Cancer data typically steal the show at the American Society of Hematology (ASH) meeting, but hemophilia often gets plenty of play as well, and this time around could be especially compelling for investors in firms with therapies in the works for the clotting disorder. Read More
Marking its first regulatory win in the U.S. blood cancer space, Tokyo's Astellas Pharma Inc. has secured approval from the FDA for its oral acute myeloid leukemia (AML) therapy, Xospata (gilteritinib). Already approved in Japan, the FMS-like tyrosine kinase 3 (FLT3) inhibitor is now approved in the U.S. for the treatment of relapsed or refractory AML patients who test positive for the mutation, which is often associated with increased risk of relapse and poor survival. Read More
HONG KONG – Jiankui He, the Chinese scientist under the spotlight following his claim of the first gene editing use in human embryos, apologized for what he called the unexpected leak of information and defended his controversial trial during the Second International Summit on Human Genome Editing. Read More
LONDON – He Jiankui has faced up to critics and defended his work in creating what are claimed to be the world's first CRISPR/Cas9 gene-edited babies. Addressing the second International Summit on Human Gene Editing in Hong Kong Wednesday, He said he is "proud" of altering the CCR5 gene of twin girls given the pseudonyms Lulu and Nana, inserting a naturally occurring mutation that blocks infection by the HIV virus. (See story this issue.) Read More
TAIPEI, Taiwan – Relatively strong flows of capital and partnerships with global players are helping Asia's biotech sector boom, said speakers at the Asia Pacific Biotech Investment Forum on Wednesday. Read More
Anelixis Therapeutics LLC, formed in 2013 as a for-profit subsidiary of the nonprofit ALS Therapy Development Institute (ALS TDI), has begun enrolling the phase I dose-escalation study of AT-1501, a humanized anti-CD40 ligand (CD40L) monoclonal antibody. The single-site study at Massachusetts General Hospital (MGH) is examining safety and pharmacokinetics in six cohorts of healthy volunteers and one of individuals with amyotrophic lateral sclerosis (ALS). Top-line data are expected at the end of March, according to Steven Perrin, founder, president, CEO and sole employee of the Cambridge, Mass.-based company. Read More
Alliqua Biomedical Inc., of Langhorne, Pa., plans to spin off its subsidiary, Aquamed Technologies Inc., and merge it with TO Pharmaceuticals LLC, of Langhorne, Pa., to create a new public company called TO Pharma. After closing TO Pharmaceuticals, shareholders will hold a majority interest in TO pharma, which plans to list on Nasdaq. The transaction is expected to close by the first quarter of 2019. Read More
Prometic Life Sciences Inc., of Laval, Quebec, entered an at-the-market equity distribution agreement with Canaccord Genuity Corp. to sell up to $50 million of common shares over the course of the next 16 months. Prometic plans to use the capital to fund ongoing operations and advance its lead drug candidates. Read More
