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.

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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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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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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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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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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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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

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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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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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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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.

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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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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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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

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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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. | It becomes the first FDA-approved gene therapy for Pfizer, which will charge $3.5 million for the single-dose drug.

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Gene Therapy Gene FDA Approval Licensing 128

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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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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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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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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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

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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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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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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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

OCTOBER 25, 2022

Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biotech Taysha demonstrates. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on.

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XTalks

JANUARY 4, 2024

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. There is a significant focus on developing gene therapies as longer-term solutions for the disease.

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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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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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pharmaphorum

FEBRUARY 11, 2022

Data from the first patients enrolled into Regenxbio’s trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the first signs of clinical activity. The post Regenxbio expands MPS I gene therapy trial after first look at data appeared first on.

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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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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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FDA Law Blog

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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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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Bio Pharma Dive

JANUARY 6, 2023

The drugmaker said it is considering licensing out some assets to shorten development timelines, and will stop work on early viral-based gene therapies.

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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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