Smack in the middle of the American Society of Hematology (ASH) annual meeting in San Diego, presenter Spark Therapeutics Inc. disclosed a licensing deal that harnesses the synthetic vaccine particles (SVP) platform technology developed by Selecta Biosciences Inc. for co-administration with Spark's gene therapy program in up to five targets, beginning with factor VIII (FVIII) in hemophilia A. Read More
Cyteir Therapeutics Inc., of Cambridge, Mass., presented preclinical data on a mechanism of cell death response used to target lymphoid malignancies preferentially. Read More
Matinas Biopharma Holdings Inc., of Bedminster, N.J., said patient dosing has commenced in the NIH-sponsored phase IIa study of lead anti-infective product candidate MAT2203 for the treatment of refractory mucocutaneous candidiasis infection. Read More
Novelion Therapeutics Inc., of Vancouver, British Columbia, said Aegerion Pharmaceuticals, an indirect, wholly owned subsidiary, entered into an agreement in principle to settle all claims in the class action shareholder lawsuit pending in the U.S. District Court for the District of Massachusetts. Read More
Republican health and appropriations leaders in the U.S. House and Senate are encouraging President-elect Donald Trump to keep Francis Collins on as the director of the NIH. Read More
SAN DIEGO – Fixing genetic diseases such as hemophilia, sickle-cell disease, and certain immunodeficiencies at their root, by replacing or repairing the faulty gene, has gone from dream to drug, with two gene therapies now approved in Europe, and a number of additional approvals expected on both sides of the Atlantic over the next few years. Read More
SAN DIEGO – Despite four decades of clinical research in acute myeloid leukemia (AML), the current standard-of-care induction therapy remains the 7 + 3 regimen, usually referring to seven days of cytarabine plus three days of daunorubicin, based on an initial paper published in 1973, said Harry Erba, during a Saturday media briefing at the American Society of Hematology (ASH) meeting. Read More
SAN DIEGO – Acute myeloid leukemia (AML) has joined the ranks of other cancers now getting the precision medicine treatment, so to speak. As researchers have identified different genetic mutations in AML patients, the next challenge has been to find ways to recruit and enroll patients quickly into appropriate treatment arms. That's where the newly launched BEAT-AML Master study comes in. Read More
SAN DIEGO – Gazyva (obinutuzumab), the drug considered a follow-on to blockbuster Rituxan (rituximab) – as well as a buffer to impending biosimilar versions of rituximab – improved progression-free survival (PFS) by 34 percent compared to Rituxan in patients with previously untreated follicular lymphoma (FL), according to detailed data presented at the American Society of Hematology (ASH) meeting, though the side-effect profile could give some physicians pause, as could the lack of an overall survival benefit. Read More
LONDON – Reneuron plc reported positive results in the phase II trial of its allogeneic human neural stem cell therapy in the treatment of disability caused by stroke, with 15 of 21 patients having a clinically significant response on at least one of several measures of efficacy. Read More
Theratechnologies Inc., of Montreal, said it closed its bought deal offering of 5.32 million shares at $3.10 each for gross proceeds of $16.5 million. Read More
