Equity Field Notes from temporal sources

[DanWebzster]

ADVM ( and AAV Gene Therapy)

ADVM is conducting a Ph1 dose escalaton study using AAV to deliver Alpha-1 Antitrypsin gene for early onset emphysema.

If their serotype-payload cassette-immunogenic purity platform work in this application, they may have a blockbuster platform on their hands.

On 3/31/18 their cash runway thru 2019


On Gene Therapy:

Gene therapies target monogenic diseases (those diseases with a single damaged or missing gene)

12/19/17, ONCE received the first FDA approval for a gene therapy. Luxterna is treats a congenital condition of the eye. Luxterna costs $850K to treat both eyes. Luxterna delivers a gene rather than modifying or editing one.

Luxterna ues adeno-associated virus (AAV ) as a vector to deliver this gene. AAV has low immunogenicity and is customizable with regard to its payload.

For companies researching AAV, the secret sauces are the specific serotype, the expression cassette and the purity of the vector preparation. Serotype determines tissue affinity.