Replies to post #223310 on Biotech Values

[linhdtu]

if indeed so, imo it's then the case of risk/reward. maybe not at the current valuation, but since it's obviously hard and unproven ,yet clinically i mean, to hack into human genome, whomever succeeds first well ...

also the requirement for a so called success may not be so high either. at this stage , safety is first and foremost, and any sign of durable effect is icing on the cake lol

[CHM_760]

re: Gene Edit and Gene Therapy:

I'm just curious if anyone on this board is watching Sangamo (SGMO) and their Zinc Finger platform? They are in the clinic with multiple programs and multiple partners, including PFE and GILD. Not CRISPR based.

Most recent Barclay's writeup:

Barclays - SGMO: Key Takeaways from Management Meeting (Overweight TP 30)

Key takeaways: We met with SGMO management and gained additional color on the progress and upcoming catalyst of clinical programs. SGMO would have a catalyst-rich 2019, with MPS I/II safety and biochemical data at WORLDSymposium (Feb 7) and other data through the year (to include ERT withdrawal and biopsy results), as well as initial data from the hemoA, hemoB and beta-thalassemia programs. Early stage programs are progressing well with a new cohort for the MPS II program using ZFN2.0 (5-30X improvement), and Ph1 initiation for the Fabry disease program, Treg program for solid organ transplant and the SCD program.

MPS I & II update at WORLDSymposium (Feb 7) to provide insight on in vivo gene editing potential. For the MPS II program, 24 week safety and biochemical data of 6 patients from all 3 cohorts (5E12, 1E13, 5E13 vg/kg) will be reported including GAG and IDS level. Management noted more sensitive assay was developed to measure IDS activity below current lower limit of detection (5.2 nmol/hr/mL). Consistent with prior guidance, ERT withdrawal and biopsy data would be reported later in 2019. For the MPS I program, up to 4-week data for first 3 patients will be reported. The company is currently enrolling the expansion cohort (2 of 3 patients enrolled) and adolescent cohort for MPS II.

Additional catalysts in 2019: 1) SB-525 Ph1/2 study in hemoA has entered the 4th dose cohort (1 patient dosed with another to be dosed imminently) and the SMC would review the 4th cohort data to decide if further escalate the dose or start an expansion cohort. Management believes initial SB-525 data would become available in 2019. 2) SB-FIX Ph1/2 study has enrolled the first patient at high dose (5E+13vg/kg) and initial data are expected in 2019. 3) MPS I/II programs would add a new cohort using the next generation ZNF2.0 (5-30x better efficiency) and patient enrollment is expected to start in 2019 (starting dose not disclosed). 4) The ST-400 Ph1/2 study for beta-thalassemia has enrolled 2 patients (unclear if have been dosed) and management plans to complete enrollment of 6 patients with initial data likely in 2019. Partner Sanofi would start the Ph1/2 study for SCD this year. 5) ST-920 Ph1/2 study is expected to start in 2019. 5) The CAR-Treg program Tx200 CTA filing and Ph1 initiation are expected in 2019 for solid organ transplant.