Founded in 1989, Vericel (formerly Aastrom Biosciences) is dedicated to the development of patient-specific expanded cellular therapies for use in the treatment of patients with severe diseases and conditions.

The company currently markets two cell therapy products in the United States: Carticel® (autologous cultured chondrocytes), an autologous chondrocyte implant for the treatment of cartilage defects in the knee, and Epicel® (cultured epidermal autografts), a permanent skin replacement for the treatment of patients with deep dermal or full thickness burns. Vericel is also developing MACI™, a third-generation autologous chondrocyte implant product, as well as ixmyelocel-T, a patient specific multicellular therapy for the treatment of advanced heart failure due to ischemic dilated cardiomyopathy (DCM).

Leaders in cell therapy manufacturing

Vericel’s historic focus on cell therapy and regenerative medicine took a major step forward in May 2014 when the company acquired the global marketing rights to three landmark autologous cell therapies from the Genzyme division of Sanofi:

  • CARTICEL is the first and only FDA-approved biologic product used to repair articular cartilage injuries in the knee. It is manufactured using a patient’s own cartilage cells and is implanted in a surgical procedure known as autologous chondrocyte implantation (ACI). Once implanted, the cells have been shown to form new hyaline-like cartilage, with properties similar to normal cartilage, and may reduce pain and improve knee function.
  • EPICEL is FDA approved as a humanitarian use device (HUD) and is indicated for use in patients who have deep dermal or full thickness burns comprising a total body surface of greater than or equal to 30%. EPICEL, which is essentially thin sheets of epidermis, is manufactured from a small sample of patients’ skin.
  • MACI is a third-generation ACI product for the treatment of articular cartilage injuries in the knee. It has been approved but is not currently marketed in Europe. In June, Vericel announced that following discussions with the U.S. Food and Drug Administration (FDA) the company plans to submit a Biologics License Application (BLA) to the FDA by the end of 2015 for MACI for the treatment of focal chondral cartilage defects in the knee. MACI has potential advantages of a shorter, less-invasive surgical procedure and a faster recovery period than CARTICEL.

For more than 20 years, the research and production teams responsible for CARTICEL and MACI have been focused on superior performance in autologous cell therapy manufacturing establishing industry standards.

  1. A) CARTICEL PROCESSING: Vericel’s cell processing manufacturing facility uses specific enzymes to free cartilage cells (chondrocytes) from the cartilage matrix. The chondrocytes then begin the initial culturing (multiplying) phase, lasting approximately 14 days. At this point, cultured chondrocytes are placed into cryopreservation until implantation surgery is scheduled.
  2. B) FINAL PHASE: Prior to the implantation, the chondrocytes are removed from cryopreservation to begin the final culturing phase. The chondrocytes are cultured until there are approximately 12 million cells per vial; up to 4 vials per patient.
  3. C) QUALITY AND SAFETY. Every step of the manufacturing process is vigorously monitored to ensure the highest quality and safety possible.

In the manufacturing process, the focus on quality and safety affects every stage of production, beginning with careful lot segregation and closed culture conditions to prevent cross-contamination. Visual inspection for sterility is based on the use of state-of-the-art technology. Cell culturing is supported by continuous monitoring of cell growth morphology and sterility. Prior to release, products undergo viability, identity, sterility, endotoxin, and morphology testing.

Multicellular therapy to improve patient health

IXMYELOCEL-T PROCESSING. To manufacture ixmyelocel-T, a small amount of bone marrow is collected from the patient. The therapy is then developed using a proprietary cell-processing system that is highly automated, fully closed and rigorously controlled. The production process is both scalable and reproducible to continually position Vericel to be able to meet demand for on-time therapy delivery through all stages of clinical research.

The production process for ixmyelocel-T expands the populations of naturally occurring cells in bone marrow that are believed to play a role in long-term tissue repair.

Targeting therapy with less risk

For patients suffering from complex, multi-factorial, severe and chronic diseases such as DCM or CLI, ixmyelocel-T is manufactured based on several unique features that are critical for treatment success:

  • Patient-specific (autologous): The process uses each patient’s own cells to produce ixmyelocel-T. This helps to ensure that the cell therapy will not be rejected by the patient’s immune system.
  • Expanded cell populations: The production process expands the number of CD90+ mesenchymal cells, CD14+ monocytes and alternatively activated macrophages, which are believed to play key roles in tissue remodeling, immuno-modulation and the promotion of angiogenesis.
  • Minimally invasive procedures: The aspiration procedure for removing a small amount of bone marrow and the administration of ixmyelocel-T can be performed in an outpatient setting.
  • Focus on safety: Therapies derived from bone marrow have been used safely and efficaciously in medicine for over three decades. The production of ixmyelocel-T leverages this body of clinical research and medical experience. Based on clinical experience in the treatment in hundreds of patients thus far, there appear to be no significant safety issues associated with treatment.

With proven effective processes and technologies in place to support production of sensitive cell therapies on a global scale, Vericel will continue to work to identify additional opportunities to expand our pipeline with new cell therapies that reflect our ongoing commitment to innovation and research. Our goal is to be the leader in production of cell therapies that address more unmet needs for patients around the world.