Bio Pharma Dive

OCTOBER 22, 2020

The biotech has joined a group of investors to form a new gene therapy developer led by one of its longtime executives and backed with $107 million.

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Bio Pharma Dive

DECEMBER 8, 2021

Passage Bio and the University of Pennsylvania's Gene Therapy Program said they have begun preclinical research aimed at simultaneously developing multiple approaches to treating Huntington's.

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Bio Pharma Dive

APRIL 28, 2021

Far along in testing for a hemophilia treatment, BioMarin is expanding its gene therapy research into diseases of the central nervous system and aims to use some of the institute's technology.

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Bio Pharma Dive

MAY 17, 2022

An unusual collaboration among gene therapy developers suggests certain mutations could be behind "peculiar" side effects experienced by several patients treated in clinical trials.

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Bio Pharma Dive

OCTOBER 24, 2022

Already an investor in gene therapy, the Japanese pharma will buy 15% of the Texas biotech’s stock and gain options to two gene therapies for central nervous system disorders.

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Bio Pharma Dive

MARCH 20, 2023

As new gene therapies are developed to treat a growing number of indications, patients may soon have access to novel treatment options and potential cures.

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Bio Pharma Dive

APRIL 1, 2022

Taysha Gene Therapies is narrowing its research efforts and cutting around 35% of its workforce, the latest in a series of layoffs announced by makers of genetic medicines.

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Pharmaceutical Technology

DECEMBER 21, 2023

Genevoyager has announced the opening of contract development and manufacturing organisation facility to manufacture gene therapy products.

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Bio Pharma Dive

JULY 28, 2021

Flagship Pioneering, the biotech incubator that created Moderna, is helping fund Ring Therapeutics, a startup hoping to disrupt gene therapy development through the use of a different viral vector.

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Bio Pharma Dive

NOVEMBER 5, 2020

The setback for Bluebird's LentiGlobin comes amid a string of manufacturing-related delays for gene therapy developers.

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Bio Pharma Dive

OCTOBER 13, 2022

Gene therapies could help treat many ultra-rare diseases. But they may not get developed if drugmakers can’t build a sustainable business around them, CBER director Peter Marks said at a conference.

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Bio Pharma Dive

FEBRUARY 23, 2023

Graphite Bio and Sangamo are stopping work on their respective sickle cell gene therapies, while Intellia revealed partner Novartis ended development of its genetic treatment for the blood disease.

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Bio Pharma Dive

OCTOBER 19, 2021

Gene therapy could be a solution for the remaining 10%, but the path forward is challenging. Drugs from Vertex can effectively treat the lung disease in as much as 90% of patients.

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Pharmaceutical Technology

JANUARY 5, 2023

Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

MAY 24, 2023

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Topic sponsors are not involved in the creation of editorial content.

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Bio Pharma Dive

FEBRUARY 4, 2022

Gene therapy developers have struggled to secure reimbursement in Europe, making Orchard's deal with the National Health Service in England and Wales a notable accomplishment.

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Bio Pharma Dive

MAY 19, 2021

A number of drugmakers have recently faced development delays after the FDA's asked for more information on how they measure the potency of their products.

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Bio Pharma Dive

JULY 20, 2022

The Swiss drugmaker, which already owns rights to a marketed gene therapy for inherited vision loss, will work with startup Avista Therapeutics to develop better delivery tools for the complex treatments.

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Bio Pharma Dive

MARCH 30, 2022

The biotech company plans to reduce its workforce by 30%, or by about 65 employees, in the latest example of a gene therapy developer restructuring.

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Bio Pharma Dive

OCTOBER 30, 2020

A longer-than-expected timeline for developing a new manufacturing process means a key study for Axovant's top gene therapy likely won't begin until 2022.

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Bio Pharma Dive

OCTOBER 18, 2022

Like with Prevail Therapeutics, Lilly’s first gene therapy acquisition, the pharma’s planned buyout of Akouos includes a contingent value right that could hike the deal’s overall cost.

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Bio Pharma Dive

JANUARY 4, 2022

The biotech's treatment was one of the furthest along in testing for Fabry, a rare inherited disease that's become a target for gene therapy developers.

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Pharmaceutical Technology

JUNE 29, 2022

Terumo Blood and Cell Technologies and BioBridge Global subsidiary, GenCure, have entered a new partnership agreement to expand and integrate cell and gene therapy manufacturing solutions. This comprises the co-development of core processes and production operations.

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Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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Bio Pharma Dive

MARCH 8, 2022

The deal, which carries a $54 million upfront payment, could give Novartis access to as many as five of Voyager's capsids, which would be used to develop gene therapies for the central nervous system.

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Bio Pharma Dive

APRIL 1, 2021

Natalie Holles, who was appointed CEO of Audentes soon after Astellas acquired the gene therapy developer in 2019, has departed amid the company's integration.

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Bio Pharma Dive

DECEMBER 21, 2020

The FDA halted the AMT-061 program, which could be the first approved gene therapy for hemophilia B, because a patient who received the treatment appears to have developed liver cancer.

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Bio Pharma Dive

NOVEMBER 13, 2023

Forge Biologics operates a contract manufacturing business as well as develops its own gene therapies, which appears to have attracted the Japan-based food and biotechnology company.

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XTalks

MARCH 19, 2025

A 16-year-old patient died after treatment with Elevidys (delandistrogene moxeparvovec), Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy (DMD). The patient suffered acute liver failure several months after receiving the therapy in December. Sarepta is reviewing all available data.

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Pharmaceutical Technology

FEBRUARY 10, 2023

4D Molecular Therapeutics (4DMT), the California-based biotechnology company focused on developing gene therapies for rare and large market diseases, has had the FDA place a clinical hold onto its Fabry disease (FD) gene therapy program (4D-310).

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Pharmaceutical Technology

AUGUST 16, 2023

Several companies are developing robotics to automate cell and gene therapy manufacturing, but some challenges persist.

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Pharmaceutical Technology

MAY 23, 2023

Avrobio has announced a deal to sell its investigational haematopoietic stem cell (HSC) gene therapy programme , designed to treat cystinosis, to Novartis in an all-cash deal valued at $87.5m. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

JANUARY 31, 2023

Gene therapy company uniQure has entered into a global licensing agreement with Apic Bio for APB-102 to treat patients with amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1). APB-102 is designed to be a one-time, intrathecally administered gene therapy for ALS patients.

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Pharmaceutical Technology

OCTOBER 17, 2023

SpliceBio will license Spark Therapeutics’ propriety protein splicing platform to develop a gene therapy for renal disease.

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Pharmaceutical Technology

FEBRUARY 8, 2023

On February 7, at a town hall organised to discuss clinical trial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases.

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Bio Pharma Dive

JUNE 28, 2022

Positive results for the treatment, which was developed by U.K. biotech MeiraGTx, could rebuild expectations after another gene therapy from Biogen failed in the retinal disease.

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Pharmaceutical Technology

MARCH 21, 2024

Biotech executives discuss why developing gene therapies for central nervous system disorders remains difficult at a recent conference.

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