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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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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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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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

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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Bio Pharma Dive

DECEMBER 2, 2020

The pharma licensed a treatment for a severe type of age-related vision loss, building on past deals that gave it a foothold in gene therapy.

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Pharmaceutical Technology

NOVEMBER 9, 2022

On November 2, the Institute for Clinical and Economic Review (ICER) released its updated evidence aimed at measuring the clinical effectiveness and cost of the two haemophilia gene therapies. Known by the brand name Roctavian, BioMarin’s haemophilia A therapy valoctocogene roxaparvovec could be fairly priced in the range of $1.95–1.96

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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.

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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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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. The therapy has also received orphan drug and fast-track designations. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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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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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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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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.

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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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XTalks

JANUARY 3, 2024

Novartis and Voyager Therapeutics have struck a licensing deal that will see Novartis pay Voyager $100 million upfront to develop gene therapy candidates targeting Huntington’s disease (HD) and spinal muscular atrophy (SMA). The total value of the deal could potentially reach $1.2 Sandrock, Jr., MD, PhD, CEO of Voyager.

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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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Fierce Pharma

SEPTEMBER 14, 2023

Otsuka teamed up with Shape Therapeutics in an eye disease gene therapy deal potentially worth $1.5 Otsuka teamed up with Shape Therapeutics in an eye disease gene therapy deal potentially worth $1.5 Insilico Medicine out-licensed an AI-discovered cancer drug candidate to Exelixis.

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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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BioPharma Reporter

APRIL 24, 2023

bluebird bio has announced the submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for lovotibeglogene autotemcel (lovo-cel) gene therapy in patients with sickle cell disease (SCD).

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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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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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BioSpace

JUNE 8, 2023

Astellas Pharma will license and further develop a gene therapy from Kate Therapeutics aimed at addressing XLMTM amid safety concerns about its own experimental XLMTM treatment.

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Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT).

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pharmaphorum

DECEMBER 22, 2021

Gyroscope Therapeutics and its gene therapy for the sight-robbing disease geographic atrophy (GA) has been acquired by a big pharma company – but likely not the one you would expect. Last year, it also signed a licensing deal with Dyno Therapeutics for its AAV capsid-based gene delivery technology focused on ocular diseases.

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pharmaphorum

APRIL 22, 2022

Astellas has said it will book a $170 million impairment charge in its fourth quarter results as a result of a decision to halt the development of three gene therapy candidates for Duchenne muscular dystrophy in preclinical development. The post Astellas takes $170m charge as it drops DMD gene therapies appeared first on.

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XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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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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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. Topic sponsors are not involved in the creation of editorial content.

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BioSpace

MARCH 11, 2021

The European Medicines Agency has launched a safety review of bluebird bio’s thalassaemia drug Zynteglo, a conditionally licensed gene therapy in Europe.

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BioTech 365

JANUARY 19, 2022

Eikonoklastes Bolsters Pipeline, Announces a License for Novel Gene Therapy to Address Neurodegenerative Diseases Eikonoklastes Bolsters Pipeline, Announces a License for Novel Gene Therapy to Address Neurodegenerative Diseases Biopharmaceutical company to develop therapies to address ALS, Alzheimer’s, Parkinson’s and others … Continue reading (..)

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The Pharma Data

MARCH 9, 2022

Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets.

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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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BioTech 365

MAY 6, 2021

Genprex In-Licenses Additional Gene Therapy Technologies for Treatment of Lung Cancer Genprex In-Licenses Additional Gene Therapy Technologies for Treatment of Lung Cancer Amendment to existing worldwide, exclusive license agreement expands Genprex’s oncology franchise Newly licensed technologies include use of Genprex’s … Continue reading → (..)

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Drug Discovery World

OCTOBER 4, 2023

The US Food and Drug Administration (FDA) has accepted Rocket Pharmaceuticals’ Biologics License Application (BLA) and granted Priority Review for RP-L201 (marnetegragene autotemcel). The post FDA acceptance advances investigational gene therapy appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JANUARY 5, 2023

CANbridge Pharmaceuticals has exercised its option to secure the exclusive global rights to develop, manufacture and commercialise a novel gene therapy to treat spinal muscular atrophy (SMA) from UMass Chan Medical School. . The benchmark therapy uses a vector similar to that used in the only gene therapy approved for SMA.

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pharmaphorum

SEPTEMBER 13, 2021

AbbVie has expanded its ophthalmology pipeline via a licensing deal with Regenxbio, paying $370 million upfront to get its hands on a gene therapy for wet age-related macular degeneration (AMD) and other eye diseases. The post AbbVie spies potential in Regenxbio eye disease gene therapy appeared first on.

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