esibio general sales presentation august update
TRANSCRIPT
HyStem, Glycosil, Premvia
HES cell lines, ESI cell lines
Products
510 (k) approval, phase II clinical trial
First hES line available GMP
Notable Achievements
PureStem Progenitors, BioLite Antibodies, Small Molecules
Commercial release of small molecules, UV HyStem kit and BioLite.
HYSTEM HYDROGELS3D Cell Culture and Cell Delivery Vehicle
Should your cells be cultured in 3D?• What are the differences
between 2D and 3D?• Shape
• 3D- cells are in ellipsoids 10-30 µm
• 2D- cells are flat with 3 µm thickness
• Environment• 3D- cells have ~100% of
surface area in contact with cells or matrix
• 2D- cells have ~%50 in contact with media and ~50% in contact with plastic.
• Behavior• Morphology• Differentiation• Drug Metabolism• Expression• General Cell Function• Proliferation• Stimulus Response • Viability
Modular Nature of HyStem1. Components
1. Glycosil2. Heprasil3. Gelin-S4. Laminin, Vitronectin,
Fibronectin, RGD peptide, etc…
5. FGF, TGF, VEGF, etc…2. Crosslinkers
1. Extralink2. PEGSSDA3. PEG Norbornene
3. Swelling Agents1. DG water2. Lactated Ringers
4. Curing Agents1. PEGcure
5. Reactive Process1. Covalent bonds formed at
RT with biological pH
Evaluating Drug Efficacy and Toxicology in Three Dimensions: Using Synthetic Extracellular Matrices in Drug DiscoveryGLENN D. PRESTWICH*
Hydrogel Matrix to Support Stem Cell Survival After Brain Transplantation in StrokeS. Thomas Carmichael et al
“The translational imperative: Making cell therapy simple and effective” Prestwich et al, 2012Fig. 4. HyStem increases engraftment of luciferase-labeled human liver progenitors injected into the livers of SCID mice [19]. The upper panel shows that cells grafted inHyStem engraft in the liver and are mostly retained after 72 h. The bottom panel shows that few, if any, cells injected as a suspension in buffer remain in the liver at 72 h.(Reproduced with permission from Hepatology.)
Success Stories
• Cell Delivery– Nueral Stem Cells at UCLA– Islets at Likarda– Hepatocytes at University of North Carolina– Cardiospheres at Cedars-Sinai
• Cell Culture–Organoids at Wake Forest– Fibroblasts at SUNY– Cardiomyocytes at Penn
Liver-Tumor Organoids at Wake Forest
REPROGRAMMING KITRNA reprogramming
ESI BIO RNA reprogramming system
Comparison of non-integrative reprogramming approaches
ESI BIO RNA reprogramming protocol
mRNA reprogramming –morphological progression adult fibroblasts
CELLS & REAGENTShES Cells, Progenitor Cells, Small Molecules, and antibodies
ESI Lines• 5 NIH registered lines
• Available as an economical R&D grade and the first cGMP grade
• R&D grade cultured in Matrigel® and TeSR™-E8™
• Full genomic data available
• Full donor medical records and informed consent avilable
“The Generation of Six Clinical-Grade Human Embryonic Stem Cell Lines” Crook et al, 2007Figure 1. Overview of Activities and Requirements for cGMP hESC Line Derivation, Banking, and CharacterizationThe project commenced with the procurement of suitable embryos, paralleled by the selection, optimization, and verification of protocols inclusive of cGMP-compliant reagents and materials for hESC derivation, culture, cryopreservation, storage, and biosafety testing. Once validated, all protocols were translated to batch record and/or standard operating procedure (SOP) format for cGMP application. Qualified personnel received instruction for protocol execution according to principles of cGMP. On-site audits of selected key organizations/facilities for cGMP compliance were performed. Appropriate documentation confirming cGMP compliance was attained for all other supporting services (e.g., reagent production). cGMP activities were subsequently undertaken within an Australian Therapeutic Goods Administration (TGA) accredited cGMP facility (Q-GEN Pty Ltd) for fibroblast feeder banking followed by hESC line derivation, expansion, and cryopreservation for MCB production. All MCBs were transferred to an accredited facility for cGMP cryogenic storage. After characterization of each line, including biosafety testing, all documentation (exceeding 1000 pages per hESC line) was collated for final review and confirmation of successful cGMP line and bank production.
“Evaluating the genomic and sequence integrity ofhuman ES cell lines; comparison to normal genomes” Funk et al 2011Figure 1 Overview of ES cell analysis strategy. ES lines ESI017, 035, 049, 051 and 053 were expanded using feeder-free culture from early passage frozen stocks. Cultures were evaluated for G-banded karyotype, STR fingerprinting and FISH-based assessment of chromosomes 12 and 17 (Methods). High molecular weight DNA was harvested for telomere length analysis, copy number variation determination and complete genome sequencing.
PureStem Progenitor Cells
