Bio Pharma Dive

AUGUST 29, 2024

Far fewer venture funding rounds were closed by cell and gene therapy developers over the first six months of 2024 than in prior years. Experts say there are several factors.

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Bio Pharma Dive

JANUARY 8, 2021

Gene therapy developers will need to address a series of setbacks that have shaded optimism in the sector's fast growth.

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Gene Therapy Gene Development 350

Bio Pharma Dive

JULY 20, 2022

The deal, which is worth $60 million upfront, broadens Vertex’s reach into gene editing and expands Verve’s research beyond heart disease.

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Gene Editing Gene Development Drugs 330

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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Gene Therapy Gene Development Manufacturing 245

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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Gene Therapy Gene Development Research 323

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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Gene Therapy Gene Development 273

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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Gene Therapy Gene Development Manufacturing 243

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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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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Gene Therapy Gene Development Genetics 260

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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Gene Therapy Development Gene Production 362

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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Gene Therapy Gene Development 351

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

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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Gene Therapy Gene Development Drugs 297

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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Gene Therapy Gene Development Engineer 237

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. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Development Manufacturing 242

Bio Pharma Dive

MARCH 16, 2021

Nearly $20 billion was invested in biotechs developing cell-, gene- and tissue-based treatments in 2020, according to a new report from industry group ARM.

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Gene Therapy Gene Development 337

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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Gene Therapy Gene Scientist Clinical Trials 343

Bio Pharma Dive

FEBRUARY 23, 2021

Guide Therapeutics, the company Beam acquired, is working on ways to more efficiently deliver gene editing therapies into the body.

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Gene Editing Gene Gene Therapy Development 287

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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Gene Therapy Gene Development 344

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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Gene Therapy Gene Manufacturing Development 330

Bio Pharma Dive

AUGUST 7, 2020

Development of EDIT-101, the first CRISPR-based therapy to be used in a company-led trial to alter genes within a person's body, will now move forward under Editas' sole ownership.

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Gene Editing Gene Trials Development 336

Bio Pharma Dive

FEBRUARY 1, 2022

The collaboration with GenEdit, a South San Francisco startup developing new ways of delivering gene editing tools into the body, dates back to December 2020, but has recently begun to advance.

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Gene Editing Gene Development 330

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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Gene Therapy Gene Development 346

Bio Pharma Dive

JUNE 30, 2021

The companies aim to develop therapies for complement-driven diseases, a specialty of Apellis and relatively new research ground for gene editing.

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Gene Editing Gene Development Research 310

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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Gene Therapy Gene Development 310

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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Gene Therapy Gene Development 271

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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Gene Therapy Gene Marketing Development 297

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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Gene Therapy Gene Research Development 288

Bio Pharma Dive

NOVEMBER 18, 2024

Explore solutions to reduce variability in gene expression during cell line development.

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Gene Expression Gene Development 246

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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Gene Therapy Manufacturing Gene Development 290

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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Gene Therapy Gene Development 297

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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Gene Therapy Gene Development 290

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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Gene Therapy Gene Development 281

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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Gene Therapy Gene Contract Manufacturing Manufacturing 281

Bio Pharma Dive

FEBRUARY 22, 2023

The messenger RNA specialist said Wednesday it is teaming up with Life Edit Therapeutics to develop therapies that can modify genes “in vivo.”

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Gene Editing Gene In-Vivo RNA 259

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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Gene Therapy Gene Development 281

XTalks

NOVEMBER 14, 2024

PTC Therapeutics has gained US Food and Drug Administration (FDA) approval for its new gene therapy, Kebilidi (eladocagene exuparvovec), for treating aromatic L-amino acid decarboxylase (AADC) deficiency. The FDA requires a confirmatory study to validate these findings, which is ongoing.

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Gene Therapy FDA Approval Gene Clinical Trials 105