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

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

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 255

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 261

Bio Pharma Dive

JANUARY 4, 2023

The Lilly-owned subsidiary will pay $55 million to gain access to Capsida’s AAV gene therapy technology in a deal aimed at central nervous system disorders.

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Gene Therapy Gene Development 321

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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Gene Therapy Gene Development 246

Pharmaceutical Technology

DECEMBER 7, 2023

GlobalData’s report assesses the drugs in the Calcitonin Gene Related Peptide Type 1 Receptor pipeline by therapy areas, indications, stages, MoA, RoA, molecule type and the key players in the development pipeline.

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Gene Development Drugs 130

Pharmaceutical Technology

DECEMBER 7, 2023

GlobalData’s report assesses the drugs in the Calcitonin Gene Related Peptide Type 1 Receptor pipeline by therapy areas, indications, stages, MoA, RoA, molecule type and the key players in the development pipeline.

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Gene Development Drugs 130

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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Gene Therapy Gene Development Genetics 270

Pharmaceutical Technology

SEPTEMBER 8, 2023

Otsuka Pharmaceutical has entered a partnership with ShapeTX for the development of gene therapies to treat ocular diseases.

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Gene Therapy Gene Development 147

Bio Pharma Dive

MARCH 16, 2022

Draft recommendations from the agency were generally straightforward, analysts said, although the document emphasized the potential safety risks of gene editing.

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Gene Editing Gene Medicine Development 316

Pharmaceutical Technology

FEBRUARY 8, 2024

Despite several ground-breaking developments, pharmaceutical and biotech companies are often reluctant to invest in cell and gene therapy.

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Gene Therapy Gene Medicine Development 246

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 271

Bio Pharma Dive

JANUARY 6, 2024

The Emeryville, California- based gene therapy developer has several preclinical programs in development with Moderna, Ionis and Affini-T.

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Gene Editing Gene Gene Therapy Development 200

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 321

Pharmaceutical Technology

DECEMBER 14, 2022

Merck and Synplogen have signed a non-binding Memorandum of Understanding (MoU) to expedite the development and manufacturing of viral vector-based gene therapy applications. The firms intend to merge their expertise to provide simplified viral vector gene therapy development, production and testing in Japan.

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Gene Therapy Gene Development Manufacturing 130

Pharmaceutical Technology

FEBRUARY 1, 2024

The French government grant will cover preclinical and clinical research for Vivet’s gene therapy for cerebrotendinous xanthomatosis.

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Gene Therapy Gene Development Clinical Research 147

BioPharma Reporter

MARCH 5, 2024

Form Bio and Ginkgo Bioworkshave joined forces in what they call a groundbreaking partnership aimed at advancing AAV gene therapy design and development.

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Gene Therapy Gene Development 105

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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Gene Therapy Gene Licensing Licensing 246

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 263

Bio Pharma Dive

JUNE 12, 2023

Beacon Therapeutics will be led by former executives of other ocular gene therapy developers, including Nightstar, Gyroscope and AGTC.

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Gene Therapy Gene Development 146

Pharmaceutical Technology

JUNE 8, 2023

GenScript ProBio has collaborated with the New York Blood Center Enterprises’ business unit, called Comprehensive Cell Solutions (CCS), to accelerate the development and manufacture of cell and gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Development Antibody 130

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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Gene Therapy Manufacturing Gene Development 246

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 254

Pharmaceutical Technology

JULY 20, 2022

Avista Therapeutics, a University of Pittsburgh Medical Center (UPMC) spinout, has entered a collaboration with Roche for developing new AAV gene therapy vectors for eyes. Roche will use these new capsids to carry out preclinical, clinical and marketing activities for gene therapy programmes. Avista is entitled to receive $7.5m

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Gene Therapy Gene Development In-Vivo 147

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. In 2022, Avrobio’s investigational gene therapy, AVR-RD-04, received a rare paediatric disease designation from the US Food and Drug Administration.

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Gene Therapy Gene Development Drugs 258

Pharmaceutical Technology

SEPTEMBER 15, 2023

Cure Genetics has entered a partnership and licensing deal with Frametact to develop gene therapy for familial neurological ailments.

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Genetics Gene Gene Therapy Licensing 246

Bio Pharma Dive

JULY 17, 2023

Scribe will receive $40 million upfront from Sanofi in a collaboration initially focused on developing an in vivo gene editing treatment for sickle cell disease.

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Gene Editing Gene In-Vivo Research 237

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 285

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 313

Camargo

DECEMBER 20, 2021

One of the biopharmaceutical industry’s most closely watched forthcoming regulatory developments is the FDA’s imminent reauthorization of the Prescription Drug User Fee Act (PDUFA). Many of the advancements included in PDUFA VII are driven in large part by an increase in the development of innovative products such as cell and gene therapies.

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Gene Therapy Gene Development Production 184

Bio Pharma Dive

MARCH 19, 2024

clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.

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Gene Therapy Gene FDA Approval Development 161

Pharmaceutical Technology

APRIL 3, 2023

The deal will see Polyplus join the German life science group’s portfolio allowing the latter to leverage expertise in transfection reagents and plasmid DNA for gene therapy. Polyplus, based in Strasbourg, France, produces key components in the production of viral vectors used in cell and gene therapies.

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Gene Therapy Gene DNA Reagent 246

Pharmaceutical Technology

MAY 30, 2023

Amplo Biotechnology has received a fast track phase I/II STTR grant from the NIH-NIAMS [National Institutes of Health’s National Institute of Arthritis and Musculoskeletal and Skin Diseases] for its gene therapy AMP-201. The company will receive substantial funding to advance AAV-ColQ gene therapy.

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Gene Therapy Gene Development Clinical Trials 244

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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Gene Therapy Gene Gene Editing Manufacturing 243

Pharmaceutical Technology

JUNE 19, 2023

Indian pharmaceutical company Laurus Labs has signed a memorandum of agreement (MoA) with the Indian Institute of Technology, Kanpur (IIT Kanpur) to bring new gene therapy products to the market. Laurus Labs CEO Satyanarayana Chava stated: “This collaboration exhibits our commitment towards the cell and gene therapy (CGT) space.

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Gene Therapy Gene Production Pharma Companies 246

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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Gene Therapy Gene Trials Marketing 264