Scrip is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By


bluebird bio LLC

This article was originally published in Start Up

Executive Summary

bluebird bio LLC has taken in $65 million in venture capital over the past year, thanks to encouraging studies with two different kinds of lentiviral vectors that transfer new copies of genes into patients' own stem cells. Given that vectors have different properties - one integrates into the DNA coding region, the other does not - the company might eventually be able to mix and match therapeutic gene "fillings" to vectors as needed. The start-up is ready to start commercializing gene therapies for serious genetic disorders, starting with ALD and beta-thalassemia. .

You may also be interested in...

Bluebird’s Share Price Skyrocketing On Beta Thalassemia Optimism

On the strength of interim Phase I/II data presented at ASH and an investor presentation Dec. 10, bluebird bio is enjoying substantial growth in its stock price for the week.

Bluebird’s Improved Vector Yields Promising Early Phase II Data In Beta Thalassemia

Two patients achieve independence from transfusions and show normalizing levels of hemoglobin in Phase II trial of experimental viral vector-based gene therapy. Bluebird’s share price soared on the promising news.

Ultragenyx Pharmaceutical LLC

Biotech veteran Emil Kakkis founded Ultragenyx Pharmaceutical LLC to develop treatments for rare disorders. First up: a potential therapy for hereditary inclusion body myopathies. There are several variants of this neuromuscular disorder, which causes progressive muscle weakness. By the end of the disease course, patients can be almost quadriplegic in terms of motor function. Ultragenyx is betting it can help restore muscle function in people diagnosed with HIBM by providing a continuous and steady source of sialic acid, likely through thrice-daily oral administration.

Related Content


Related Companies

Related Deals




Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

Thank you for submitting your question. We will respond to you within 2 business days. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts