Clinical Trials & Products

Clinical Trials & Regenerative Medicine / Advanced Therapies Products

At the close of 2016, there were 804 clinical trials underway, with numerous approved and/or marketed products worldwide, many approved to be marketed in specific regions and/or countries.

This list only includes products reviewed and approved by internationally-recognized regulatory agencies, as well as products brought to market in the U.S. under The Food, Drug and Cosmetic Act and The Public Health Service Act. 

This includes such products as:

Strimvelis (GSK) - Strimvelisis an ex-vivo stem cell gene therapy which uses retroviral vector encoding adenosine deaminase gene transfer into hematopoietic stem/progenitor cells. Strimvelis is indicated for the treatment of adenosine deaminase severe combined immune deficiency. Approved by the EMA in May 2016. 

TEMCELL (JCR Pharmaceuticals Co Ltd, licensee of Mesoblast Ltd) – TEMCELL is an allogeneic mesenchymal stem cell product indicated for the treatment of acute radiation injury, chronic obstructive pulmonary disease, Crohn’s disease, graft-versus-host disease, Type I diabetes and myocardial infarction. Fully approved by the Japanese Ministry of Health, Labour and Welfare in October 2015 and conditionally approved in Canada & New Zealand (also known at Prochymal). 

Holoclar (Chiesi Farmaceutici) - Holoclar is a cell therapy based on autologous cultures of limbal stem cells. It regenerates a functional corneal epithelium allowing recovery of visual acuity. Holoclar is indicated for the treatment of moderate to severe limbal stem cell deficiency due to ocular burns. Granted conditional marketing authorization by the European Commission in February 2015.

Glybera (uniQure) - an adeno-associated virus-based gene therapy. It contains alipogene tiparvovec, which stimulates intravascular triglyceride metabolism (lipid breakdown) in lipoprotein lipase deficient patients. Glybera is indicated for the treatment of genetic lipoprotein lipase deficiency (LPLD). Approved by the European Commission in November 2012.

ChrondoCelect (TiGenix NV) - an autologous cell therapy product for the structural and functional repair of cartilage defects of the knee, indicated for the treatment of cartilage regeneration of single symptomatic cartilage defects of the femoral condyle of the knee. Approved by the European Medicines Agency in October 2009 and renewed for marketing authorization in July 2014.

Cupistem (Anterogen) - An autologous adipose derived mesenchymal stem cell treatment to reduce inflammation and regenerate damage joint tissues, indicated for the treatment of Crohn’s fistula. Approved for marketing by South Korea’s Food and Drug Administration in July 2012.

Caritstem (MEDIPOST) - a cellular therapeutic agent containing allogeneic human umbilical cord blood-derived mesenchymal stem cells, indicated for the treatment of knee cartilage defects such as traumatic articular cartilage, degenerative arthritis and rheumatoid arthritis. Approved for marketing by South Korea’s Ministry of Food and Drug Safety in January 2012.

Hearticellgram-AMI (FCB Pharmicell) - An autologous intracoronary bone marrow-derived mesenchymal stem cell injection for the treatment acute myocardial infarction. Approved for marketing by South Korea’s Food and Drug Administration in July 2011.

Hemacord / HPC Cord Blood - (Manufactured by the New York Blood Center) The U.S. FDA's only approved stem cell product to-date, a cord blood-derived product used for specified indications in patients with disorders affecting the body’s blood-forming system. Approved in 2011, name change to HPC Cord Blood in 2013. 

Dermagraft (Advanced Tissue Sciences) - a skin substitute used to help in the wound closure of diabetic foot ulcers. It is made from human cells (fibroblasts), placed on a dissolvable mesh material. Approved by U.S. FDA in September 2001.

Apligraf (Organogenesis, Inc. & Novartis AG) - a bi-layered living skin substitute made from a dermal layer of human cells (fibroblasts) in a bovine type I collagen and an overlying cornified epidermal layer of living human keratinocytes. Apligraf is indicated for the treatment of chronic venous leg ulcers, diabetic foot ulcer, epidermolysis bullosa and Keloids. Approved by U.S. FDA in June 2000. (Also marketed as Gintuit for a different application)


Examples of major milestones and key data events: 2016

Cell-based immuno-oncology programs

  • Kite Pharma files first US CAR-T therapy BLA submission for KTE-C19, investigational anti-CD19 CAR-T Therapy for treatment of relapsed/refractory aggressive b-cell non Hodgkin lymphoma – December 4, 2016
  • Novartis reports positive data from pivotal trial of CTL019 CAR-T cell therapy for treatment of r/r ALL, the first global CAR-T therapy trial – December 4, 2016

Gene therapy programs

  • Spark Therapeutics announces positive Phase III data for voretigene neparvovec to treat inherited retinal disease – August 10, 2016
  • BioMarin announces positive interim results from Ph I/II trial of BMN 270 gene therapy for treatment of severe hemophilia A – July 27, 2016
  • GSK’s ex vivo stem cell gene therapy Strimvelis receives European Marketing Authorization to treat very rare disease ADA-SCID – May 27, 2016

Cell-based therapy programs

  • Vericel’s MACI, the first autologous cellularized scaffold, approved by FDA for the repair of cartilage defects of the knee – December 13, 2016
  • ReNeuron announces positive results in Ph II trial of CTX cell therapy in patients with stroke disability – December 5, 2016
  • TiGenix announces positive 52-week results in Phase III trial of Cx601 in complex perianal fistulas in Crohn’s disease patients – March 7, 2016
  • Mesoblast licensee JCR Pharmaceuticals launches allogeneic cell therapy product TEMCELL in Japan for treatment of acute GvHD – February 24, 2016