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....
14:45 , Nov 2, 2018 |  BC Extra  |  Clinical News

Adverum reviewing capsid after discontinuing AAT gene therapy

Adverum Biotechnologies Inc. (NASDAQ:ADVM) sank $1.21 (27%) to $3.34 on Friday after it said it will discontinue development of gene therapy ADVM-043 to treat α-1 antitrypsin (AAT; A1AT; SERPINA1) deficiency. The company said late Thursday...
16:35 , Oct 26, 2018 |  BC Week In Review  |  Clinical News

Intellia CRISPR-Cas9 platform creates gene insertion edits in vivo

Intellia Therapeutics Inc. (NASDAQ:NTLA) presented data at the European Society of Gene and Cell Therapy meeting in Lausanne, Switzerland, showing that its CRISPR-Cas9 platform is capable of creating both gene disruption and the more challenging...
18:03 , Sep 28, 2018 |  BC Week In Review  |  Financial News

Gene therapy play LogicBio proposes IPO

Gene therapy company LogicBio Therapeutics Inc. (Cambridge, Mass.) proposed to raise up to $86 million in an IPO on NASDAQ underwritten by Jefferies, Barclays, William Blair and Chardan. LogicBio is developing gene therapies to address...