Pfizer receives FDA approval for haemophilia B gene therapy
Pharmaceutical Technology
APRIL 29, 2024
Pfizer has received approval from the US Food and Drug Administration (FDA) for its one-time gene therapy BEQVEZ for haemophilia B.
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AskBio’s AB-1002 gene therapy receives FDA fast-track status
Pharmaceutical Technology
APRIL 19, 2024
The US Food and Drug Administration (FDA) has granted fast-track designation to Bayer subsidiary Asklepios BioPharmaceutical’s (AskBio) investigational gene therapy AB-1002, aimed at treating congestive heart failure (CHF).
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Delivery tech remains challenging with gene therapies for brain disorders
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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CRISPR gene therapy Casgevy secures approval in Europe
Pharmaceutical Technology
FEBRUARY 13, 2024
The regulatory greenlight makes it the only gene therapy available in Europe for sickle cell disease and TDT.
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Genevoyager opens CDMO facility for gene therapy development
Pharmaceutical Technology
DECEMBER 21, 2023
Genevoyager has announced the opening of contract development and manufacturing organisation facility to manufacture gene therapy products.
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FDA fast tracks Solid Biosciences’s Duchenne gene therapy
Pharmaceutical Technology
DECEMBER 7, 2023
The FDA has granted fast track designation to Solid Biosciences’s gene therapy SGT-003 for the treatment of DMD.
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“Lack of harmonisation in Europe” hindering gene therapy growth
Pharmaceutical Technology
APRIL 18, 2024
The future of cell and gene therapy access in Europe could be more precarious than across the pond.
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Forge Biologics and Labcorp partner for gene therapies development
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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Magazine: CRISPR gene therapies cut through in 2023
Pharmaceutical Technology
FEBRUARY 2, 2023
If things go as per plan, in a few months, the US Food and Drug Administration (FDA) will deliberate on the first-of-its-kind CRISPR-based gene therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia. You can also subscribe here to receive email notifications when a new issue is available.
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Spark Therapeutics partners with SpliceBio for gene therapy
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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Avrobio to sell cystinosis gene therapy to Novartis for $87.5m
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. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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First gene therapies for sickle cell disease secure FDA approval
Pharmaceutical Technology
DECEMBER 11, 2023
The FDA has approved Vertex/CRISPR’s Casgevy and bluebird bio’s Lyfgenia gene therapies for the red blood cell disorder.
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Astellas to support development of Taysha’s gene therapy programmes
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.
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Lilly agrees to acquire gene therapy developer Akouos for $610m
Pharmaceutical Technology
OCTOBER 19, 2022
Akouos focuses on developing adeno-associated viral gene therapies to treat inner ear conditions, including sensorineural hearing loss. It has combined expertise in otology, gene therapy and inner ear drug delivery to meet the requirements of individuals with disabling hearing loss globally.
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Beacon Therapeutics enters gene therapy arena with $120m launch
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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Research consortium picks eight rare diseases to advance gene therapies
Pharmaceutical Technology
MAY 16, 2023
The Foundation for the National Institutes of Health (FNIH) has announced its plans to prioritise eight rare diseases to provide industry standards for manufacturing, preclinical testing and product analytical testing for gene therapy development. This will include pairing up indications with manufacturers amongst the BGTC’s partners.
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Laurus Labs and IIT Kanpur partner for new gene therapy products
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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Sartorius upscales gene therapy prowess with $2.6bn Polyplus acquisition
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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Alexion to acquire Pfizer’s rare disease gene therapies for $1bn
Pharmaceutical Technology
JULY 31, 2023
Alexion has signed a definitive agreement for acquiring and licencing Pfizer’s early-stage gene therapy portfolio for rare diseases.
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Amplo Biotechnology receives NIH-NIAMS grant for gene therapy AMP-201
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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Generation bio cuts 40% of staff in blow for non-viral gene therapy sector
Pharmaceutical Technology
NOVEMBER 30, 2023
The move is a strategic response amid challenges faced by the non-viral gene therapy sector, with several startups closing this year.
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Forge Biologics joins BGTC for new AAV gene therapies development
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. Topic sponsors are not involved in the creation of editorial content.
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Delivery and manufacturing hurdles remain in the cell and gene therapy
Pharmaceutical Technology
MARCH 21, 2024
Panellists discuss opportunities and challenges in cell and gene therapies at the Advanced Therapies conference in London, UK.
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Terumo and GenCure sign agreement for cell and gene therapies
Pharmaceutical Technology
JUNE 29, 2022
Terumo Blood and Cell Technologies and BioBridge Global subsidiary, GenCure, have entered a new partnership agreement to expand and integrate cell and gene therapy manufacturing solutions. GenCure will be able to offer a comprehensive solution for cell and gene therapy developers using the reference site.
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Cell and gene therapy: Global Innovation and Opportunity
Drug Discovery World
MAY 2, 2024
Hailed as a revolution in the treatment of many diseases, cell and gene therapy (CGT) is the fastest growing area of therapeutics. 2023 was a record year for CGTs, with seven new CGTs approved by the US Food and Drug Administration (FDA) across a variety of different disease areas.
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Capsida Biotherapeutics and Prevail to develop CNS gene therapies
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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New facility boosts UK’s cell and gene therapy manufacturing capacity
Pharmaceutical Technology
MARCH 10, 2023
On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UK’s ability to develop and manufacture cell and gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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Krystal Biotech receives FDA approval for DEB topical gene therapy
Pharmaceutical Technology
MAY 26, 2023
Krystal Biotech has received approval from the US Food and Drug Administration for topical gene therapy VYJUVEK to treat dystrophic epidermolysis bullosa (DEB) in adults and in children aged six months and above. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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Can gene therapies for haemophilia defend their high price tags?
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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FDA will not meet ahead of bluebird bio’s sickle cell disease gene therapy PDUFA date
Pharmaceutical Technology
AUGUST 16, 2023
Bluebird bio eyes hattrick of gene therapy approvals, with a PDUFA date for sickle cell disease therapy lovo-cel set in December.
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Vivet wins $5.3m grant to develop gene therapy for rare metabolic disorder
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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ElevateBio raises funds to advance cell and gene therapies
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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EC grants approval for BioMarin’s gene therapy to treat Hemophilia A
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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Kate Therapeutics and Capsida Biotherapeutics collaborate on gene therapies
Pharma Times
OCTOBER 4, 2023
The companies aim to bring gene therapies to patients sooner than traditional approaches - News - PharmaTimes
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NAYA buys gene therapy company Florida Biotech for $20m
Pharmaceutical Technology
JANUARY 24, 2024
NAYA’s acquisition includes Florida Biotechnologies’ AAV gene therapy for the treatment of a rare genetic disorder.
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4DMT’s CF gene therapy posts early cues for improving patient outcomes
Pharmaceutical Technology
JUNE 8, 2023
The use of 4D Molecular Therapeutics’ (4DMT) aerosolised gene therapy 4D-710 has improved the quality-of-life and spirometry-measured outcomes in three cystic fibrosis patients , based on early results from a Phase I/II study presented at this year’s annual meeting of the European Cystic Fibrosis Society (ECFS).
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FDA grants RMAT designation to Taysha’s Rett syndrome gene therapy
Pharmaceutical Technology
MAY 3, 2024
The FDA has awarded the designation following a review of initial safety and efficacy data from two Phase I/II clinical trials.
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Otsuka and ShapeTX to develop gene therapies for eye diseases
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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Editas continues to trend efficacy signals with sickle cell disease gene therapy
Pharmaceutical Technology
MAY 12, 2023
Editas Medicine will release new efficacy and safety results of its gene therapy EDIT-301 in severe sickle disease as part of an oral presentation at the European Hematology Association’s (EHA) Hybrid Congress. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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Rocket Pharmaceuticals’ gene therapy receives FDA RMAT designation
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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Gene therapy could reduce seizures in childhood epilepsy
Drug Discovery World
DECEMBER 18, 2023
University College London (UCL) researchers have developed a new gene therapy to cure focal cortical dysplasia, which a new study shows can significantly reduce seizures in mice. They then injected either the virus carrying the EKC gene or a control virus into the affected brain area.
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Rentschler Biopharma and Ikarovec partner to expedite new gene therapies
Pharmaceutical Technology
JUNE 2, 2023
Rentschler Biopharma has collaborated with Ikarovec for expediting the new gene therapies to treat ophthalmic disease. The company is developing new and differentiated gene therapies for the treatment of serious, but common eye diseases. Topic sponsors are not involved in the creation of editorial content.
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Forge receives QP declaration to manufacture AAV gene therapies in Europe
Pharmaceutical Technology
APRIL 25, 2023
Forge Biologics has received a qualified person (QP) declaration to manufacture adeno-associated virus (AAV) gene therapies to support European clinical programmes. The FBX-101 is an AAV serotype rh10 gene therapy, given intravenously following haematopoietic stem cell transplantation infusion.
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Lexeo wins fast track designation for Friedreich’s ataxia gene therapy
Pharmaceutical Technology
APRIL 17, 2024
LX2006 consists of adeno-associated virus (AAV) vector carrying the frataxin gene, responsible for causing Friedreich’s ataxia.
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