Drug Discovery World

DECEMBER 19, 2023

The first patient has been dosed with an engineered B cell investigational therapy in a Phase I trial in Mucopolysaccharidosis type I (MPS I). Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication.

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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 company will also have the option to lead late-stage development and commercialisation activities on all products across the world.

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Roots Analysis

NOVEMBER 30, 2021

Bioassay – A Prerequisite for the Cell and Gene Therapy Development. Over the past few years, investigational new drug (IND) filings for cell and gene therapy product have significantly increased. Companies Offering Bioassay Services for Cell and Gene Therapies.

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Pharmaceutical Technology

OCTOBER 13, 2022

Inceptor Bio has partnered with cell engineering technology company Avectas to improve CAR-T cell therapies’ development and manufacturing to treat solid tumours. The process aims to increase the engineered T cells’ quality as well as their durability in the tumour microenvironment.

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Pharmaceutical Technology

AUGUST 11, 2022

GentiBio and Bristol Myers Squibb (BMS) have signed a multi-year partnership agreement for developing new engineered Treg therapies for inflammatory bowel disease (IBD) patients. These therapies will be developed for re-instating immune tolerance and repairing tissue in IBD.

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The Pharma Data

JANUARY 4, 2021

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc.

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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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Pharmaceutical Technology

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Scribe is also entitled to receive potential payments worth over $1bn on meeting development and commercial milestones.

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pharmaphorum

OCTOBER 22, 2020

Sarepta chief commercial officer Bo Cumbo has left to head up gene therapy venture – AavantiBio – with $107 million in backing from his former employer and three high-profile life sciences investors. Unlike a one-shot gene therapy, omaveloxolone would require continuous dosing to maintain its effects.

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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). DEB is a genetic disorder that affects the connective tissue in the skin and nails and results from mutation(s) in the COL7A1 gene.

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Drug Discovery World

SEPTEMBER 6, 2022

In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. ViaCyte, Beam, and Verve are all due to begin gene editing trials this year.

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Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs.

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Pharmaceutical Technology

APRIL 11, 2023

Biotechnology company CTRL Therapeutics has announced $10m in seed financing to boost the development of a next-generation cell therapy platform to treat solid tumours. The platform is being developed using inputs from tumour-infiltrating lymphocyte (TIL) therapies.

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Pharmaceutical Technology

APRIL 20, 2023

Targovax has unveiled plans to rebrand as Circio , reflecting its strategic shift to focus on expediting the development of its innovative circular RNA (circRNA) platform. Circio aims to develop new circRNA medicines initially for cancer, then plans to expand rapidly into vaccines and gene therapy.

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Pharmaceutical Technology

APRIL 14, 2023

The company and the researchers, with Professor Krishanu Saha as principal investigator (PI) and Dr Christian Capitini as co-PI, will develop a GD2 CAR T-cell therapeutic candidate to treat neuroblastoma, a cancer type that generally affects young children. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Scienmag

JUNE 9, 2021

Credit: RCSI Scientists have developed polypeptide-based materials that act as effective vectors for delivering gene therapies. The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.

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STAT News

JULY 11, 2022

Twenty-three years ago, the field of gene therapy was bursting with the promise of breakthrough treatments. Then it was almost instantly derailed by the death of an 18-year-old clinical trial volunteer named Jesse Gelsinger after he received a genetically engineered virus that had been developed to treat his rare liver condition.

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Pharmaceutical Technology

JUNE 12, 2023

Quell Therapeutics and AstraZeneca have entered a collaboration, exclusive option and licence deal for the development, manufacturing and commercialisation of engineered T-regulator (Treg) cell therapies for autoimmune diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Drug Discovery World

APRIL 19, 2024

Autolomous and BioCentriq have collaborated to digitise and accelerate process development and manufacturing activities for cell therapy. Autolomous’ software platform is designed to materially affect the cell and gene therapy sector.

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The Pharma Data

JANUARY 25, 2021

a biotechnology company developing cell and gene therapy treatments for patients suffering from vascular disease, today announced that it raised up to $5.5M that will lead clinical testing of a novel gene therapy for a serious vascular disease in Europe. MIAMI, Jan. Velazquez and Jun Zhao-Liu.

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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. This increases the expression of fetal haemoglobin.

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Drug Discovery World

MARCH 7, 2024

Ginkgo Bioworks has acquired Patch Biosciences, with the intention to strengthen its gene therapy services, cell therapy services, and RNA therapeutics services. The collaboration achieved its goals of enhancing the AAV production titres of Biogen’s gene therapy manufacturing processes.

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Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

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Drug Discovery World

JANUARY 23, 2024

Developed by DDW in partnership with SLAS, the programme will feature leading research from experts in academia, government and industry. The post SLAS2024 update: New tech accelerates drug development appeared first on Drug Discovery World (DDW). See the full programme and register your interest in this event.

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XTalks

MAY 22, 2024

In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology. PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision.

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Drug Discovery World

APRIL 22, 2024

Dr O’Donnell-Tormey will review the various types of therapeutic antibodies in use and in development, she will address the advantages and challenges in using antibodies in therapy, and finally she will discuss the innovations and opportunities in the therapeutic antibody space. Register for free now to secure your place.

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Pharmaceutical Technology

MAY 3, 2023

BrightPath will obtain non-exclusive rights to the STAR-CRISPR editing platform of Artisan Bio for accelerating the development of BrightPath’s iNKT cells. Artisan will receive an upfront payment and research milestone payments, along with licence fees and development milestones from BrightPath.

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The Pharma Data

AUGUST 24, 2020

The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. This requires an 11-day programme which involves extracting blood from an HIV patient and separating their T cells, which are then engineered in a lab to make them immune to HIV.

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Pharmaceutical Technology

AUGUST 26, 2022

ElevateBio has signed a long-term strategic collaboration with the University of Pittsburgh, US, to establish a biomanufacturing centre for expediting cell and gene therapy development. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

MAY 31, 2023

The company is also looking at developing therapies based on individual patient neoantigens in patient human leukocyte antigens (HLAs). Anocca’s platform features libraries of engineered human cells that allow for standardised analysis of T-cell biology, per the company’s website.

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The Pharma Data

OCTOBER 18, 2020

In late August, Pfizer announced it was investing an additional $500 million into its state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina. Ricci told BioSpace the company has been “investing heavily in gene therapy and in the Raleigh-Durham Research Triangle Park area.”

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Pharmaceutical Technology

JANUARY 4, 2023

Cell-engineering company MaxCyte has signed a strategic platform licence (SPL) with biotechnology company Catamaran Bio to support the chimeric antigen receptor (CAR)-NK cell therapy programmes. These CAR-NK cell therapies combine new functional attributes with the NK cells’ inherent cancer-fighting properties.

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Drug Discovery World

MAY 3, 2024

ExoXpert has partnered with Neucore Bio to leverage ExoXpert’s platform for evaluation of advanced exosome loading. Partnering with Neucore will enable ExoXpert to further extend ExoXpert’s expertise in loading exosomes to Neucore’s FiXE platform.”

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Pharmaceutical Technology

DECEMBER 8, 2022

Kite Pharma and Daiichi Sankyo have updated a partnership agreement signed in 2017 for the former’s CAR T-cell therapy, Yescarta (axicabtagene ciloleucel). Under the prior deal, Daiichi Sankyo acquired exclusive rights for the development, manufacturing and commercialisation of Yescarta in Japan. By Cytiva Thematic.

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Pharmaceutical Technology

MARCH 8, 2023

Global science and technology innovator Danaher has entered a strategic collaboration with the University of Pennsylvania for cellular immunotherapies to address manufacturing challenges impacting cell therapy uptake. The multi-year collaboration aims to develop new technologies which will help in improving clinical outcomes for patients.

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Drug Discovery World

JUNE 23, 2023

Advanced Therapy Medicinal Products (ATMPs) have been making the news this week. Evox Therapeutics acquires exosome AAV technology from Codiak Evox Therapeutics has purchased Codiak Biosciences engEx-AAV technology platform, including all intellectual property rights, and exclusive access to certain engineering and manufacturing rights.

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Drug Discovery World

MARCH 20, 2024

The proceeds will be used to advance CPTX2309, Capstan’s lead in vivo chimeric antigen receptor T cell (CAR-T) candidate, to early clinical proof-of-concept in autoimmune disorders, and to further develop Capstan’s tLNP pipeline.

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