Industry Update: Business Development
Latest business developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non-academic institutions July 01 – August 31, 2017.
Latest business developments compiled from July 01 – August 31, 2017 scheduled to be published in Volume 12 Issue 8 of Regenerative Medicine.
Collaborations, partnerships & alliances
Collaboration agreement: CRISPR and MGHCC
CRISPR Therapeutics (Switzerland; www.crisprtx.com) and Massachusetts General Hospital Cancer Center (MGHCC; MA, USA; www.massgeneral.org/cancer) have entered into a two-year research collaboration and license option agreement to develop novel T cell therapies for cancer. As part of the collaboration, CRISPR/Cas9 gene editing will be utilized to improve upon current T cell therapies in development, ultimately addressing unmet needs in both hematologic and solid tumors.
Collaboration agreement: CRISPR and Neon
CRISPR Therapeutics (Switzerland; www.crisprtx.com) and Neon Therapeutics (MA, USA; www.neontherapeutics.com), an immuno-oncology company developing neoantigen-based therapeutic vaccines and T cell therapies to treat cancer, have announced a research collaboration to explore the combination of each company’s proprietary technologies to develop novel T cell therapies.
Collaboration agreement: Evotec and Frauhofer IME-SP
Evotec (Germany; www.evotec.com) has announced a strategic collaboration on induced pluripotent stem cell (iPSC) technology with the Fraunhofer Institute for Molecular Biology and Applied Ecology IME Screening Port (Germany; www.ime.fraunhofer.de/en/Research_Divisions/business_fields_TM/screeningport.html), as an important element of the existing Evotec/Fraunhofer cooperation agreement signed in 2014. Through this partnership, Fraunhofer IME-SP will provide access to its technology portfolio including advanced genetic characterization techniques and innovative quality control procedures to support Evotec's patient-derived iPSC platform. The contract will run for an initial period of two years. No financial details were disclosed.
Partnership agreement: Pairnomix and StemoniX
Pairnomix (MN, USA; www.pairnomix.com) and StemoniX (MN, USA; www.stemonix.com) have announced a new strategic partnership combining Pairnomix's expertise in epilepsy modeling with StemoniX's proprietary iPSC-based microBrain technology to create a new in vitro seizure-in-a-dish model system. The microBrain model is composed of human iPSC-derived neural cells containing populations of glutamatergic and GABAergic neurons and astrocytes. A key feature of the new model system is the ability to induce seizure-like electrical activity at the network level. This sophisticated design captures functional neuronal circuitry and allows for the detection of subtle pharmacological effects against seizure-like activity that might otherwise be difficult to measure in a more complex cellular environment. This system creates a platform that is amendable to high throughput drug screening to discover new anti-seizure medications.
Launching new projects, products & services
Celularity (NJ, USA; www.celularity.com), a newly formed biotechnology company focused on human placenta-derived cellular therapeutics and biomaterials, has completed their Series A financing with contributions from several biopharma companies and entrepreneurial investors.
The Kyoto University Institute for Frontier Life and Medical Sciences (IFLMS; Japan; www.infront.kyoto-u.ac.jp/?lang=en) has announced that its plan to establish human embryonic stem (hESC) lines, and produce hESC stocks has been approved by Japan's science ministry MEXT and health ministry. The initiative was confirmed to be in compliance with both ministries' guidelines, making IFLMS the first Japanese institute to receive government approval for producing hESC for clinical use. IFLMS' plan is to make human ES cells from unused fertilized eggs, obtained with consent from patients receiving infertility treatment at a local hospital. The production site will be the Institute's newly established Cell Processing Facility (CPF), which received certification in January 2017 as a "clinical-use cell-culture facility" to manufacture specified cell products as per the Act to Ensure the Safety of Regenerative Medicine. IFLMS' hESC stocks will be made available to medical institutions, including the National Center for Child Health and Development, for use in clinical research.
Pluristem Therapeutics (Israel; www.pluristem.com) has announced that a pilot study of the company’s PLX-R18 cell therapy will be initiated by the US Department of Defense’s (DOD) Armed Forces Radiobiology Research Institute (AFRRI), part of the Uniformed Services University of Health Sciences (USU). The study will examine the effectiveness of PLX-R18 as a treatment for Acute Radiation Syndrome (ARS) prior to, and within the first 24 hours of exposure to radiation. ARS results from exposure to high levels of radiation, such as in the case of a nuclear accident or attack, and can lead to severe health consequences including death. Pluristem recently reported positive data from studies of PLX-R18 cells as a treatment for ARS conducted by the National Institute of Allergy and Infectious Diseases (NIAID) at the NIH, US Department of Health and Human Services (DHHS). Data demonstrated improvement in survival rates and enhancement of blood lineages recovery.
A key difference in the NIAID study and the upcoming DOD study is the timeframe of exposure that is being examined: in the NIAID study, PLX-R18 was administered to subjects 24 hours post exposure, while the new DOD study will be designed to support the needs of the US Armed Forces and examine subjects receiving treatment prior to, or within the first 24 hours of radiation exposure. The DOD studies will be conducted in parallel with the NIH/DHHS studies, allowing broader understanding of the potential therapeutic effects of PLX-R18 as a novel medical countermeasure for ARS. The study will be conducted in accordance with the FDA Animal Rule pathway, the regulatory pathway followed when human efficacy trials are not feasible, in this case due to the ethics of exposing humans to nuclear radiation. Product approval via this pathway is granted following large animal efficacy studies and human safety data.
StimLabs (GA, USA; www.stimlabs.com) is releasing two new products: Revita®, the first full thickness placental membrane allograft in a shelf-stable format, and Ascent™, an amniotic fluid allograft that captures key amniotic fluid components in a shelf-stable powder. Processed using the patent-pending Clearify™ method, Revita preserves amnion, chorion and the intermediate layer of the membrane, making it the ideal barrier membrane in surgical applications and the optimal wound covering for chronic wounds. Processed using the patent-pending Selectify™ method, Ascent captures a consistent set of selected non-viable cells, proteins, and growth factors to protect, cushion, lubricate and reduce inflammation in fluid environments, such as joints and tendons.