SOUTH SAN FRANCISCO, Calif. — Denali Therapeutics Inc. (NASDAQ:DNLI), a biopharmaceutical company developing a broad portfolio of therapeutic candidates for neurodegenerative diseases, today announced that it has commenced dosing of its small molecule inhibitor of RIPK1 in a Phase 1 clinical trial in healthy volunteers and achieved proof of concept of its large molecule blood-brain barrier delivery platform technology in nonhuman primates. In addition, the Company reported financial results for the fourth quarter and full year ended December 31, 2017 and appointed Peter Klein to the Board of Directors.

“The recent clinical progress with our RIPK1 inhibitor and LRRK2 inhibitor programs, along with achieving proof of concept for our blood-brain barrier delivery platform for biologics in nonhuman primates, are important milestones towards our goal of developing medicines for patients suffering from neurodegenerative diseases,” said Ryan Watts, Ph.D., CEO. “Furthermore, we are very excited about initiating our collaboration with Takeda, which has allowed us to expand efforts on our blood-brain barrier delivery technology platform.”

Read More

In a fashionable fitted blazer, Rachel Haurwitz looks the part of a polished executive when she meets me in the largest conference room of her company, Caribou Biosciences. She’s sharp and tactical as she speaks, even though it was just a few years ago that Haurwitz traded her lab coat for a tailored suit and stepped up to lead a staff of 46 as Caribou’s CEO. And while the company’s Berkeley, California, office has the unassuming look of most Bay Area startups — open floor plan with scattered desks, swivel chairs and the mandatory Ping-Pong table — tucked away in back is a bright white laboratory where Haurwitz, 32, and her team are using CRISPR, a first-of-its-kind gene editor, to revolutionize food and medicine.

To date, research on CRISPR has focused on treating disease: Clinical trials in China have used the gene editor to fight cancer cells, labs in the U.S. will soon begin doing the same and there’s hope that it will eventually cure rare genetic diseases like Huntington’s. But Haurwitz believes that CRISPR can and should be mobilized to meet challenges beyond the human health field — to include agriculture and industrial biology — and she built her company around that objective. “Any market with bio-based products will be changed by gene editing,” Haurwitz tells OZY.

Read More

Adaptimmune has seen partial responses in three of the first four myxoid/round cell liposarcoma (MRCLS) patients treated with its T cells. The early data raised hopes the GlaxoSmithKline-partnered drug will be effective in multiple types of solid tumor, sending Adaptimmune shares up nearly 20%.

Abingdon, U.K.-based Adaptimmune first posted clinical data linking NY-ESO SPEAR T cells to responses in synovial sarcoma patients in 2015. Those results gave Adaptimmune preliminary validation that it can engineer T cells to take out solid tumors but left the biotech needing more evidence to show its platform is effective against a range of cancers.

The MRCLS data mark a small step toward that goal. MRCLS, like synovial sarcoma, is a soft-tissue sarcoma but the underlying genetics and clinical manifestations of the two diseases are different.

Read More

Cambridge, UK and Indianapolis: Acacia Pharma Group plc (“Acacia Pharma”, the “Company” or the “Group”), (EURONEXT: ACPH), a hospital pharmaceutical company focused on the development and commercialisation of new nausea & vomiting treatments for surgical and cancer patients, announces that data and analysis from its Phase 3 prophylaxis trial with intravenous amisulpride (BAREMSIS®) in combination with standard antiemetics have been published in the Online First edition of Anesthesiology, the peer-reviewed medical journal of the American Society of Anesthesiologists (ASA) (Kranke et al.). Positive headline results were first announced by the Company in January 2016.

The Phase 3 trial demonstrated that BAREMSIS is safe and resulted in a statistically significant reduction in the emergence of post-operative nausea & vomiting (PONV) when given in combination with an antiemetic drug from another class to adult patients undergoing elective procedures who are at high risk of PONV.

“Tens of millions of Americans undergo surgery each year and many suffer with nausea and vomiting after their operation,” said Peter Kranke, M.D., Professor of Anaesthesiology at the University of Würzburg in Germany and the trial’s lead investigator. “PONV contributes to patient distress, can delay recovery after surgery and increases hospital costs. Patients with multiple risk factors for PONV require a multimodal approach for its prevention, including using a combination of anti-nausea drugs with different mechanisms of action, since it cannot be predicted which pathway(s) will be active in a patient.”

Read More

London, UK  — Orchard Therapeutics, a clinical-stage biotechnology company dedicated to transforming the lives of patients with rare disorders through innovative gene therapies, today announced that one of its scientific founders and scientific advisory board member, professor Donald Kohn, has been recognized with the Pediatric Blood and Marrow Transplant Consortium (PBMTC) Lifetime Achievement Award on Feb. 22, 2018 in Salt Lake City.

The PBMTC Lifetime Achievement Award recognizes outstanding individuals who have made major contributions to the field of pediatric hematopoietic stem cell transplantation. The award highlights and honors the contributions and careers of individuals whose work exemplifies the PBMTC mission statement: “To support research and education to improve the availability, safety, and efficacy of hematopoietic cell transplantation and other cellular therapeutics for children and adolescents.”

Professor Kohn has focused his career on the development of hematopoietic stem cell gene therapy for immunodeficiencies, hemoglobinopathies and inborn errors of metabolism. His work includes the development of tools for efficient gene addition or correction, and persistent transgene expression, for the amelioration of genetic disease.

Read More

HONG KONG (Reuters) – Chinese drug developer Hua Medicine is planning to raise at least $400 million in an initial public offering in Hong Kong in the latest in a series of biotech floats in the city, said people with knowledge of the matter.

Hong Kong is formulating new rules for early-stage drug developers in an effort to woo companies in the fast-growing sector – notably those in China – into choosing the city over New York, the traditional hub for initial public offerings from the industry.