Clinical Trials & Products

Clinical Trials & Regenerative Medicine / Advanced Therapies Products

At the close of Q2 2016, there are 728 clinical trials underway, with numerous approved and/or marketed products worldwide, many only approved to be marketed in specific regions and/or countries.

This list 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.

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: Q2 2016

• Pluristem reports data showing PLX-PAD cells effective in treating Duchenne muscular dystrophy – June 22, 2016
• Adaptimmune receives positive opinion for Orphan Drug Designation in EU for SPEAR T-cell therapy targeting NY-ESO for treatment of soft tissue sarcoma – June 20, 2016
• Fate Therapeutics announces FDA Fast Track designation for ProTmune for the reduction of incidence and severity of acute GvHD in patients undergoing allogeneic hematopoietic cell transplantation – June 20, 2016
• Sangamo BioSciences announces FDA clearance of IND application for SB-913, ZFN-mediated genome editing treatment of MPS II – June 20, 2016
• AGTC announces Orphan Medicinal Product Designation in the EU for gene therapy to treat X-linked retinitis pigmentosa – June 7, 2016
• Kite Pharma grants access to EMA’s Priority Medicines (PRIME) regulatory support for KTE-C19 in the treatment of chemorefractory diffuse large B-cell lymphoma – June 1, 2016
• GSK’s ex-vivo stem cell gene therapy Strimvelis receives European Marketing Authorization to treat very rare disease ADA-SCID – May 27, 2016
• Abeona Therapeutics announces FDA allowance of investigational new drug for Phase I/II clinical study with ABO-101 gene therapy for patients with Sanfilippo syndrome type B – May 24, 2016
• Bone Therapeutics announces further positive efficacy in ALLOB Phase I/IIA delayed-union fracture trial – May 17, 2016
• Caladrius Biosciences receives Orphan Drug Designation for CLBS03 to treat type 1 diabetes – May 12, 2016
• European Commission grants Cytori Therapeutics Orphan Drug status to a broad range of Cytori cell therapy formulations when used for the treatment of hand dysfunction and Raynaud’s Phenomenon in patients with scleroderma – May 10, 2016
• Cellectis announces second baby with leukemia remains in remission six months after UCART19 T-cell treatment – May 6, 2016
• Agilis Biotherapeutics announces Orphan Drug Designation approval in Europe for treatment of Angelman syndrome – May 4, 2016
• Kite Pharma announces FDA Orphan Drug Designations for KTE-C19 in five additional indications: primary mediastinal B cell lymphoma, mantle cell lymphoma, follicular lymphoma, acute lymphoblastic leukemia and chronic lymphocytic leukemia – May 2, 2016
• Fibrocell receives Orphan Drug Designation from the FDA for FCX-013 for the treatment of localized scleroderma – April 25, 2016
• BioMarin provides encouraging preliminary data on first eight patients in hemophilia A gene therapy program – April 20, 2016
• Juno investigational CAR T cell product candidates JCAR018 and JTCR016 demonstrate encouraging clinical responses in patients with B-cell and mesothelioma cancers – April 20, 2016
• Kiadis Pharma presents positive data on the primary endpoint of its single dose Phase II trial with ATIR101 – April 4, 2016
• Mesoblast cell therapy shows disease-modifying effects on knee osteoarthritis – April 1, 2016