00:28 , Jun 7, 2019 |  BioCentury  |  Product Development

How early biomarkers and constant iteration fuel Vertex's R&D efficiency

As Vertex prepares its fourth NDA for a cystic fibrosis drug in six years, EVP of Global Research and CSO David Altshuler credits the biotech’s R&D efficiency to a focus on agents that show more...
22:20 , May 31, 2019 |  BC Extra  |  Company News

May 31 Company Quick Takes: NMPA accepts two BeiGene applications, approves Hengrui's PD-1 inhibitor; and more

NMPA accepts applications for BeiGene's tislelizumab, Abraxane  China's National Medical Products Administration has accepted an sNDA from BeiGene Ltd. (NASDAQ:BGNE; HKSE:6160) for tislelizumab to treat previously treated, locally advanced or metastatic urothelial carcinoma. The anti-PD-1...
22:37 , May 30, 2019 |  BC Extra  |  Company News

May 30 Company Quick Takes: Unity in-licenses protein for cognitive decline, plus Astellas, Organovo, Iovance

Unity in-licenses UCSF protein for cognitive improvement  Unity Biotechnology Inc. (NASDAQ:UBX) licensed from the University of California San Francisco exclusive, worldwide rights to IP relating to KLA, including its use to reverse or prevent cognitive...
17:48 , Mar 28, 2019 |  BC Innovations  |  Translation in Brief

Tuning metabolism in liver organoids

An Alnylam-MIT collaboration has used RNAi technology to tune enzyme expression in hepatocyte-based organoids to better model drug metabolism during tox screens. The collaboration with Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) built on a series of papers...
21:22 , Jan 31, 2019 |  BC Innovations  |  Emerging Company Profile

Apic: Dual-gene therapies

Apic Bio Inc.'s APB-101 is a dual-function gene therapy for AAT deficiency that silences the mutant AAT gene and augments wild-type AAT expression, which the company believes gives it greater efficacy and lower liver toxicity...
03:40 , Jan 18, 2019 |  BC Innovations  |  Product R&D

Modality moves at Vertex

In licensing a suite of newly discovered gene editing tools, Vertex Pharmaceuticals Inc. is broadening its options for solving problems that its core small molecule technology can’t address. On Jan. 3, Vertex announced a deal...
17:41 , Jan 11, 2019 |  BC Week In Review  |  Financial News

Venture roundup: Apic, Stelexis, Biond, Keros

Gene therapy company Apic Bio Inc. (Cambridge, Mass.) and cancer company Stelexis Therapeutics LLC (New York, N.Y.) each announced series A rounds of at least $40 million. Other companies that announced venture rounds include Biond...
23:42 , Jan 8, 2019 |  BC Extra  |  Financial News

Venture roundup: Apic, Stelexis, Biond, Keros

Gene therapy company Apic Bio Inc. (Cambridge, Mass.) and cancer company Stelexis Therapeutics LLC (New York, N.Y.) each announced series A rounds this week of at least $40 million. Other companies that announced venture rounds...
21:02 , Dec 14, 2018 |  BC Week In Review  |  Company News

Mereo, OncoMed announce merger

Mereo BioPharma Group plc (LSE:MPH) announced a merger with OncoMed Pharmaceuticals Inc. (NASDAQ:OMED); the resulting entity will focus on development of Mereo's orphan disease pipeline and will be listed on London Stock Exchange and NASDAQ....
18:27 , Nov 9, 2018 |  BC Week In Review  |  Clinical News

Adverum reviewing capsid after discontinuing AAT gene therapy

Adverum Biotechnologies Inc. (NASDAQ:ADVM) will discontinue development of gene therapy ADVM-043 to treat α-1 antitrypsin (AAT; A1AT; SERPINA1) deficiency. The company said that although ADVM-043 was well tolerated among six patients in the open-label, U.S....