News

Warning about gene editing

Country
United Kingdom

Researchers at the Wellcome Sanger Institute in the UK have reported unexpected genetic changes in mouse embryonic stem cells following the use of the CRISPR-Cas9 gene editing tool. They warn that the scientific community may be overestimating the potential for gene editing as a human therapy, and urge that more work be done to better understand the safety profile of the technology.

Abzena extends antibody agreement with Telix

Country
United Kingdom

Abzena Plc, the UK service provider, is to manufacture antibodies for two prospective radiopharmaceuticals being developed by Telix Pharmaceuticals Ltd of Australia for the treatment of prostate cancer, one of the four most common cancers in the world.

FDA issues new guidance on gene therapies

Country
United States

With three gene therapies on the US market, the Food and Drug Administration has provided six new guidance documents for developers to clarify the regulatory standards for these new medicines.

Three of the new guidance documents relate to regulatory standards for manufacturing and three are product specific. The product-specific guidelines are intended to assist developers of prospective gene therapies for haemophilia, retinal disorders and rare diseases.

First drug for smallpox

Country
United States

To address the risk of bioterrorism, the US Food and Drug Administration has approved a drug for the treatment of smallpox, an acute contagious viral disease that was declared eradicated in 1980, but could still be reintroduced into the environment at a time of conflict.

“To address the risk of bioterrorism, Congress has taken steps to enable the development and approval of countermeasures to thwart pathogens that could be employed as weapons,” Scott Gottlieb, the FDA commissioner, announced on 13 July.

Promising results from mosaic HIV-1 vaccine

Country
United States

An international research group has reported promising data from an early clinical trial of a prophylactic vaccine against HIV-1 in healthy volunteers and a parallel study of the same vaccine in rhesus monkeys. The study results were published online in The Lancet on 6 July.

Forbion raises €270 million for fourth fund

Country
Netherlands

Forbion Capital Partners of the Netherlands has raised €270 million at the first close of its fourth life science fund, exceeding the target amount of €250 million. Forbion IV will support promising biotech companies predominately in the UK and the EU.

About 20% of the funds will target opportunities in North America.

Wellcome to fund ‘blue-sky’ research

Country
United Kingdom

The Wellcome Trust is to use its expertise in life science and financial muscle to launch a new investment fund that will enable high-risk research projects to develop quickly and move towards commercialisation. The Wellcome Leap Fund will have capital of £250 million to be allocated over five years starting in 2020.

The fund will be wholly owned by the Trust with its own chief executive officer and an independent board of directors. Recruitment of the CEO is expected to start shortly, the Trust announced on 10 July.

Abingworth raises $315 million for new fund

Country
United Kingdom

London, UK-based Abingworth has raised $315 million for its twelfth life science fund bringing the total amount of funds under management to over $1.2 billion. The new fund, Abingworth Bioventures VII, will invest in companies in Europe and the US.

Abingworth invests across all stages of drug development including early and late-stage venture deals, clinical co-development projects and public equities. The typical investment per company is in the range of $15 to $30 million.

New support for amyloid hypothesis in Alzheimer’s disease

Country
United States

A prospective drug for Alzheimer’s disease that failed to show efficacy in late 2017 based on a Bayesian analysis of patient data, has now demonstrated its promise using more conventional statistical methods, according to Eisai Co Ltd and Biogen Inc.

The drug, BAN2401, is a monoclonal antibody targeting beta-amyloid, protein fragments in the brain which are thought to contribute to the neurodegenerative process in Alzheimer’s disease.

€47 million for oligonucleotide candidate

Country
France

A consortium of predominately European investors is providing €47 million to Dynacure SAS of France to bring its candidate antisense oligonucleotide drug into the clinic for the treatment of patients with centronuclear myopathies.

Centronuclear myopathies are genetic disorders characterised by muscle weakness and wasting in the skeletal muscles. Dynacure’s lead product, Dyn101, is intended to treat these disorders by targeting dynamin 2, a protein that is implicated in cell processes such and cell motility and endocytosis.