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The state stem cell agency

2012 Disease Team 1 Update

Presentation to ICOC March 21, 2012 Agenda Item # 11

Overview - CIRMs Disease Team 1


Summarize current status of CIRMs 14 Disease Teams as of March 2012
Context of Disease Team programs at CIRM Brief outline of each Disease Team project Key progress and accomplishments to date Status of their progress towards filing a successful IND within 4 years Budget allocated Process for assessment

Disease Teams align with CIRMs mission and clinical strategic objective
CIRMs mission is to advance stem cell research for the discovery and development of cures, therapies, diagnostics and research technologies to relieve human suffering from chronic disease and injury. Key strategy to progress stem cell science into clinical trials, CIRM funded 14 multidisciplinary Disease Teams in 2010
Initiate the development studies, potentially leading to successful filing of an Investigational New Drug (IND) application with the US Food and Drug Administration (FDA) for these stem cell based therapies to enter human clinical trials over the next 4 years

CIRM Programs cover product development spectrum


Candidate Discovery Research Phase 1 Clinical Research Phase 2 Clinical Research

Basic Research Research

Preclinical Research

Preclinical Dev.

Fundamental Biology

Select Preclinical Development Proof of Concept (POC) Candidate (DC)

File IND

Early Translational Research I,II,III Disease Team Research I

Targeted Clinical Development Tools & Technologies Disease Team Research II Transplantation Immunology

CIRM Translational Portfolio


43 grants (Early Translation I, II; Disease Team I)
14 (Disease Team I) target an Investigational New Drug (IND) filing 20 (Early Translational I, II) target identification and selection of a Development Candidate (DC) 9 (Early Translational II) conduct a subset of the studies to identify a development candidate or a feasible development candidate

Cancer and neurodegenerative are the most represented


Cancer (8 awards: 5 Disease Teams & 3 Early Translational) Neurodegenerative (14 awards: 2 Disease Teams and 12 Early Translational)

CIRMs Disease Teams, addressing major unmet clinical needs, are moving towards the clinic
Basic Basic Research Research Discovery Discovery Research Research Preclinical Preclinical Research Research
DEVELOPMENT CANDIDATE

Preclinical Preclinical Dev. Dev.

Clinical Clinical Research Research

Early

FILE IND

Diabetes (1) Neurological Disorders (2) Eye Disease (1) Cardiac Disease (1) HIV/AIDS (2) Cancer (5) Blood Disorders (1) Skin Disease (1)

Disease Team 1 Overview


Grant/PI DR1-01421/Aboody DR1-01426/Berger DR1-01430/Carson DR1-01444/Humayun DR1-01477/Slamon DR1-01485/Weissman DR1-01471/Goldstein DR1-01461/Marban DR1-01431/Chen DR1-01452/Kohn DR1-01454/Lane DR1-01423/Robins DR1-01480/Steinberg DR1-01490/Zaia Proj Start 3/2010 4/2010 3/2010 4/2010 5/2010 3/2010 6/2010 3/2010 6/2010 3/2010 5/2010 2/2010 2/2010 5/2010 Total Award 18,015,429 19,162,435 19,999,826 15,904,916 19,979,660 19,317,614 11,500,429 5,560,232 19,999,580 9,212,365 11,709,574 19,999,937 20,000,000 14,583,187 Current Status Continue Terminating 3/31/2012 Continue with revisions Continue Continue Continue Continue Continue Continue Continue Continue Continue Continue Continue

Milestones and expert advice, to better position the Disease Teams (DTs) for success
After ICOC approval and before funding, CIRM and DTs agreed on Go/No Go and progress milestones and criteria to inform decisions; budget to support proposed activities Complementing quarterly and annual reporting with CIRM, DTs met with CIRM and panels of clinical development advisors at 12 to 18 months of their progress; will continue to meet on annual basis Convening of clinical development advisors to provide external input into milestone process was approved in concept plan for DT solicitation, identified in the DT RFA, and subsequently communicated to all of the DTs
To prevent even the appearance of conflict of interest (COI), CIRM applied GWG COI rules. Clinical development advisors who had a financial, professional or personal COI with respect to one of the awards were disqualified from participating in the panels deliberations

Clinical development advisor panels, process for assessing DTs


Clinical development advisor panels are composed of experts in product development
preclinical and clinical research, process development and manufacturing, regulatory standards, stem cell/disease-specific biology, disease-specific clinical expertise, and commercial relevance

Provided advice and recommendations on project strategy, progress in meeting goals and go/no-go decision points, and approaches to meeting challenges in their product development plans Recommendations provided to Senior Vice President, Research and Development, and CIRM considered this advice in subsequent internal deliberations about the merit of continued support, and under the guidance of the President, made final decisions and followed-up with the DTs on these issues

Advisors
Deborah Armstrong, MD Johns Hopkins Kimmel Cancer Ctr Jonathan Auerbach, PhD GlobalStem Merry Lee Bain, MS Partner, NDA Partners, LLC Nessan Bermingham, PhD BioEquity Capital, LLC Lauren Black, PhD Charles River Laboratories Rajesh Chopra, PhD, BSc; MBBS;FRCP, FRCPath Celgene Corporation Alan F. Cruess, MD, FRCSC Dalhousie University David T. Curiel, MD, PhD Wash Univ School of Medicine Howard Fine, MD Natl Cancer Inst; Natl Inst Neurol Disorders and Stroke Joyce Frey-Vasconcells Pharmanet Derek Hei, PhD Univ of Wisconsin Anthony Hollander, PhD Univ of Bristol Daniel Hoth, MD Hoth Consulting, Inc Judith Karp, MD Johns Hopkins Kimmel Cancer Ctr Robert Langer, ScD Massachusetts Inst. of Technology Edith P. Mitchell, MD, FACP Kimmel Cancer Ctr at Jefferson

Advisors
Bruce Montgomery, MD CEO Cardeas Pharma Jon Odorico, MD Univ of Wisconsin Raymond Roos, MD Univ of Chicago Michel Sadelain, MD, PhD Memorial Sloan Kettering Cancer Center David Savello, PhD XenoPort, Inc Sean Savitz, MD Univ of Texas Ed Stanley, PhD Monash University Eckhard Thiel, MD Univ Berlin Jeffrey Touchman, PhD Arizona State University Darin Weber, PhD Mesoblast James Wilson, MD, PhD Univ of Pennsylvania Wolfram Zimmermann, MD University Medical Center Gottingen

11

Collaborative Funding Partners on 4 of the 14 DTs


UK
Catriona Crombie Medical Research Council Robert Buckle, PhD Medical Research Council

Canada
Karen Dewar, PhD Genome Canada Stephane Pion, MD, PhD CIHR Inst of Cancer Research

CIRM Science Officers involved in day to day management of DTs


Karen Berry, DVM, PhD Ingrid Caras, PhD Pat Olson, PhD Bettina Steffen, MD Sohel Talib, PhD Also providing Scientific support : Zach Scheiner, PhD Kevin Whittlesey, PhD

DR1-01421 Aboody Recurrent Glioblastoma (brain tumor)


Therapeutic candidate
Allogeneic hNSC line to target tumors, engineered to deliver enzyme, carboxylesterase, to convert chemotherapy drug CPT-11 to more potent metabolite SN-38, at the site of the invasive tumor

Key Accomplishments
Select final therapeutic candidate, develop under good manufacturing practices for the clinic Investigated optimal route, dose of NSC to achieve max % of tumor coverage will inject intracranial; also develop intravenous approach 1000 fold more toxic to brain tumor cells than chemotherapy alone Developed novel iron nanoparticle track stem cells, non-invasive imaging 1 publication, two under review, 11 presentations Founded company, TheraBiologics

Status
Complete preclinical POC efficacy fall/2012, and pre-IND discussion with FDA; no financial or timeline impact to project at this time

DR1-01426 Berger Recurrent Glioblastoma (brain tumor)


Therapeutic candidate
Allogeneic hNSC or MSC (from best of 3 sources) to target tumors, engineered to deliver gene product toxic to tumors (2 distinct gene modifications - TRAIL or cytosine deaminase and suicide gene)

Key Accomplishments
Stem cells did not reach brain tumors by intravenous route NSCs and MSCs delivered directly to tumors similar dispersion in tumor Unmodified NSCs had insufficient proliferative capacity for production as therapeutic stem cells In vitro studies showed superior anti-tumor activity when stem cells modified with cytosine deaminase in presence of pro-drug 5FC, compared to TRAIL; in vitro anti-tumor activity of CD + UPRT more rapidly produces toxic metabolites than CD alone 2 publications, developed 2 methods for detecting human cells and xenograft preclinical model for use in therapeutic testing

Status not able to select a cell candidate according to selection criteria


no go; terminating 3/31, and estimated savings $13M

DR1-01430 Carson Cancer stem cells in Leukemias


Therapeutic candidate
3 small molecules and 3 monoclonal antibodies against CSCs in acute (TALL, B-ALL, AML) and chronic (CLL, CML) leukemias; team is working with Canadian CFP (John Dick -Toronto) to dev therapies targeting specific survival and self-renewal pathways in leukemic stem cells

Key Accomplishments
Shown efficacy with anti-ROR1 monoclonal antibody blocking Wnt5 signaling in CLL stem cells, and with small molecule inhibiting JAK2 kinase in AML in rigorous serial transplant models in preclinical models using patient leukemia cells conducting extensive genomics studies to dev leukemia stem cell biomarker signature of response/resistance for potential utility in individualizing therapies and improving response

Status
CIRM recommended team focus their work on two candidates and two diseases (anti-ROR1 in CLLand JAK2 inhibitors in AML); budgets and timelines being assessed and realigned accordingly

DR1-01444 Humayun Age-related macular degeneration


Therapeutic candidate
hESC derived retinal pigment epithelium (RPE) on synthetic matrix; matrix mimics membrane at back of the eye - important for attachment, survival and differentiation of RPE (intact RPE imp for photoreceptors and vision). UK MRC CFP (Peter Coffey)

Key Accomplishments
Design ultra-thin matrix ,sufficient mechanical strength for culturing and surgical handling; permeability characteristics mimic membrane back of eye (Bruchs membrane), allows passage of proteins, prevents migration of stem cells 7 patent filings covers the matrix, placement of cells on matrix, methods to produce RPE, method to track cells after implantation and specialized instrumentation for surgical delivery of the implant POC after implantation in preclinical model, showed successful interdigitation of transplanted RPE/matrix with host photoreceptors, with morphological and functional rescue of photoreceptors, rescue of vision Spin-off company Regenerative Patch Technologies

Status Timelines and financials unchanged

DR1-01477 Slamon Cancer stem cells in solid cancers


Therapeutic candidate
Novel small molecule inhibitors of 2 kinases, targeting CSCs in glioma, colon carcinoma and ovarian cancer. Working in collaboration with Canadian CFP (Tak Mak Toronto)

Key Accomplishments
First lead candidate molecule has shown efficacy in preclinical models using human tumors and specifically targeting CSCs, and is moving into IND-enabling studies. Team is working on a second molecule targeting a different novel pathway in CSCs.

Status Timelines and financials unchanged

DR1-01485 Weissman Cancer stem cells in acute leukemia


Therapeutic candidate
Hu anti-CD47 monoclonal Ab, directed against cell surface target preferentially expressed on acute myeloid leukemia/ other CSCs. CD47 functions as a dont eat me signal by binding to a receptor on phagocytic macrophages and inhibiting their signaling. UK MRC CFP (Paresh Vyas - UK)

Key Accomplishments
Humanized and evaluated a number of candidate anti-CD47 antibodies aimed at CSC in acute myelogenous leukemia(AML) Filed patent covering sequence, uses for their lead therapeutic candidate POC efficacy, eradicated leukemia in tumor-bearing xenograft model of primary leukemic cells from patients with AML Conducting preclinical dose exploration and pilot safety studies MRC funded UK component collecting/analyzing samples from patients enrolled in AML trials to evaluate diagnostic and prognostic value of CD47 and other markers of interest re: drivers of leukemia relapse

Status Pursuing preclinical safety and efficacy; timelines and financials


unchanged

DR1-01471 Goldstein Amyotrophic Lateral Sclerosis (ALS)


Therapeutic candidate
hESC derived astrocyte precursor cells for subsequent development of product to inject into ALS patients spinal cord

Key Accomplishments
Generated cells from different sources of hESCs and are in the process of identifying cell lines with best characteristics of minimal toxicity and efficient production of final cell type POC demonstrating hESC-derived neural stem cells can protect motor neuron viability in preclinical model

Status
On track for selecting a single cell line in mid- 2012; timelines and financials unchanged

DR1-01461 Marban Advanced ischemic cardiomyopathy


Therapeutic candidate
Autologous cardiac-derived cardiospheres (CSps) or cardiosphere-derived cells (CDCs) for advanced ischemic cardiomyopathy (heart failure)

Key Accomplishments
Discovered that allogeneic cells appear to be as effective as autologous cells in improving cardiac function in preclinical model, and immune consequences of allogeneic appear to be neglible, and with CIRM support, extended that finding to another preclinical model Allogeneic cells can be obtained in large numbers from donor heart, greatly facilitating scale up and manufacture, enabling highly standardized off the shelf product; CIRM approved switch towards an allogeneic product Selected CSps as therapeutic candiaate and will deliver them by direct injection in to the heart using a special magnetically-guided catheter

Status
IND filing for allogeneic product anticipated in mid-2012; no change in timeline or financials.

DR1-01431 Chen HIV/AIDS


Therapeutic candidate
Autologous hematopoietic stem cells (CD34+) genetically modified with lentiviral vector encoding short hairpin RNA against CCR5 and HIV fusion inhibitor for patients with HIV infection, consider patients with AIDS/lymphoma

Key Accomplishments
Identified unique anti-HIV small RNAs to prevent HIV infection through preventing HIV entry into cells by targeting CCR5 as well as HIV replication through inhibiting different steps of HIV replication cycle. Ongoing studies demonstrate HIV inhibition both in vitro as well as in vivo preclinical model.

Status
No impact on timelines and financials at this time

DR1-01452 Kohn Sickle cell diseases


Therapeutic candidate
Autologous human bone marrow hematopoietic stem cells (HSC) genetically modified using lentiviral vector encoding anti-sickling human bglobin for patients with sickle cell disease

Key Accomplishments
Selected the single therapeutic candidate with disease modifying activity demonstrated in vitro and in vivo preclinical model, has had their pre-IND meeting with the FDA, and are planning preclinical toxicology studies Presented results of ongoing studies at the American Society of Hematology annual meeting in San Diego in December 2011

Status
If team is able to demonstrate feasibility of obtaining sufficient numbers of CD34+ cells by dual bone marrow harvest then there will be no impact on timelines and financials. The team has already done preclinical POC studies with bone marrow derived CD34+ cells. Other activities will continue as planned.

DR1-01454 Lane Dystrophic epidermolysis bullosa (DEB)


Therapeutic candidate
Autologous iPSC-derived, gene-corrected keratinocytes; DEB is caused by mutations in type VII collagen, and team is targeting 2 forms of DEB, the recessive form and dominant form.

Key Accomplishments
Team has successfully generated iPSC lines from several patients with the recessive form of DEB

Status

No immediate impact on timelines or financials

DR1-01423 Robins Type 1 Diabetes


Therapeutic candidate
Allogeneic hu ESC-derived pancreatic progenitors, mature in vivo to beta cells secreting insulin in response to glucose, delivered in retrievable immunoisolation device implanted subcutaneously

Key Accomplishments
Prototypes tested in preclinical model; in preclinical POC studies, this celldevice combination cured drug-induced diabetes ViaCyte established cell mfr, device mfr proceeding at a scale, level of quality to enable preclinical, clinical testing of the combo product; specifically:
made, qualified cGMP master cell bank that met defined criteria; made cGMP working cell bank; finalized progenitor cell manufacturing process, determined final device configuration for clin testing; device manufacturing facility was designed and built; established preclinical models in collaboration with world-renowned immunologists and testing ability of device to protect cells from allo- and auto-immunity; preparing for pivotal IND enabling studies; developing clinical plan for FIH testing

Two invention disclosures Status received suppl funding from JDRF; in discussion with potential partner;
CIRM needs to consider providing bridging funds to support activities needed to support IND filing within the 4 year period of award

DR1-01480 Steinberg Stroke


Therapeutic candidate
Allogeneic hESC derived NSC line transplanted alone or in combination with matrix material into infarcted area of brain, with concomitant immunosuppression

Key Accomplishments
Utilizing research bank of candidate cells, functional recovery demonstrated in 3 preclinical models of stroke in 3 independent labs Continues to establish cell mfr and testing processes to enable preclinical and clinical testing; produced 2 qualification lots that can be elected as master cell bank and working cell bank, based on extensive characterization, func and safety testing Conducted pre-pre-IND meeting with FDA Pursuing less complex mfr process (without hydrogel) in order to bring candidate to IND and FIH testing expeditiously (exploring hydrogel as part of CIRM tools and technology award to optimize technology)

Status
no impact to original timelines and financials at this time

DR1-01490 Zaia HIV/AIDS


Therapeutic candidate
Adenovirus modified hematopoietic stem cells (CD34+) expressing zinc finger nuclease targeting CCR5; targeted to AIDS leukemia patients infected with CCR5 tropic HIV and undergoing autologous HSC transplantation; ultimately, as method is improved using non-ablative HSC transplantation, this indication would expand to include AIDS patients with retroviral drug failure

Key Accomplishments
Optimizing methods for adenoviral vector mediated transduction of HSCs and CCR5 disruption; optimized methods for expression of ZFN in HSCs to target CCR5 Dev preclinical model to test stem cell therapy for HIV
Used ZFN to alter CCR5 gene in HSCs, and demonstrated these cells can be transplanted into preclinical model. CCR5 gene encodes cell surf receptor used by HIV to enter cells. When altered HSCs given to preclinical model that lacked effective immune system, cells colonized in bone marrow and created new blood system with mutated CCR5. When exposed to HIV, recipients beat it back.

Status progressing towards IND enabling studies for scale up cell processing;
no imp-act on timelines or budget; clinical dev advisor meeting planned for Q4 2012

Disease Team portfolio


Disease Area, # Projects (n = 14) Disease Area, CIRM Disease Area, CIRM & CFP Investment ($225.6 MM) Investment ($270.4 MM)

Back up

Eye Disease

Progression of age-related macular degeneration, from www.clevelandsightcenter,org

Award
DR-1444 USC, UCSB Univ. College London (UK) TR-1219 Scripps Res. Institute TR-1272 UCLA

Goal
IND

Disease
Age-related Macular Degeneration (Dry form) Age-related Macular Degeneration (Dry form) Age-related Macular Degeneration (Dry form)

Approach
Allogeneic functionally polarized hESC-derived RPE monolayers on synthetic substrate implanted subretinally Autologous iPSC-derived RPE (generated without integrating vectors) Autologous adult SC (CMZ) or iPSCderived RPE +/- ex vivo engineering to express negative regulators of complement cascade

DC

DC

Eye Disease
Limbal epithelial stem cells reside in the basal layer of the epithelium, from GA Secker NCBI Bookshelf
Limbal stem cells

Progression of Retinitis Pigmentosa, from www.medgadget.com

Award
TR-1794 UCI TR-1768 UCLA

Goal
DC POC

Disease
Retinitis Pigmentosa Limbal stem cell deficiency

Approach
Allogenic retinal progenitor cells Ex vivo expansion of corneal epithelial stem / progenitor cells also known limbal stem cells (LSC)

Cancer

A child is stricken with leukemia, from infomationhealth.blogspot.com

Award
DR-1430 UC San Diego; Uni Health Network (Canada)

Goal
IND

Disease
Hematologic malignancy (AML, CML, ALL, CLL)

Approach
Existing candidate molecules (3 small molecule, 3 MAb) targeting leukemic stem cells (LSC) by blocking survival and self-renewal pathways that function preferentially Monoclonal antibody against CD47 Dont eat me antigen that is expressed on LSC and inhibits their phagocytosis by macrophages

DR-1485 Stanford University; Weatherall Institute, Oxford Univ. (UK)

IND

Hematologic malignancy (AML)

Cancer

A small-molecule drug selectively kills tumor cells by activating p53 protein, from www.cancer.med.umich.edu

Award
TR-1789 UCSD TR-1816 Children;s Hospital of L.A.; Univ. of Jena (Germany)

Goal
DC DC

Disease
Hematologic malignancy (CML) Hematologic malignancy (AML, ALL)

Approach
Small molecule pan BCL-2 inhibitor targeting LSC Small molecule inhibitor of BCL6 targeting LSC

Cancer

A confocal image showing -crystallin (green), a small heat shock protein in the perinuclear Golgi in dividing human U373 glioblastoma cells, from S.P. Bhatt UCLA

A dividing cancer cell, from www.integraldeeplistening.com

Award
DR-1477 UCLA; Stanford Univ., USC, Univ. Health Network (Canada)

Goal
IND

Disease
Solid tumors (Colon cancer, ovarian cancer, glioblastoma)

Approach
Small molecules specific for either of two drug targets in cancer stem cells (CSC)

Cancer

A glioblastoma multiforme cell, from www.radiologia.blog.hu

An image of glioblastoma brain tumor, from WikiCommons

Award
DR-1421 City of Hope Med. Center DR-1426 UCSF, Ludwig Instit. for Cancer Res., SanfordBurnham Instit. for Medical Res. TR-1791 UCLA

Goal
IND

Disease
Recurrent Glioblastoma

Approach
Allogeneic established hNSC line to target tumor, engineered ex vivo to deliver carboxylesterase to locally convert CPT-11 to SN-38 Allogeneic hNSC (hMSC) to target tumor, engineered ex vivo to deliver a tumorcidal gene product (TRAIL or cytosine deaminase, and a suicide gene) Tumor homing by MSC genetically engineered to produce replication competent retrovirus encoding a suicide gene

IND

Recurrent Glioblastoma

DC

Solid tumor (Glioblastoma)

Cardiovascular

Award
DR1-01461 Cedars-Sinai Med. Center

Goal
IND

Disease
Advanced Ischemic Cardiomyopathy (heart failure) Multiple (Stroke, Heart disease, Skin ulcers, Bone fractures)

Approach
Autologous cardiac derived, cardiospheres, expanded and delivered by direct catheter injection into heart muscle Recombinant Wnt in a sustained release formulation to stimulate endogenous stem cells to repair tissues

TR1-01249 Stanford University

DC

Neurological Disorders Neurodegenerative Disease


Award
DR1-01471 UCSD, Salk Institute

Goal
IND

Disease
ALS

Approach
Allogeneic hESC-derived astrocyte precursors delivered into spinal cord (delivery device) The best of either hNSC derived from tissue, ESC, or iPSC; or hVM (ventral mesencephalon) precursors derived from ESC, NSC, or tissue

TR1-01267 Sanford-Burnham Institute; Howard Florey Institute (State of Victoria, AS) TR2-01856 Buck Institute; City of Hope National Medical Center

DC

Parkinsons Disease

DC

Parkinsons Disease

Allogeneic hPSC-derived dopaminergic neurons

TR2-01778 Salk Institute; University of Erlangen (BMBF, Germany)

POC

Parkinsons Disease

Small molecule modulator of neuroinflammation identified by screening on astrocytes/microglia from patient-derived iPSCs

Neurological Disorders Neurodegenerative Disease

Award
TR1-01245 UCI, Monash University (State of Victoria, AS)

Goal

Disease

Approach
Allogeneic hESC-derived NSC or hESC-derived NSC genetically modified with a beta-amyloid degrading enzyme or a transcription factor that promotes neuronal differentiation for transplantation Allogeneic MSC engineered ex vivo to express siRNA targeting mutant huntingtin mRNA. Injected intracranially Allogeneic hESC-derived neural stem or progenitor cells for transplantation

DC

Alzheimers Disease

TR1-01257 UCD TR2-01841 UCI

DC

Huntingtons Disease

DC

Huntingtons Disease

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Neurological Disorders

Award
TR2-01785 UCLA DR1-01480 Stanford University, UCLA TR2-01767 UCI

Goal
POC

Injury
Spinal Cord Injury

Approach
Allogeneic hESC-derived motor and autonomic precursor neurons Allogeneic hESC-derived NSC line transplanted alone or in combination with matrix

IND

Stroke

POC

Traumatic Brain Injury

Allogeneic hESC-derived NSC

Neurological Disorders

Award
TR2-01844 iPierian, Inc. TR2-01832 City of Hope National Medical Center, University of Bonn (BMBF, Germany)

Goal
DC

Disease
Spinal Motor Atrophy

Approach
Small molecule that increases SMN1 gene product in patient iPSCderived motor neurons Autologous iPSC-derived neural or oligodendrocyte progenitors, genetically modified to correct mutant (aspartoacylase) ASPA gene Neurons from ASD (and control) iPSC for phenotype screening, assay development and validation, drug screening and biomarker identification Allogeneic hESC-derived progenitors from GABAergic inhibitory neurons analogous to those in medial ganglionic eminence

POC

Canavan Disease

TR2-01814 UCSD

POC

Autism Spectrum Disorders

TR2-01749 UCSD

POC

Refractory Epilepsy

Diabetes & Complications

Award
DR1-01423 ViaCyte Inc., UCSF

Goal

Disease

Approach
Allogeneic hESC-derived pancreatic cell progenitors in a device implanted subcutaneously that mature in vivo to beta cells that secrete insulin in response to glucose. Transient immunosuppression.

IND

Diabetes

TR2-01787 UCD, Technical University of Munich (BMBF, Germany)

DC

Chronic diabetic foot ulcers

Allogeneic hMSCs on a dermal regeneration scaffold

41

HIV/AIDS

Award
DR1-1431 UCLA, Calimmune, Inc. DR1-1490 City of Hope National Medical Center, USC, Sangamo Biosciences TR2-1771 City of Hope National Medical Center

Goal

Disease
AIDS Lymphoma

Approach
Autologous HSC transduced ex vivo with a lentiviral vector engineered to express a shRNA against CCR5 & fusion inhibitor. IV administration after myeloablation. Autologous HSC transduced ex vivo with non-integrating vector engineered to express zinc finger nuclease against CCR5. IV administration after myeloablation Autologous HSC genetically modified ex vivo with multiple anti-HIV resistance genes and a drug resistance gene

IND

IND

AIDS Lymphoma

DC

AIDS Lymphoma

Blood, Genetic Disorders

Award
DR1-01452 UCLA, Childrens Hospital, LA TR1-01273 Salk Institute
DR1-01454 Stanford University

Goal

Disease
Sickle Cell Anemia

Approach
Autologous HSC, genetically corrected ex vivo by lentiviral vector mediated addition of a hemoglobin gene that blocks sickling. IV administration after myeloablation Autologous iPSC-derived HSC genetically corrected by homologous recombination
Epidermal sheets from expanded autologous genetically corrected (to express wild type COL7A1) iPSC-derived keratinocytes Autologous skeletal muscle precursor cells derived from human iPSCs genetically modified to correct the dystrophin gene

IND

DC

Fanconi Anemia, X-SCID


Dystrophic Epidermolysis Bullosa Duchenne muscular dystrophy

IND

TR2-01756 Stanford University

POC

CIRM Industry Engagement Initiatives

Strategic Partner Funding Program

Offers a streamlined procedure to provide funding to programs demonstrating commercial validation

Regenerative Medicine CIRM researchers to be featured Pharma & Investor Premier event for regenerative medicine industry Partnering Conference Patent funding and Technology Transfer Support
$5 M allocated for technology transfer support; program being developed

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