Companies to Watch: Sensorion
Sensorion is a public company in France developing new medicines for diseases of the inner ear, with two compounds in the pipeline: SENS- 111 for treating acute unilateral vestibulopathy...
Is Hearing Loss a Necessary Price for Children to Pay for Life-Saving Chemotherapies
We spoke to Nawal Ouzren, CEO of Sensorion, which will participate in the FDA meeting and is developing a treatment for sudden hearing loss that may be able to help pediatric cancer patients experiencing cisplatin-induced ototoxicity.
Daniel de Boer, CEO of ProQR, Explains RNA Treatment, QR-313, for DEB
In a recent interview with Daniel de Boer, CEO of ProQR, Rare Disease Report® discussed the company’s new RNA therapy, QR-313, for the treatment of dystrophic epidermolysis bullosa (DEB).
Cancer Treatment Developer Rain Therapeutics Closes $18.4 Million Series A
Rain Therapeutics Inc., a Fremont, Calif.-based developer of small molecule therapeutics for patients with cancer, has secured $18.4 million in Series A funding. Biotechnology Value Fund led the round, with participation from investors including Perceptive Advisors and Auckland UniServices Ltd.’s Inventors Fund.
Rain Therapeutics nets $18M to push lung cancer asset into phase 2
Rain Therapeutics raised $18.4 million in series A funding, which will advance its lead program, a hypoxia-activated prodrug, in a population of patients with non-small cell lung cancer for whom there are no treatments.
The Case for a Daily Self-Injection to Treat Hemophilia
We spoke to Nassim Usman, CEO of Catalyst, about the company’s approach to hemophilia, how its drugs work, and why he believes his company’s pipeline may provide better alternatives for hemophilia patients.
Samumed CMO Hopes To Reach Drug Approvals Via The Wnt Pathway
The clinical-stage biotech has diverse pipeline generated by its Wnt signalling pathway technology platform, and aims to get its first drug to market in 2020.
“This is the First Time CAR-T Has Ever Worked on Acute Myeloid Leukemia“
Celyad has reported clinical results showing the first time a patient has fully recovered from acute myeloid leukemia thanks to a CAR-T therapy, and without the need for chemotherapy preconditioning.
Bloomberg Markets: ICO Compliance, Drug Development
Amit Munshi President/CEO Arena Pharmaceuticals Inc Discussing drug development in ulcerative colitis space.
Arena CEO: We have two great assets and are trying to build a company around that
Looking at the performance of Arena Pharmaceuticals with CEO Amit Munshi and CNBC's Meg Tirrell.
Orchard Gains GSK’S Rare Disease Gene Therapy Portfolio
GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) said it will divest its rare disease gene therapy portfolio, including Strimvelis, to Orchard Therapeutics Ltd. (London, U.K.).
UK gene therapy firm Orchard plans stock offer after GSK deal
Britain’s Orchard Therapeutics, which has already raised more than $140 million to fund its work in gene therapy, plans another private sale of shares following its acquisition of a portfolio of GlaxoSmithKline...
OSE Immunotherapeutics Adds Boehringer Ingelheim To Its Pharma Partnering List
Collaborating with the German family-controlled drug maker on OSE's novel checkpoint inhibitor aligns with French firm's strategy of partnering its portfolio candidates.
Reata Pharmaceuticals: Return & Reach Beyond
The Enterprisers: Life Science Leadership In Action. What good can come of setback? What is the value, if any, in so-called failure? Must a “failed” clinical trial wind up in the great waste bin of misbegotten science?
Trials for potential ALS treatment spark cautious hope
As an engineering professor at Lehigh University in Pennsylvania, Richard P. Vinci has run plenty of experiments. He specializes in figuring out what makes materials break...
Boehringer, OSE tie up in I-O deal that could total more than $1.4B
Boehringer Ingelheim and OSE Immunotherapeutics are partnering to develop the latter's checkpoint inhibitor, OSE-172, for the treatment of advanced solid tumors.
ALS patient hopes stem cell treatment will be “a powerful weapon”
Forty-one-year-old David Neufeglise was a healthy family man and mechanical engineer when he started noticing constant twitching in his arm...
RNAi Therapeutics: To the Liver … and Beyond
RNAi Therapy Components Are Surviving Degradation, Staying Potent, and Reaching More Targets Than Ever