SILVER SPRING, Md., Feb. 15, 2019 — “We’re at an inflection point when it comes to cell and gene therapies. These treatments have the potential to address hundreds of vexing human diseases and conditions. When we first issued our comprehensive regenerative medicine policy framework in November 2017, our goal was to achieve a balanced and risk-based approach to support product development in cell-based therapies, while clarifying the FDA’s authorities and enforcement priorities to make sure we were protecting patients,” said FDA Commissioner Scott Gottlieb, M.D. [Read more…]
On January 4, 2019, Sana Biotechnology became the latest regenerative medicine company to enter the marketplace, founded by former Juno Therapeutics executives Hans Bishop and Steve Harr, among other all-star executives. With operations in Seattle, WA, Cambridge, MA and San Francisco, CA, the company will be focused on creating engineered cells as medicines for patients.
Based on preliminary company statements, Sana seems to be interested in reproducing the business model and the financial success that characterized Juno Therapeutics – specifically, its ability to attract top talent from across the marketplace, accumulate investor funds, and exit for a major windfall. [Read more…]
A new behemoth is entering the cell therapy CDMO market, FUJIFILM Cellular Dynamics (FUJIFILM CDI). CDMOs are companies that provide manufacturing services to cell and gene therapy companies on a contract basis. In some cases, they may also support product development, clinical trial support, or commercial supply.
On January 3, 2019, FUJIFILM Cellular Dynamics, Inc. (FCDI), a US subsidiary of FUJIFILM Corporation, announced it will be spending $21M to open a new cGMP-compliant production facility in Madison, Wisconsin. According to FCDI, the facility will support FCDI’s internal cell therapeutics pipeline, as well as serve as a Contract Development and Manufacturing Organization (CDMO) for iPS cell products. [Read more…]
Kymriah® (tisagenlecleucel), CAR-T therapy from Novartis, receives TGA approval for treating two aggressive blood cancers
- Kymriah® is an immunocellular therapy that is a one-time treatment manufactured individually for each patient using the patient’s own T cells, genetically reengineered and programmed to recognise and destroy cancer cells.
- Kymriah® is approved for the treatment of relapsed or refractory patients with paediatric B-cell acute lymphoblastic leukaemia (ALL) and adult diffuse large B-cell lymphoma (DLBCL)
- Kymriah® could be a treatment option for about 50 paediatric and young adult ALL patients and 420 adult DLBCL patients who relapse or do not respond to initial therapy
- Kymriah® is the only chimeric antigen receptor T cell (CAR-T) therapy to receive TGA approval in Australia
It is official. Australian stem cell company Cynata Therapeutics has completed the world’s first Phase I trial involving an induced pluripotent stem cell (iPSC) derived therapeutic product. The landmark study involved the company’s lead product, CYP-001, an iPSC-derived mesenchymal stem cell (MSC) product that can be manufactured at therapeutic grade and at mass scale using Cynata’s Cymerus™ platform technology.
Next, the company will be initiating Phase II trials in graft-versus-host disease (GvHD), critical limb ischemia (CLI) and osteoarthritis, showcasing the power of Cynata’s platform technology to be leveraged across multiple indications. Osteoarthritis will be Cynata’s largest clinical trial to date and also one of the largest ever for an MSC product. [Read more…]