More current and relevant then third quarter statements from 2012 is this information from ACTC January 2013 power point. This is why this is an exciting company to watch!
About their current trials.
No Adverse Events -No signs of hyperproliferation -No signs of abnormal growth, -No signs of rejection or retinal detachment.
Persistence of cells -Anatomical evidence of hESC-RPE survival and engraftment. -Increased pigmentation within the bed of the transplant.
Impact on Acuity -Recorded functional visual improvements in both patients
at a 14 month follow up • Visual acuity gains remain relatively stable for both patients • SMD Patient continues to show improvement.
U.K. SMD01 Patient (at 9 month follow-up) • ETDRS: Improved from 5 letters to 10 letters, and stable • Subjective: Reports significantly improved ability to read text on TV
Why its an exciting investment? • Immense unmet medical need • Small Doses • Immunoprivileged – permits central (allogeneic) source of cells • Noninvasive monitoring of retina Market potential: More than 50 million patients in major markets. 1% market penetration may represent $5-10B market opportunity. Orphan indications are meaningful: Estimating a 10% market penetration with reoccurring treatments every 3-5 years, Stargardt’s disease can be a $100+ million/year product.
-has enough cash on hand up to 2015 (I cant remember where i read this)
-Applying for NIH funding, i believe they are applying for NIH "grants", which do not have to be repayed as long as they meet the requirements. However they might be applying for a NIH loan,in that case it does have to be paid back.
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