopharma M&A scorecard
    • BioWorld 2024 review
    • BioWorld MedTech 2024 review
    • BioWorld Science 2024 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Coronavirus
    • More reports can be found here

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe
Home » gene therapies

Articles Tagged with ''gene therapies''

Lysogene reveals patient death in phase III Sanfilippo syndrome study

Oct. 15, 2020
By Michael Fitzhugh
One of 19 children dosed with an experimental gene therapy for Sanfilippo syndrome has died following the treatment, given as part of a phase II/III trial of the medicine run by its developer, Paris-based Lysogene SA. The trial has been on clinical hold since June, but the immediate cause of the death is unknown, the company said.
Read More
Central nervous system

Dyno sparks a $1.8B deal with Roche to develop CNS therapies

Oct. 14, 2020
By Lee Landenberger
Privately held Dyno Therapeutics Inc., an early stage biotech company applying artificial intelligence to gene therapy, entered a collaboration and license agreement with Spark Therapeutics Inc. that could bring Dyno milestone payments exceeding $1.8 billion.
Read More
Bar chart, downward arrow

Biopharmaceutical sector suffers from the summer blues

Aug. 31, 2020
By Peter Winter
It has proved to be a lackluster summer for the biopharmaceutical sector, with the BioWorld Biopharmaceutical index dropping about 2% in value during the past two months, in contrast to the general markets that have enjoyed a much stronger period.
Read More
DNA in test tubes

Carmine inks $900M+ deal to develop nonviral gene therapies for Takeda

July 7, 2020
By Michael Fitzhugh
Carmine Therapeutics Inc. said Tuesday that Takeda Pharmaceutical Co. Ltd. has engaged it in a research collaboration aimed at the discovery, development and commercialization of new nonviral gene therapies for two rare disease targets.
Read More
Person in wheelchair

Sarepta enlists Hansa to clear the way for AAV gene therapy

July 2, 2020
By Michael Fitzhugh
Shares of Sweden-based Hansa Biopharma AB (NASDAQ:HNSA) jumped 32.7% to SEK53.90 (US$5.81) on July 2 as Sarepta Therapeutics Inc. became the first partner to license its lead asset, imlifidase, to enable gene therapies to treat muscular dystrophy patients harboring neutralizing antibodies to the adeno-associated virus (AAV) vectors the medicines employ.
Read More
Connecting puzzle pieces

CSL buys rights to Uniqure's late-stage gene therapy for hemophilia B

June 30, 2020
By Michael Fitzhugh
CSL Behring has licensed global rights to Uniqure NV's late-stage hemophilia B gene therapy candidate, AMT-061, for $450 million up front, plus up to $1.6 billion in potential milestone payments and royalties. Uniqure positioned the deal as a boon for its broader gene therapies pipeline, anchored by a Huntington's disease (HD) program. But the move appeared to sour investors hoping for a rumored near-term M&A takeout, sending company shares (NASDAQ:QURE) falling 21.8% to $49.22 on June 25.
Read More
DNA in test tubes

Carmine inks $900M+ deal to develop nonviral gene therapies for Takeda

June 30, 2020
By Michael Fitzhugh
Carmine Therapeutics Inc. said Tuesday that Takeda Pharmaceutical Co. Ltd. has engaged it in a research collaboration aimed at the discovery, development and commercialization of new nonviral gene therapies for two rare disease targets. Terms of the deal included an up-front payment of undisclosed value for Carmine, plus research funding, and more than $900 million in potential milestone payments.
Read More

Second close pushes Freeline’s series C round to $120M

June 30, 2020
By Cormac Sheridan
DUBLIN – Gene therapy developer Freeline Therapeutics Ltd. added $80 million in new investment to take its series C round to $120 million in total. The new cash will help to fund a pivotal trial of its lead gene therapy program in hemophilia B, enable it to continue a phase I/II trial of a gene therapy in Fabry disease and allow it to progress its preclinical programs in Gaucher disease and hemophilia A, while also making ongoing investments in its adeno-associated virus (AAV) gene therapy technology and its manufacturing platform.
Read More
IPO, coins, financial chart

Twin peaks: Akouos, Fusion go high in matching $212M IPOs

June 26, 2020
By Randy Osborne
A pair of upsized IPOs packed away cash by way of identical terms, with Boston-based Akouos Inc. and Fusion Pharmaceuticals Inc., of Hamilton, Ontario, separately selling 12.5 million shares at $17 each for $212.5 million in gross proceeds.
Read More
Connecting puzzle pieces

CSL buys rights to Uniqure's late-stage gene therapy for hemophilia B

June 25, 2020
By Michael Fitzhugh
CSL Behring has licensed global rights to Uniqure NV's late-stage hemophilia B gene therapy candidate, AMT-061, for $450 million up front, plus up to $1.6 billion in potential milestone payments and royalties. Uniqure positioned the deal as a boon for its broader gene therapies pipeline, anchored by a Huntington's disease (HD) program. But the move appeared to sour investors hoping for a rumored near-term M&A takeout, sending company shares (NASDAQ:QURE) falling 21.8% to $49.22 on June 25. Despite the market reception, analyst reaction was largely supportive of the move, embracing CSL as a natural fit for the program and, in the words of SVB Leerink analyst Joseph Schwartz, appreciating the transfer of "any potential remaining risk in hemophilia B to CSL Behring as QURE rings the cash register."
Read More
Previous 1 2 3 4 5 6 7 8 9 10 Next

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for July 14, 2025.
  • Today's news in brief

    BioWorld MedTech
    BioWorld MedTech briefs for July 14, 2025.
  • Scientific figure illustrating astrocytes and neuron synapse

    Glia 2025: Microglial crosstalk could be early Alzheimer’s targeting opportunity

    BioWorld
    “Loss of synapses and dysfunctional synapses in a region-specific way is important in Alzheimer’s. It’s actually the strongest correlate of cognitive decline, far...
  • New oral BRD4 inhibitor blocks pathogenic expansion of epithelial progenitors in IPF models

    BioWorld Science
    Idiopathic pulmonary fibrosis (IPF) is a progressive and fatal lung disease with limited treatment options. IPF is thought to result from repeated microinjuries...
  • Merger-arrows-black-white.png

    BD’s biosciences & diagnostics unit in $17.5B merger with Waters

    BioWorld MedTech
    Three months earlier than an expected update on a deal, Becton, Dickinson and Co. reported its biosciences and diagnostic solutions business will combine with...
  • BioWorld
    • Today's news
    • Analysis and data insight
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld MedTech
    • Today's news
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld Asia
    • Today's news
    • Analysis and data insight
    • Australia
    • China
    • Clinical
    • Deals and M&A
    • Financings
    • Newco news
    • Regulatory
    • Science
  • BioWorld Science
    • Today's news
    • Biomarkers
    • Cancer
    • Conferences
    • Endocrine/Metabolic
    • Immune
    • Infection
    • Neurology/Psychiatric
    • Patents
  • More
    • About
    • Advertise with BioWorld
    • Archives
    • Article reprints and permissions
    • Contact us
    • Cookie policy
    • Copyright notice
    • Data methodology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • Podcasts
    • Privacy policy
    • Share your news with BioWorld
    • Staff
    • Terms of use
    • Topic alerts
Follow Us

Copyright ©2025. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing

BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld MedTech
  • BioWorld Asia
  • BioWorld Science
  • Data Snapshots
    • BioWorld
    • BioWorld MedTech
    • Infographics: Dynamic digital data analysis
    • Index insights
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • Biopharma M&A scorecard
    • BioWorld 2024 review
    • BioWorld MedTech 2024 review
    • BioWorld Science 2024 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Coronavirus
    • More reports can be found here

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe