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May 16, 2019
 |  BC Innovations  |  Product Development

Abeona tackles gene therapy’s problems in cystic fibrosis with chimeric AAVs

Why Abeona thinks its gene therapy platform will work for cystic fibrosis where others fell short

As it builds up its preclinical pipeline, Abeona is switching gears to create chimeric AAV vectors that it hopes will do a better job than naturally occurring ones of dialing in the right properties for the right indication. A big win would be to finally gain gene therapy a foothold in cystic fibrosis.

On April 30, Abeona Therapeutics Inc. presented data at the American Society for Cell and Gene Therapy meeting in Washington, showing one of the company’s chimeric AAV capsids can deliver a functional copy of the CFTR gene to mouse lungs and can correct chloride ion transport in airway cells from cystic fibrosis (CF) patients.

Abeona has been developing gene therapies for rare diseases since its inception, but it began with non-engineered vectors. Launched as Abeona Therapeutics LLC in 2013, the company was acquired by PlasmaTech Biopharmaceuticals Inc. in 2015 and went public the same year under its new name Abeona Therapeutics Inc.

Its lead program, which is expected to enter Phase III mid-year, uses a retroviral vector to deliver the COL7A1 gene to treat the rare skin disorder recessive dystrophic epidermolysis bullosa. It has two Phase I/II programs and one preclinical program that use an AAV9 vector in-licensed last year from RegenxBio Inc.

Abeona’s next growth phase involves deploying its home-grown chimeric AAV technology to build out its preclinical pipeline. The mix-and-match vectors, dubbed AIMs, piece together parts of different AAV capsids to create chimeras with desired delivery properties.

The company believes the platform will enable it to crack cystic fibrosis, an indication that has seen over 25 years of gene therapy trials but not a single approval.

The reasons for the failures range from severe immune reactions to a host of lung delivery challenges. The lungs are well-equipped to fend off viruses, and the thick mucus in CF patients creates a physical barrier between the vectors and the cells they need to enter. Formulating an efficient aerosolized gene therapy to reach airway cells is complicated.

Given the lower levels of pre-existing immunity and the ability to tune tropism to particular tissues, including the lung, CSO Timothy Miller thinks Abeona’s engineered capsids could yield a step change in efficacy in CF and other...

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