Please login to the form below

Not currently logged in

Biogen bulks up in Alzheimer’s again with $2.7bn Sangamo deal

Gains rights to tau-targeting drug ST-501 as part of broader alliance


Biogen has made another big investment play in Alzheimer’s disease, penning a $350m upfront agreement with gene-editing specialist Sangamo for rights to tau-targeting drug ST-501 as part of a broader R&D alliance.

The tie-up also covers a second drug called ST-502 – in development for Parkinson’s disease and other neurodegenerative diseases characterised by the abnormal clumping of alpha-synuclein protein (synucleinopathies) – as well as a third unnamed candidate for a neuromuscular disease. Biogen also claims exclusive rights to nine additional undisclosed neurological targets.

The deal is sizeable considering both ST-501 and ST-502 are still in preclinical development, with the upfront payment – split between $125m in cash and $225m in new Sangamo stock – backed up by $2.37bn in milestones tied to development, regulatory, and commercial targets.

Tau is emerging as a target for drug development in Alzheimer’s as well as other disorders like frontotemporal dementia.

The licensing deal comes as Biogen prepares to file its amyloid-targeting Alzheimer’s therapy aducanumab, which had been written off last year after disappointing clinical trial results but was resurrected after it appeared to show a benefit on longer-term follow-up.

Sangamo’s specialises in the development of gene-editing drugs based on its zinc finger nuclease (ZFN) technology, which can be used to regulate the expression of genes but had disappointing results in a mucopolysaccharidosis type II trial last year.

Biogen’s deal is an endorsement of the technology platform at a time when rival gene-editing technologies like CRISPR/Cas9 seem to be gathering momentum, with encouraging preliminary results from  trial of Vertex and CRISPR Therapeutics’ CTX001 in blood disorders last November.

“As a pioneer in neuroscience, Biogen will collaborate with Sangamo on a new gene regulation therapy approach, working at the DNA level, with the potential to treat challenging neurological diseases of global significance,” said Al Sandrock, Biogen’s head of R&D.

Sangamo’s zinc finger protein transcription factor (ZFP-TF) approach involves introducing double-stranded DNA breaks engineered to target specific sequences in a cell.

The cell tries to fix the break using another copy of the sequence as a backup, such as the other unbroken chromosome in the pair but – by supplying a new template using an adeno-associated virus (AAV) vector – the system can be forced to insert a desired sequence instead.

“Highly specific, potent, and tunable repression of tau and alpha synuclein has been demonstrated in preclinical studies using AAV vectors to deliver tau-targeted (ST-501) and alpha synuclein-targeted (ST-502) ZFP-TFs,” according to the partners.

Sangamo also has a gene therapy platform, with a first candidate for haemophilia A heading for late-stage development at partner Pfizer later this year.

Article by
Phil Taylor

28th February 2020

From: Sales



COVID-19 Updates and Daily News

Featured jobs


Add my company
Real Science Communications

Real Science Communications is a scientific centre of excellence, infusing credibility and scientific rigour into the conversations around health and...

Latest intelligence

The Pivot
In times of uncertainty it is critical for businesses to take action to protect their brand image and longevity....
Peter Jackson
The global antibiotic crisis: a 2020 perspective
A report from the CDC says the ‘approaching’ post-antibiotic era is already here...
Women in science
The role of women in science and technology
Why the lack of female leaders in pharma and life sciences is a problem that still needs to be fixed...