Gene therapy upstart Avrobio raises $25M for Fabry disease, AML trials

Life science VC firms have stumped up $25 million in a Series A raise for Cambridge, MA-based biotech Avrobio as it looks to take its gene therapy work into midstage tests. 

The startup is currently working on early trials in Fabry disease and acute myeloid leukemia (AML) using lentiviral-based gene therapies. Perennial investor Atlas Venture led the round, followed by Clarus and SV Life Sciences. The biotech will use the cash to fund its Phase I studies.

“Avrobio’s highly innovative therapies offer potentially life-altering impact for patients following a single infusion of genetically-modified cells,” said Geoff MacKay, its president and CEO.

“We are grateful for the funding and support we have received from our investors, as we continue to focus on displacing the standard of care for patients with Fabry disease or AML through the development of these disruptive gene therapies.”

Gene therapies have had a tough few years, with uniQure’s ($QURE) Glybera being knocked back by the FDA and struggling to gain any traction in Europe, where it is approved--but with a $1 million price tag.

GlaxoSmithKline ($GSK) recently gained European approval for its ultra-rare gene therapy Strimvelis, but as each drug is tailor-made for each patient, a launch in the traditional sense hasn’t happened, and it is still assessing just how it should price its med. There have also been a lot of promise but few genuine breakthroughs in the lab, with a number of gene therapy programs cut in recent years. 

But it’s still an area of research that many do not want to abandon despite the difficulties and questions over ROI, with Biogen ($BIIB) being one Big Biotech which has been ramping up its time and investment in the area. Pfizer ($PFE) and BioMarin ($BRMN) are also following suit.

Avrobio’s Phase I gene for Fabry disease seeks to use a patient’s stem cells, which are extracted and genetically modified by adding a new, functional copy of the faulty gene. The modified cells are then infused back into the patient via a one-time procedure.

And for its AML treatment, the biotech is seeking to recruit the patient’s own immune system to better detect and eradicate cancer cells.

After initial chemotherapy, some of the patient’s lingering cancer cells are taken out and genetically modified to express IL-12--a powerful activator of the immune system. The modified cells are then put back into the patient in a one-time procedure, with a goal to kickstart an anti-cancer immune response against remaining AML blasts in the patient’s body.

There are already treatments on the market for both conditions, so Avrobio will need to prove high efficacy, and likely come with a lower price, should it gain approval in the future for these meds.

- check out the release

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