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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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 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

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

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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XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). It has been modified so that it cannot replicate in normal cells.

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Gene Therapy Gene FDA Approval Dermatology 97

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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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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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

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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Fierce Pharma

NOVEMBER 6, 2023

The drug launched after an FDA approval in May 2023.

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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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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

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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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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XTalks

MAY 31, 2023

In this episode, Ayesha talked about the FDA approval of the first topical gene therapy for the treatment of a rare skin condition. It’s the first treatment for the rare disease. The drug, called Miebo, is also the first treatment that targets tear evaporation, which is a leading cause of dry eye disease.

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Drug Discovery World

DECEMBER 19, 2022

Researchers have found that a topical gene therapy gel helps heal wounds in patients with life-threatening blistering skin disease epidermolysis bullosa (EB). . The post ‘Life-changing’ topical gene therapy gel heals EB wounds appeared first on Drug Discovery World (DDW).

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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. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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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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

JUNE 8, 2023

GenScript ProBio has collaborated with the New York Blood Center Enterprises’ business unit, called Comprehensive Cell Solutions (CCS), to accelerate the development and manufacture of cell and gene therapies. The core objective of the collaboration is to make the therapies affordable and accessible to patients.

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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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XTalks

JANUARY 2, 2024

Chiesi Global Rare Diseases, a specialized division within the Chiesi Group dedicated to pioneering therapies for individuals affected by rare diseases, has received approval from the US Food and Drug Administration (FDA) for Filsuvez (birch triterpenes) topical gel.

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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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Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Background: The Advancement of Cell and Gene Therapies.

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Drug Discovery World

FEBRUARY 8, 2024

In 2023, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers. How is the titer determined?

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Drug Discovery World

JULY 27, 2023

A dystrophic epidermolysis bullosa (DEB) patient at the Bascom Palmer Eye Institute, University of Miami, US, has regained his vision after being the first person to receive a formulation of gene therapy Vyjuvek suitable for the eyes. This is a platform that can potentially treat diseases that have a genetic component.”

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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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Drug Discovery World

OCTOBER 3, 2023

Earlier this year, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

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FDA Law Blog

MARCH 13, 2023

This meeting will host a panel of rare disease patients, caregivers, and advocates to have a focused discussion on the emerging field of gene therapy (regulated at the FDA by OTP). At least some (if not all) of the panelists will have had experience participating in gene therapy trials.

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Drug Discovery World

JULY 3, 2023

Cytiva and BioCentriq, a New Jersey-based cell and gene therapy contract development and manufacturing organisation (CDMO), have been awarded funding from the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), a public-private partnership whose mission is to accelerate biopharmaceutical innovation.

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Drug Discovery World

MAY 29, 2023

The US Food and Drug Administration has approved Krystal Biotech’s Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of dystrophic epidermolysis bullosa (DEB). Vyjuvek is mixed into an excipient (non-active ingredient) gel prior to topical application.

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Drug Discovery World

AUGUST 29, 2023

Join DDW for this free event, ‘Discover how iPSC derived cells can be used to support the development of drug and gene therapies’, which is supported by DefiniGEN. The webinar will cover topics including functionality, modelling disease phenotypes, therapeutic modalities and delivery and efficacy.

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Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Roots Analysis

AUGUST 29, 2023

Over the years, cell and gene therapies (CGT) and other advanced therapy medicinal products (ATMPs) have managed to capture the interest of both drug developers and healthcare investors. It is worth highlighting that some therapeutic interventions can be considered both cell and gene therapies.

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