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Marlborough, MASSACHUSETTS, United States

Patent
Ocata Therapeutics | Date: 2015-02-23

The present invention generally relates to novel preparations of mesenchymal stromal cells (MSCs) derived from hemangioblasts, methods for obtaining such MSCs, and methods of treating a pathology using such MSCs. The methods of the present invention produce substantial numbers of MSCs having a potency-retaining youthful phenotype, which are useful in the treatment of pathologies.


Grant
Agency: Department of Health and Human Services | Branch: National Institutes of Health | Program: SBIR | Phase: Phase I | Award Amount: 216.07K | Year: 2015

DESCRIPTION provided by applicant Advanced Cell Technology Inc ACT is a clinical stage biotechnology company focused on the development and commercialization of regenerative medicine and cell therapy technology The companyandapos s most advanced products are in pioneering clinical trials for the treatment of a variety of eye related debilitating diseases he overall objective of this SBIR Phase I application is to extend previous studies and provide proof of concept that human pluripotent stem cell PSC derived photoreceptor progenitors PSC PhRPs are able to prevent progression of retinitis pigmentosa RP by transplantation of PhRPs derived from human embryonic stem cells hESCs and induced pluripotent stem cells iPSCs using a rat model of RP Investigations will determine whether these grafted PSC PhRPs will promote survival of host photoreceptors and differentiate into mature photoreceptors maintain retinal connections preserve vision and rationalize further development of PhRP cell based approaches for RP therapy Retinal degenerative diseases such as RP age related macular degeneration and rod con dystrophies are characterized by loss of photoreceptor cells resulting in permanent loss of vision and often blindness currently no curative therapy exists These conditions exert extensive societal burdens on quality of life productivity and health care costs and thus an urgent need exists to develop strategies for retinal survival repair and replacement to combat RP and other degenerative diseases of the retina Recent animal studies have shown that photoreceptor cell replacement is a promising therapeutic strategy for retinal degeneration although robust cell integration and recovery of visual function has yet to be achieved Impediments to progress in the field include a limited supply of donor cells and questionable cell purity In order to overcome these barriers ACT has developed a unique method for robust differentiation of human PSCs into pure renewable populations of retinal photoreceptor cells successfully using multiple hESC and iPSC lines as starting material In initial studies ACT has demonstrated that these PSC PhRPs are able to further differentiate in vitro and form mature photoreceptors expressing rhodopsin and opsin and when transplanted into the vitreous of RCS rats differentiate into mature rod photoreceptors expressing rhodopsin and recoverin Additional preliminary studies in end stage retinal degenerated mice demonstrated that PSC PhRPs migrated and integrated into the outer nuclear layer and were therapeutically active in improving optokinetic responses In this proposal these promising studies will be extended to determine whether ACTandapos s human PSC PhRPs when grafted into retinas of RP rats are able to protect and rescue photoreceptors at an early stage of disease to limit progression of degeneration and whether they are also able to replace degenerated photoreceptors in late stage disease to restore visual function If successful IND enabling studies will initiate in a Phase II program toward eventual clinical trial PUBLIC HEALTH RELEVANCE Degenerative diseases of the eye such as retinitis pigmentosa RP and age related macular degeneration which lead to vision loss and often blindness are devastating conditions with currently no known cure Excitement in the field has recently been generated to suggest that replacing cells in the eye that normally degenerate in these diseases with healthy cells of the same type may offer a new therapeutic approach Advanced Cell Technology has developed a unique process to produce large quantities of pure human replacement cells and in this proposal will determine whether they are effective in treating RP in an animal model at both early and late stages of disease If so studies will continue toward advancing its cell based therapy approach into human clinical trials


Grant
Agency: Department of Health and Human Services | Branch: National Institutes of Health | Program: SBIR | Phase: Phase I | Award Amount: 225.64K | Year: 2015

DESCRIPTION provided by applicant Advanced Cell Technology Inc ACT is a biotechnology company focused on the development and commercialization of regenerative medicine and cell therapy technology It is currently the only company with ongoing clinical trials in the U S and Europe for testing the safety and efficacy of a human embryonic stem cell hESC derived product ACTandapos s clinical focus involves a variety of eye related indications as well as non ocular disorders involving autoimmunity inflammation and wound healing The overall objective of this SBIR Phase I application is to extend ACTandapos s preclinical stem cell technology platform for the treatment of systemic lupus erythematosus SLE and lupus nephritis LN using transplanted hESC derived mesenchymal stromal cells MSCs SLE is a devastating systemic autoimmune disease that presents significant disease management challenges with no currently known cure While patients with mild to moderate SLE are to some extent clinically manageable with current protocols there remains a subset of SLE patients that resists all forms of current interventions and suffers severe disease A critical need therefore exists for strategies that deal with this patient population particularly for LN where remission is slow and response to current treatments limited Although MSCs can be isolated from several sources issues such as the scarcity of naturally occurring MSCs in tissues loss of immunomodulatory properties upon in vitro expansion and a lack of reliable quality control have led to inconsistencies in their reportd in vivo effectiveness ACT has overcome many of these obstacles through a novel and efficient method that uses hESCs as a source for MSCs to derive unlimited replenishable amounts of early passage MSCs of consistent quality Importantly ACT has demonstrated that its hESC MSCs exert therapeutic effects in several autoimmune disease models including prolonged survival of lupus prone NZB W F mice Due to the genetic complexity of human SLE it is imperative to examine potential new therapeutics in LN disease models harboring different underlying genetic susceptibilities Studies proposed here through two Specific Aims will employ an additional classic mouse model MRL lpr to extend previous findings using NZB W F mice and rationalize further development of a hESC MSC based approach for SLE LN Aim will determine minimal effective and maximum tolerated doses of ACTandapos s hESC MSCs for reducing LN disease severity in lupus prone MRL lpr mice and compare the effects to those of human umbilical cord hUC derived MSCs which are showing promise in human clinical trials for lupus Aim will define the molecular and cellular targets of hESC MSC therapeutic activity in tissues sera from mice injected in Aim and compare these effects to those of hUC MSCs PUBLIC HEALTH RELEVANCE Advanced Cell Technology Inc ACT is a Massachusetts clinical stage biotechnology company focused on the development and commercialization of regenerative medicine and cellular therapeutics For the proposed study ACT will perform preclinical experiments in naturally lupus prone mice that will test the ability of its human embryonic stem cell derived mesenchymal stromal cells for reducing the severity of lupus nephritis LN an autoimmune kidney disease The ultimate goal is to develop a cell based therapy for systemic lupus erythematosus SLE patients particularly those with severe disease and or who may suffer from treatment resistant LN a disorder that could lead to kidney failure or eventually death


Methods for production of platelets from pluripotent stem cells, such as human embryonic stem cells (hESCs) and induced pluripotent stem cells (iPSCs) are provided. These methods may be performed without forming embryoid bodies or clusters of pluripotent stem cells, and may be performed without the use of stromal inducer cells. Additionally, the yield and/or purity can be greater than has been reported for prior methods of producing platelets from pluripotent stem cells. Also provided are compositions and pharmaceutical preparations comprising platelets, preferably produced from pluripotent stem cells.


Methods are provided for the production of photoreceptor cells and photoreceptor progenitor cells from pluripotent stem cells. Additionally provided are compositions of photoreceptor cells and photoreceptor cells, as well as methods for the therapeutic use thereof. Exemplary methods may produce substantially pure cultures of photoreceptor cells and/or photoreceptor cells.

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