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BioWorld - Tuesday, December 9, 2025
Home » Topics » Drugs » Gene therapy

Gene therapy
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DNA illustration

No drama as Horama raises $25M, becomes Coave Therapeutics to reflect wider focus

July 21, 2021
By Cormac Sheridan
DUBLIN – Coave Therapeutics unveiled a new identity and a new gene therapy platform, as it closed a €21.2 million (US$25 million) extension to its long-running series B round, which takes the total raise to €33 million.
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RNA strand

Shape of RNA to come: Series Bs boost gene therapy companies

July 15, 2021
By Lee Landenberger
Private biopharma investment in 2021 continues to outpace that of the past two years as two gene therapy companies, Shape Therapeutics Inc. and Kriya Therapeutics Inc., posted series B financings this week totaling more than $200 million.
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National pool needed for cell and gene therapies?

July 9, 2021
By Mari Serebrov
While U.S. lawmakers continue their debate on reducing spending for prescription drugs, government payers are exploring innovative reimbursement ideas to cover gene and cell therapies that could cost millions of dollars for a cure or a durable effect against rare diseases.
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Biogen’s Nightstar buy questioned as depression drug makes progress in phase III

June 15, 2021
By Richard Staines
Biogen Inc. has announced contrasting results from phase III trials of therapies for a rare eye disease and depression, following last week’s controversial FDA approval of Alzheimer’s drug Aduhelm. The Cambridge, Mass.-based firm said a phase III gene therapy study in the rare retinal disease choroideremia missed its primary and secondary endpoints, although the news was better from a potential therapy for major depressive disorder.
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Brain and DNA

Vectory taking gene therapy approach to Alzheimer’s with $37.6M seed round

June 15, 2021
By Nuala Moran
LONDON – The pioneers of gene therapy in Europe now aim to take the field to the next level, moving into the development of virally delivered antibodies in the treatment of central nervous system and muscular diseases, with Alzheimer’s disease one of the first targets.
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Red blood cells, DNA

Holds on four Bluebird gene therapy studies are released by the FDA

June 7, 2021
By Lee Landenberger
The FDA has lifted clinical holds on four studies from Bluebird Bio Inc., following recent similar actions with other gene therapy programs. Two of the studies concern phase I/II and phase III clinical trials of the gene therapy Lentiglobin (BB-1111) in treating sickle cell disease. The remaining two studies are phase III clinical trials of betibeglogene autotemcel gene therapy, which share a vector with Lentiglobin, for treating transfusion-dependent beta-thalassemia.
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CAR T cell attacking cancer cells

In CAR T therapy, China excels in speed but quality remains a concern

June 1, 2021
By Elise Mak
China is making strides in cell and gene therapy, notably so with a 61% surge in the number of clinical trials in six years, a new report by Ernst & Young showed. While analysts noted the increasing innovation efforts and cross-border collaborations, concerns remain if quality will be compromised by speed.
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In CAR T therapy, China excels in speed but quality remains a concern

May 28, 2021
By Elise Mak
China is making strides in cell and gene therapy, notably so with a 61% surge in the number of clinical trials in six years, a new report by Ernst & Young showed. While analysts noted the increasing innovation efforts and cross-border collaborations, concerns remain if quality will be compromised by speed.
Read More
Eye and DNA

Vedere successor launches with $77M series A financing

May 18, 2021
By Michael Fitzhugh
Less than a year after Novartis AG's acquisition of optogenetics specialist Vedere Bio Inc., its successor Vedere Bio II Inc. is launching with $77 million in series A financing, led by Octagon Capital. The company will develop earlier-stage assets than those Novartis purchased, including new, mutation-agnostic optogenetics technology to improve upon current gene therapies aimed at restoring functional vision to people with vision loss due to photoreceptor death.
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Capsigen enters AAV deal with Biogen worth up to $1.3B

May 11, 2021
By Lee Landenberger
Little more than a year ago, when COVID-19 lockdowns began, turned out to be a prime time for finding the right funding and partners. That’s when privately held Capsigen Inc. was on a search that ended with a Biogen Inc. deal that could add up to $1.3 billion.
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