Replies to post #200301 on Biotech Values

[jellybean]

Gene therapy is a temporary fix intended to deliver a product by the presence of an extrachromosomal entity such as a virus or plasmid. The entity does not integrate into the genome and is diluted during cell division. That means over time the efficacy wanes and so the expectation is that the patient will need further treatment. BLUE uses the AIDS virus to integrate a copy of a gene into the genome. This is a permanent fix as the introduced gene is copied as a part of the chromosome and so is passed into each of the daughter cells. SGMO is also using an alteration to the genome that will be inherited into each daughter cell. This is a fundamental difference between gene therapy and genome editing.

[biocqr]

BLUE > up 9% on stem cell manufacturing improvement ...

Bluebird bio adds a key process to pioneering gene therapy as it ramps up a pivotal PhIII

http://endpts.com/bluebird-bio-adds-a-key-process-to-pioneering-gene-therapy-as-it-ramps-up-a-pivotal-phiii/

Today, bluebird says it has begun the pivotal Phase III study for beta-thalassemia. And Davidson tells me that they’re making a crucial correction in the manufacturing process, adding two unidentified small molecules — a pair of “transduction enhancers” added to its stem cell manufacturing process — that the bellwether biotech has evidence to believe can have a marked impact on the vector copy number and the potential to cure its top slate of disease targets.

The market bid up bluebird’s shares by 9% Thursday morning.

“We’re very excited about the two small molecules,” Davidson tells me this morning, with in vitro evidence of an improvement in the vector copy numbers as well as in vivo data from a mouse model to back it up.

“We’re planning to apply the same manufacturing process with the improvement across the LentiGlobin program, including sickle cell,” he adds. That involves the pivotal study in patients with transfusion-dependent beta-thalassemia and non-ß0/ß0 genotypes as well as an upcoming study with ß0/ß0 genotypes.

Bluebird isn’t revealing exactly what these two molecules are, in part for competitive reasons, says Davidson. They’ll be able to reveal more about it at ASH at the end of the year, and the investigator says that they’ll be able to get firm patient responses 6 to 12 months out, making this more of a 2017 story.

It’s particularly significant for bluebird that the company can proceed right into Phase III without getting a new IND, says Wedbush’s David Nierengarten, who’s bullish on the company’s prospects.