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

Pharmaceutical Technology

DECEMBER 8, 2022

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. By Cytiva Thematic.

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Licensing Licensing Gene Therapy Gene 305

Pharmaceutical Technology

OCTOBER 25, 2022

Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy 264

Gene Therapy Gene Development Genetics 264

Bio Pharma Dive

JUNE 8, 2023

Two-year-old Kate Therapeutics is launching publicly with $51 million in funding and a licensing deal with Astellas Pharma for a neuromuscular disease gene therapy.

Gene Therapy 246

Gene Therapy Gene Licensing Licensing 246

Bio Pharma Dive

MAY 15, 2023

The Dutch biotech sold a portion of royalties owed by CSL Behring on sales of Hemgenix, adding to a cash pile it has built since out-licensing the treatment.

Gene Therapy 294

Gene Therapy Gene Licensing Licensing 294

Pharmaceutical Technology

AUGUST 25, 2022

The European Commission (EC) has granted conditional marketing authorisation (CMA) for BioMarin Pharmaceutical ’s gene therapy, Roctavian (valoctocogene roxaparvovec), to treat adults with severe haemophilia A (congenital Factor VIII deficiency). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Clinical Trials Clinical Trials 264

Bio Pharma Dive

JULY 10, 2023

The licensing agreement for the biotech’s viral vector platform follows recent Astellas agreements with Taysha Gene Therapies and Kate Therapeutics.

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Gene Therapy Gene Licensing Licensing 215

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.

Gene Therapy 246

Gene Therapy Gene Production Pharma Companies 246

Bio Pharma Dive

DECEMBER 14, 2021

Fitusiran, which Sanofi licensed from Alnylam, could finally get to market after trial delays and safety concerns slowed its progress. But gene therapies and other new medicines might provide competition.

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Gene Therapy Licensing Licensing Gene 317

Pharmaceutical Technology

JUNE 13, 2023

Beacon Therapeutics has kickstarted its entry into the gene therapy field with a $120m Series A financing. Amongst it was AGTC’s lead clinical candidate, AFTC-501, an adeno-associated virus (AAV) gene therapy for XLRP. Beacon Therapeutics is the third gene therapy company Syncona has launched.

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Gene Therapy Gene Trials Clinical Trials 246

pharmaphorum

APRIL 9, 2024

Great Ormond Street Hospital is applying for the license to a gene therapy for rare disease ADA-SCID that was abandoned by a biotech developer on commercial grounds.

Gene Therapy 119

Gene Therapy Licensing Licensing Gene 119

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.

Gene Therapy 244

Gene Therapy Gene Development Clinical Trials 244

Bio Pharma Dive

OCTOBER 22, 2024

The gene editing company will focus on “in vivo” medicines, while seeking to license out or find a development partner for its clinical-stage treatment reni-cel.

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Gene Editing In-Vivo Licensing Licensing 280

Bio Pharma Dive

MARCH 2, 2022

A federal patent board ruled Broad Institute scientists were first to a key gene editing advance, weakening the patent position of Intellia and CRISPR Therapeutics, which hold licenses through the University of California.

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Gene Editing Gene Licensing Licensing 194

Bio Pharma Dive

MARCH 27, 2023

Vertex will license CRISPR technology to develop insulin-producing islet cells that are more resistant to immune rejection, adding to other efforts directed at Type 1 diabetes.

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Gene Editing Insulin Gene Licensing 281

Pharmaceutical Technology

JUNE 21, 2023

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. per dose, it is the most expensive single-use gene therapy in the US.

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Gene Therapy Gene Protein Sales 246

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. By Cytiva Thematic.

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Gene Therapy Gene Development Engineer 237

pharmaphorum

JANUARY 10, 2023

Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene therapy for inherited neuromuscular disorder Pompe disease. The post Astellas licenses companion drug for Pompe gene therapy appeared first on.

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Gene Therapy Gene Licensing Licensing 119

Pharmaceutical Technology

SEPTEMBER 17, 2024

MaxCyte has entered into a license agreement with Kamau Therapeutics, a clinical-stage company focused on stem cell therapy gene correction.

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Licensing Licensing Gene 130

Pharmaceutical Technology

JUNE 27, 2023

The FDA has accepted a biologics license application (BLA) for Pfizer's fidanacogene elaparvovec to treat adults with haemophilia B.

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Gene Therapy Licensing Licensing Gene 240

Bio Pharma Dive

JANUARY 19, 2023

Shoreline will acquire a preclinical NK cell therapy Editas has been developing, as well as a license to use the CRISPR biotech’s gene editing technology.

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Gene Editing Licensing Licensing Gene 256

Pharmaceutical Technology

DECEMBER 14, 2023

The payment, which encompasses newly approved Casgevy, is the latest deal in a long line of CRISPR licensing twists and turns.

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Gene Editing Licensing Licensing Gene 130

Bio Pharma Dive

JANUARY 31, 2023

For $10 million up front, UniQure has licensed rights to its second experimental treatment for the condition, a medicine from startup Apic Bio that should enter human testing later this year.

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Gene Therapy Gene Licensing Licensing 210

Bio Pharma Dive

JANUARY 14, 2025

Nippon Shinyaku will pay Regenxbio $110 million upfront for U.S. and Asia rights to treatments for Hunter and Hurler syndromes.

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Gene Therapy Licensing Licensing Gene 139

Fierce Pharma

APRIL 26, 2024

Ten years after dipping its toes into the gene therapy pool in a licensing deal with Spark Therapeutics, Pfizer has gained FDA approval for the acquired treatment. | Ten years after dipping its toe into the gene therapy pool in a licensing deal with Spark Therapeutics, Pfizer has gained an FDA approval for the acquired treatment.

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Gene Therapy Gene FDA Approval Licensing 128

Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow.

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Gene Editing Gene Gene Therapy In-Vivo 189

Bio Pharma Dive

AUGUST 4, 2021

The biotech company followed through on an option deal it struck two years ago with Nationwide Children's, licensing an experimental treatment for the most common form of limb-girdle muscular dystrophy.

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Gene Therapy Gene Licensing Licensing 177

Pharmaceutical Technology

MAY 24, 2023

It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene. It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene.

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Gene Therapy Gene Licensing Licensing 130

BioPharma Reporter

OCTOBER 10, 2024

The Japanese big pharma adds the new gene therapy candidate to its pipeline in a US$50 million deal in equity and upfront payments.

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Gene Therapy Gene Licensing Licensing 80

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. By Cytiva Thematic.

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Gene Therapy Gene Development Manufacturing 130

Pharmaceutical Technology

APRIL 13, 2023

ImaginAb will be responsible for licensing and supplying the clinical doses of 89Zr crefmirlimab berdoxam, its investigational CD8 ImmunoPET tracer, to Leucid Bio. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Licensing Licensing Gene Therapy Gene 130

pharmaphorum

JANUARY 2, 2024

Novartis has doubled down on its gene therapy collaboration with Voyager Therapeutics, licensing candidates for Huntington's disease and SMA

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Gene Therapy Gene Licensing Licensing 103

Pharmaceutical Technology

APRIL 13, 2023

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.

Gene Therapy 147

Gene Therapy Gene FDA Approval Licensing 147

BioSpace

SEPTEMBER 13, 2023

AlveoGene has licensed the U.K. Respiratory Gene Therapy Consortium’s InGenuiTy platform for all uses excluding the CTFR gene, which is already licensed to Boehringer Ingelheim for cystic fibrosis.

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Gene Therapy Gene Licensing Licensing 93

Bio Pharma Dive

OCTOBER 4, 2024

The gene editing company is selling to DRI Healthcare Trust future license fees that are owed to it under an agreement with Vertex last year.

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Gene Editing Licensing Licensing Gene 148

BioSpace

OCTOBER 11, 2021

Takeda entered into a licensing pact with Poseida, Spark entered into a licensing agreement with CombiGene AB, while BridgeBio Pharma announced plans for its second annual R&D Day.

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Licensing Licensing Gene Therapy Gene 80

Pharmaceutical Technology

OCTOBER 14, 2022

Pharmaceutical companies Xcell Biosciences (Xcellbio) and aCGT Vector have partnered to expedite the development of cell and gene therapies. The two companies will aim to improve the manufacturing and analytic procedures used to develop personalised cell and gene therapies to treat cancer patients.

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Gene Therapy Gene Development Manufacturing 130