STAT

JULY 24, 2023

The other, taken after months of gene therapy  given through eyedrops, revealed no scarring on either eye. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. One showed cloudy scars covering both eyeballs.

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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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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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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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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 Companies Pharmaceutical Companies 98

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). This would allow the companies to boost the development of new therapy options for Rett syndrome and GAN patients.

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European Pharmaceutical Review

MAY 24, 2023

The US Food and Drug Administration (FDA) has approved the first-ever redosable gene therapy for the rare skin disease dystrophic epidermolysis bullosa (DEB). The gene therapy is a topical gel that restores functional copies of the COL7A1 gene to provide wound healing and sustained functional COL7 protein expression with redosing.

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FDA 101

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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STAT

MAY 19, 2023

The Food and Drug Administration on Friday approved the first treatment for a devastating condition that causes the skin to be so fragile that even a touch can cause it to splinter, bringing another gene therapy onto the market. Scarring can cause fingers and toes to fuse together, and patients face high rates of skin cancer.

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FDA 103

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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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

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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FDA 96

Pharmaceutical Technology

APRIL 14, 2023

Twist Bioscience has introduced Twist T-cell receptor (TCR) and Twist chimeric antigen receptor (CAR) libraries to facilitate the development of cell therapies. Researchers will now be able to speed up the creation of tailor-made libraries that can help in identifying and developing new cell therapies.

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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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Hospital Pharmacy Europe

JUNE 12, 2023

Dystrophic epidermolysis bullosa is caused by a gene mutation, and the recent FDA approval of the corrective gene therapy VYJUVEK represents the first specific treatment to become available. The FDA approval of VYJUVEK – also known as beremagene geperpavec or B-VEC – provides the first novel topical gene therapy.

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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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Pharmaceutical Technology

NOVEMBER 16, 2022

ElevateBio has entered a partnership with Affini-T Therapeutics to progress the latter’s engineered TCR-T therapies focused on Kirsten rat sarcoma viral oncogene homolog (KRAS), a dominant oncogenic driver mutation in solid tumours. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Immunization 98

The FDA Law Blog

MARCH 13, 2023

To further expand patient input into product development, the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products or “OTP” (formerly the Office of Tissues and Advanced Therapies, or “OTAT”) is holding a free public workshop titled, Clinical Trials: The Patient Experience.

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FDA Immunization 98

Pharmaceutical Technology

APRIL 28, 2023

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. g/dL and 45.4%

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FDA 105

pharmaphorum

NOVEMBER 15, 2022

The day will continue with other hot topics for pharma, including health equity, cell and gene therapy, oncology, and pharmacy model disruption. Then we’ll hear from Aptar Digital Health’s Sai Shankar and Pierre Laurent in a session all about patients as partners in healthcare.

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Pharmaceutical Technology

MAY 22, 2023

Vyjuvek is a non-invasive, topical, re-dosable gene therapy that delivers functional human COL7A1 gene copies to offer wound healing. 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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FDA 52

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. Free Whitepaper.

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Communication 95

Pharmaceutical Technology

MARCH 28, 2023

Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). The gene-editing technology allows for precise, directed changes to genomic DNA.

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Communication 94

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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Communication 105

European Pharmaceutical Review

OCTOBER 5, 2023

Ahead of her presentation on the future of biomanufacturing at CPHI Barcelona, Sheila Ann Mikhail, the co-founder and former CEO of AskBio, engages in a conversation about gene therapy, artificial intelligence, and the importance of being a well-informed innovator when selecting a manufacturing partner. at Conference Centre 4.

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Immunization 91

Pharmaceutical Technology

MARCH 29, 2023

Regeneron Pharmaceuticals has collaborated with Sonoma Biotherapeutics to discover, develop and commercialise new regulatory T cell (Treg) therapies for autoimmune diseases. The two companies will together research and develop T cell therapies for Crohn’s disease and ulcerative colitis, as well as two other undisclosed indications.

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Pharmaceutical Technology

JUNE 8, 2023

DTx-1252 is a fatty acid ligand conjugated oligonucleotides (FALCON) small interfering RNA (siRNA) therapeutic which represses the PMP22 gene in Schwann cells. 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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FDA 98

Pharmaceutical Technology

APRIL 20, 2023

Circio aims to develop new circRNA medicines initially for cancer, then plans to expand rapidly into vaccines and gene therapy. 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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Vaccines 96

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 98

Pharmaceutical Technology

APRIL 12, 2023

Clinical-stage biotechnology firm LyGenesis has formed a joint research collaboration with Imagine Pharma to develop new cell therapies for type 1 diabetes (T1D). 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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pharmaphorum

FEBRUARY 23, 2022

As investment in gene therapies continues to grow rapidly, more effectively engaging patients throughout development has become a priority for any company serious about building safer and more meaningful gene therapy programs. The post 2nd Annual Gene Therapy Patient Engagement Summit appeared first on.

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FDA 52

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 111

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. and Hbs dropped to 33.2%.

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FDA 98

European Pharmaceutical Review

JULY 20, 2023

As proven by recent regulatory approvals sweeping the cell and gene therapy industry, particularly within Europe and the US, these pioneering treatments have demonstrated great capacity in helping to resolve hard-to-treat diseases. Batty discussed some of the capabilities of haemophilia gene therapies in 2023.

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FDA 64

Pharmaceutical Technology

JUNE 9, 2023

KT430 is a preclinical, next-generation investigational gene therapy designed for the treatment of X-linked myotubular myopathy (XLMTM), a rare and life-threatening neuromuscular disease. It delivers a functional copy of the MTM1 gene through a new MyoAAV capsid to treat XLMTM.

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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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FDA 52

Pharmaceutical Technology

MAY 19, 2023

Myeloid Therapeutics has raised $73m to support the continued clinical development of its lead cell therapy programme, MT-101, in Phase I/II trials for T cell lymphoma. 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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Labelling 98