As the dust settled following the FDA’s precedent-setting approval of Kymriah (tisagenlecleucel, previously CTL-019), the chimeric antigen receptor T-cell (CAR T) immunotherapy developed by Novartis AG, analysts and observers began to parse the long-term impact to the players and broader implications for the field. Read More
Kymriah (tisagenlecleucel, CTL-019, Novartis AG) and other chimeric antigen receptor (CAR) T cells have been making their way through clinical trials as cell therapies. So the FDA’s Aug. 30 announcement that its approval of Kymriah was “a historic action today making the first gene therapy available in the United States” initially led to some head-scratching. Read More
Nightstar Therapeutics Ltd. filed to raise about $86.2 million in an IPO to advance its gene therapy candidate, NSR-REP1, for choroideremia (CHM), due to enter phase III trials in the first half of next year. Read More
Shares of Seattle-based Aptevo Therapeutics Inc. (NASDAQ:APVO) rose 58.7 percent on Friday to $2.11 as the company unveiled the sale of three noncore hematology drugs for up to $74.5 million to Saol Therapeutics and regained full rights to an experimental bispecific antibody immunotherapy for metastatic castration-resistant prostate cancer (mCRPC) from Morphosys AG. Read More
Ten years after Pfizer Inc. voluntarily yanked it from the market due to toxicity issues, acute myeloid leukemia (AML) drug Mylotarg (gemtuzumab ozogamicin) is returning to the market, following FDA approval Friday for use in adults with newly diagnosed disease whose tumors express the CD33 antigen. Read More
The implantation of dopaminergic neurons derived from human induced pluripotent stem cells (iPSCs) has for the first time been shown to restore nerve function and improve movement in monkey models of Parkinson’s disease (PD), in a new Japanese study. Read More
Jazz Pharmaceuticals plc, of Dublin, said the initial purchasers of its private offering of $500 million aggregate principal amount of 1.5 percent exchangeable senior notes due 2024 by Jazz Investments I Ltd., its wholly owned subsidiary, have exercised in full their option to purchase an additional $75 million aggregate principal amount of notes. Read More
Iovance Biotherapeutics Inc., of San Carlos, Calif., said the FDA granted fast track designation for LN-144, its adoptive cell therapy using its tumor-infiltrating lymphocyte technology, for the treatment of advanced melanoma. Read More
Sosei Group Corp.’s wholly owned subsidiary Heptares Therapeutics, of London, dosed the first healthy subject in its phase I trial testing HTL-0016878, a muscarinic M4 receptor agonist. Read More
As far as adult regeneration goes, the lung and liver are far better than most human tissues. Even those organs, however, can become unable to repair themselves in the face of persistent injuries, such as those caused by smoking or excessive drinking. Experimentally, regenerative capacity can be restored by stem cell transplants. Read More
