Pharmaceutical Technology

MAY 4, 2023

Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration. This will improve accessibility to services for AAV-mediated gene therapy programmes.

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Bio Pharma Dive

DECEMBER 8, 2023

Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2 million, while a competing genetic medicine also cleared Friday is priced at $3.1

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Bio Pharma Dive

JANUARY 24, 2024

An 11-year-old boy born deaf can hear after receiving Lilly's genetic medicine. Others, including biotechs in China and Regeneron in the U.S., are taking aim at the same drug target.

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Bio Pharma Dive

AUGUST 24, 2022

The New York biotech will invest in and develop up to three drugs with Gensaic, an emerging startup aiming to use the viruses that infect bacteria to deliver genetic medicines.

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Bio Pharma Dive

SEPTEMBER 12, 2024

Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.

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

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Bio Pharma Dive

FEBRUARY 12, 2021

Lysosomal storage disorders, diseases of cellular metabolism gone wrong, are the focus of a fast-expanding lineup of experimental genetic medicines, a research boom that recalls biotech's roots.

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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

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: Gene therapy delivery using viral vectors. Sanofi is one of the leading patent filers working with viral vectors.

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Bio Pharma Dive

OCTOBER 24, 2022

The biotech is the latest in a growing list of genetic medicine developers to sell themselves near record stock lows amid a challenging funding environment.

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Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.

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Bio Pharma Dive

MARCH 15, 2021

Softbank and Fidelity joined a large group of investors in a $525 million Series C investment into Elevate, a high-powered genetic medicine startup with an unusual business model.

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Pharmaceutical Technology

JANUARY 16, 2023

CARsgen Therapeutics has announced a col labor ation with Huadong Medicine to commercialise zevorcabtagene autoleucel (zevor-cel), CT053, in mainland China. It comprises autologous T cells that are modified genetically with a CAR including a complete human anti-BCMA single-chain fragment variant that has a high binding affinity.

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Pharmaceutical Technology

FEBRUARY 27, 2024

Making therapies feasible from biological, regulatory, and commercial standpoints drives scalable genetic medicine.

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AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 30, 2024

Researchers see a greater need for new generation therapeutics to propel precision medicine. The development of innovative therapeutic approaches in healthcare encompass a variety of fields, including gene therapy, personalized medicine, immunotherapy, and advanced biologics.

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Bio Pharma Dive

DECEMBER 16, 2021

The new facility cost nearly $70 million to build, and is part of a major push by the pharma giant to become a leader in genetic medicine manufacturing.

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Pharmaceutical Technology

APRIL 9, 2025

The potential for cell and gene therapies is growing, offering groundbreaking treatments for rare genetic disorders and promising to reshape the future of precision medicine.

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Bio Pharma Dive

MARCH 20, 2024

million list price, the highest of any genetic medicine to come to market. Orchard is counting on the long-term data it’s accrued to convince insurers to cover Lenmeldy’s $4.25

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Pharmaceutical Technology

MAY 17, 2023

Under the deal, Prevail obtains exclusive rights to use Scribe’s CRISPR X-Editing (XE) technologies to develop the medicines. The deal will see the integration of Scribe’s new CRISPR by Design approach and Prevail’s expertise in developing genetic medicines for neurological disorders for specific genetic targets.

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Bio Pharma Dive

FEBRUARY 1, 2023

Wilson Bryan, a key decision maker in the agency’s oversight of genetic medicines, is expected to leave next month amid an organizational revamp of his office, the FDA confirmed.

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Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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Pharmaceutical Technology

JUNE 22, 2023

Ultragenyx Pharmaceutical has opened its new gene therapy manufacturing facility in Bedford, Massachusetts, US. The company will produce gene therapies in the new advanced facility, using its proprietary Pinnacle PCL (producer cell line) manufacturing platform.

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Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Background: The Advancement of Cell and Gene Therapies.

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Pharmaceutical Technology

MAY 24, 2023

The RMAT designation programme is intended to accelerate the drug’s development and review processes for products, including gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Topic sponsors are not involved in the creation of editorial content.

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Pharmaceutical Technology

FEBRUARY 24, 2023

AbbVie is expanding its strategic collaboration with Capsida Biotherapeutics for the development of targeted genetic medicines to treat eye diseases with high unmet needs. In pursuing the promise of genetic medicine-based therapeutics, AbbVie continues to expand our capabilities, and we are pleased to have Capsida as a partner.”

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Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years. According to GlobalData’s clinical trials database, there are currently 1,231 planned and ongoing trials for gene therapies and gene-modified cell therapies alone.

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pharmaphorum

FEBRUARY 23, 2022

Takeda has forged another alliance as it continues a push into gene therapy, agreeing a deal worth up to $2 billion with Code Biotherapeutics for opt-in rights to four candidates for rare diseases. The post Takeda grows in gene therapies again with $2bn Code Bio deal appeared first on. Last October it signed a $3.6

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pharmaphorum

MAY 23, 2022

PTC Therapeutic’s Upstaza gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of the commercial prospects for gene therapies in the bloc. The post PTC gets green light for gene therapy Upstaza in EU appeared first on.

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XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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pharmaphorum

JANUARY 3, 2023

As December 2022 closed out, Pfizer announced positive top-line results from its phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec (SPK-9001), its investigational gene therapy for treatment of adult males with moderately severe to severe haemophilia B.

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XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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pharmaphorum

MAY 21, 2021

US biotech bluebird bio has had a challenging time in the last few months, so a recommendation for EU approval of its gene therapy for adrenoleukodystrophy (ALD) will give it a lift. After two years’ follow-up, 90% of the boys given the gene therapy showed minimal loss of function and were still alive.

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Medical Xpress

DECEMBER 21, 2022

Ten young children born without functioning immune systems and lacking the ability to fight infections are on track for healthier lives thanks to a new gene therapy treatment pioneered at UC San Francisco, reports a Dec. 22 study in the New England Journal of Medicine.

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Pharmaceutical Technology

JUNE 29, 2022

The European Commission (EC) has granted marketing authorisation for Gilead Sciences’ subsidiary Kite’s CAR T-cell therapy, Yescarta (axicabtagene ciloleucel), to treat adults with relapsed or refractory follicular lymphoma (FL). . The treatment has maintained orphan medicinal product designation for this indication.

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XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

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Medical Xpress

JANUARY 3, 2025

Researchers at Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine pioneered a first of its kind gene therapy model that offers a potential breakthrough in treating X-linked sideroblastic anemia (XLSA), a rare congenital anemia caused by mutations in the ALAS2 gene crucial for the synthesis of heme, (..)

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Pharmaceutical Technology

JUNE 29, 2022

The next wave of medicine is well on course to be cell and gene-based. In September 2021, GlobalData figures revealed there to be 1,320 industry-sponsored regenerative medicine and advanced therapy trials ongoing worldwide. Meanwhile, the pharma industry is undergoing somewhat of a transformation itself.

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