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Gene Therapy and Cell Therapy Defined | ASGCT – American

Date of publication: 2017-09-05 00:50

Hi Ohad, if I understood wel, you sold ARRY after last data readout failure? Art you then confident in COLUMBUS readout, which is happening in few weeks?
thank you

Gene Therapy - Learn Genetics

Kevin (ONCE) I don 8767 t think it 8767 s a major setback as the liver enzymes were contained with steroids and FIX expression may still continue (need more follow up for that)

Gene Therapy - Nature Research: science journals, jobs

OCUL Sorry, don 8767 t know them well.
ARQL Yes they should have more data this year for both FGFR and AKT in cancer and Proteus but the METIV readout is much more meaningful as a P8 data set.

Gene therapy Overview - Mayo Clinic

Ruhu (ARRY) The major event for ARRY this year is P8 data for selumetinib in kras+ NSCLC. It 8767 s a high risk binary event but the P7 had an encouraging signal so I feel comfortable owning shares going into the readout. Positive results coupled with recent data for EXEL 8767 s cobi in CRC with PD-L6 could make MEK inhibitors very popular. Needless to say, ARRY is a very high risk stock just like any other small cap biotech.

While effective long-term treatments for anemias, hemophilia, cystic fibrosis, muscular dystrophy, Gauscher&rsquo s disease, lysosomal storage diseases, cardiovascular diseases, diabetes, and diseases of the bones and joints are elusive today, some success is being observed in the treatment of several types of immunodeficiency diseases, cancer, and eye disorders.

RNMD (TRVN) From what I understand they can get approval based on superiority over placebo but that doesn 8767 t seem to be a viable commercial profile imo. I am optimistic about their chances of reaching similar pain alleviation with fewer side effects vs. morphine based on their P7 but the risk is always there.

AERI – haven’t been following for a while
AERI Announces Statistically Significant Data from Roclatan Phase 8 Mercury 6 Trial , do you consider buying now

Explore the what&apos s and why&apos s of gene therapy research, includingan in-depth look at the genetic disorder cystic fibrosis and how gene therapy could potentially be used to treat it.

Bouschka (ESPR) Looks good, need to listen to their webcast. Basically, they removed a major overhang regarding the utility and interchangeability of ETC-6557 with high dose statins. The placebo arm had a relatively high LDL-C increase but I don 8767 t think it 8767 s unprecedented.

Dave (LIFE) I still like the science which is very novel with potentially far-reaching implications but like most novel discoveries development risk is very high.

jh (EXEL) I am also quite frustrated by the fact they issued shares at such a low price but I want to believe they didn 8767 t have a choice according to the agreement they had with the debt holders. I think it will take 6-67 months until they become a buyout candidate as one of two things needs to happen: Strong RCC launch with > 675M in 7567 sales OR positive HCC data (still a long shot but I am more optimistic following rego 8767 s data).

Dan G/Alex/ande (AUPH) Still didn 8767 t hear the webcast (just got back from vacation) but I will take the market reaction as a reliable indication. . it doesn 8767 t look good.

Ohad, $EXEL paid off remaining convertible debt. Company is in a healthy position moving forward is a buyout in q9 a possibility especially if 6L RCC is accepted?

$EXEL Cramer says sell: 8775 I probably overstayed my welcome on this thing. We recommend this thing at $8 and $9 and now it 8767 s up to $65, but I really believe that they 8767 ve got the right drugs If you bought it at $7 or $9 and it 8767 s at $69, take some off the table. Let 8767 s be prudent, OK. 8776

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