Latest developments compiled from 1—30 September 2016
AiVita Biomedical (CA, USA; www.aivitabiomedical.com) has been awarded a Research Project Grant (R01) from the National Eye Institute of the NIH for the development of stem cell-derived 3D-transplantable retinas to treat vision loss. The project aims to address the loss of retinal pigment epithelium and photoreceptors typically observed in patients with advanced degenerative eye disease, such as macular degeneration and retinitis pigmentosa. AiVita will manufacture the 3D-retinal organoids and render the process both clinically and commercially compliant, and collaborators at the University of California at Irvine’s Sue & Bill Gross Stem Cell Research Center will test the product for safety and efficacy in relevant models of retinal degeneration.
Allele Biotechnology and Pharmaceuticals (CA, USA; www.allelebiotech.com) has been awarded a Phase I Small Business Innovation Research grant to develop a novel manufacturing system to produce stem cell-derived human tissue and cells for clinical therapy from the NIH’s National Heart, Lung and Blood Institute. This grant will allow Allele to further move its patented method of reprogramming somatic cells into iPSCs into commercially viable clinical cell therapies.
Capricor Therapeutics (CA, USA; http://capricor.com) has been awarded a grant of approximately US$2.4 million from the US Department of Defense toward establishing a scalable, commercially ready process to manufacture CAP2003 (Cardiosphere-Derived Cell Exosomes). Capricor has identified ocular GvHD as the first clinical development opportunity for CAP2003, and expects to submit an IND for this indication in the first half of 2017. CAP2003 represents exosomes isolated from the company’s proprietary cardiosphere-derived cells  which are currently in clinical evaluation by Capricor for the treatment of several cardiac conditions, including that associated with Duchenne muscular dystrophy (www.clinicaltrials.gov, ID: NCT02485938).
Cytori Therapeutics (CA, USA; www.cytori.com) announced that the Biomedical Advanced Research and Development Authority (BARDA; www.phe.gov/about/BARDA/Pages/default.aspx), a division of the US Department of Health and Human Services, increased the contract option originally signed in August 2014 to fund continued investigation and development of Cytori Cell Therapy for use in thermal burn injuries. Cytori Cell Therapy is based on the use of adipose derived regenerative cells isolated from a patient’s own adipose tissue. The amended contract option is valued at US$16.6 million, an increase of approximately US$2.5 million from its previous value of US$14.1 million. Upon Investigational Device Exemption approval by the FDA, if received, Cytori will request that BARDA provide additional funding to cover costs associated with the completion of a pilot clinical trial. This trial will employ in vivo administration of Cytori Cell Therapy. The supplemental funds from this amended contract will be used to support the remaining activities necessary to seek approval of the Investigational Device Exemption and support clinical readiness. The original contract includes additional options, exercisable at BARDA’s discretion, valued at up to US$68 million to fund pilot clinical trials and additional work in thermal burn complicated by radiation exposure.