Worldwide Clinical Trials

JUNE 15, 2022

At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Around 40% of clinical holds are for gene therapy programs.

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Pharmaceutical Technology

DECEMBER 7, 2023

Synplogen and Ginkgo Bioworks have signed an MOU to expedite DNA manufacturing and gene therapy platform services in Japan.

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XTalks

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

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pharmaphorum

FEBRUARY 23, 2022

Takeda has forged another alliance as it continues a push into gene therapy, agreeing a deal worth up to $2 billion with Code Biotherapeutics for opt-in rights to four candidates for rare diseases. The post Takeda grows in gene therapies again with $2bn Code Bio deal appeared first on. Last October it signed a $3.6

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pharmaphorum

NOVEMBER 20, 2020

Just over a year after its first phase 3 trial of its Engensis gene therapy for painful diabetic peripheral neuropathy (DPN) bombed, Helixmith has started dosing patients in a new study. The South Korean biotech says the DNA plasmid-based therapy has been administered to a patient at Innovative Research of West Florida.

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pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. “We are committed to understand and mitigate any risk of cancer causation.”

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Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

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Pharmaceutical Technology

FEBRUARY 23, 2023

GenScript ProBio has announced a strategic collaboration with RVAC Medicines to manufacture GMP-grade plasmid DNA (pDNA) for the latter’s RVM-V001, an mRNA Covid-19 vaccine candidate. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.

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XTalks

DECEMBER 14, 2022

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.

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Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. The company’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

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Fierce Pharma

JUNE 3, 2024

subsidiary Bionova Scientific is launching a new business line to provide services around plasmid DNA (pDNA), the companies announced Monday. As part of the pDNA pivot, Bionova is setting up a new facility in The Woodlands, Texas.

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BioPharma Reporter

OCTOBER 20, 2022

Forge Biologics has added plasmid DNA manufacturing to its roster of services, enabling it to vertically integrate its production process.

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Pharma Mirror

FEBRUARY 23, 2021

SOMERSET, N.J.

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Scienmag

MAY 6, 2021

Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Credit: St. Jude Children’s Research Hospital St.

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BioPharma Reporter

JANUARY 17, 2022

Andelyn Biosciences says the FDA's acceptance of its GMP plasmid DNA drug master file (DMF) enables the CDMO to vertically integrate its clientsâ manufacturing process, condensing timelines for developers to begin manufacturing to just three months.

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pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

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pharmaphorum

APRIL 27, 2021

Dosing can start once again in uniQure’s phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by the treatment. . The post FDA lifts hold on uniQure gene therapy after cancer case review appeared first on.

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Gene Therapy Gene Genome Genomics 52

Cloudbyz

JULY 31, 2024

This revolution is enabling the growth of innovative biomarker-based precision medicine and cell and gene therapy, transforming both clinical research and post-market care. These therapies involve modifying a patient’s cells or genes to treat or prevent diseases, with the promise of long-lasting effects.

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Pharmaceutical Technology

MAY 17, 2023

In addition, the AAV and LVV platforms feature in-network plasmid DNA (pDNA) supplies, in-house capability for major testing and the use of cell factories and fixed bed bioreactor for adherent protocols along with wave and stirrer bioreactors for suspension. Topic sponsors are not involved in the creation of editorial content.

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Scienmag

FEBRUARY 11, 2021

Study in The CRISPR Journal reports the app advances CRISPR gene therapy R&D; 18-year-old software savant helped lead the app’s development Wilmington, DE, Feb. The app reveals potentially risky DNA […].

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Gene Gene Therapy DNA Scientist 73

Scienmag

FEBRUARY 23, 2021

CRISPR gene editing has transformed research, but it is not perfect, and can sometimes target unintended genes; to watch CRISPR enzymes respond to different genes, Leipzig University researchers developed a new method using DNA origami and were able to me Credit: Image courtesy of Julene Madariaga Marcos.

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The Pharma Data

JANUARY 18, 2021

Use of AAVnerGene capsid library provided to Neurophth for ophthalmic gene therapy. Next Generation AAVs enhance gene therapies by increasing transduction efficiency and specificity while reducing immune responses and cost. gene therapy company (hereafter Neurophth), and AAVnerGene Inc. ,

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XTalks

DECEMBER 27, 2022

Adstiladrin is an adenoviral vector-based gene therapy that is non-replicating, so it cannot multiply in human cells. XTALKS WEBINAR: Importance of Early Integrated Evidence Strategy for Cell & Gene Therapies. How Does the Gene Therapy Adstiladrin Work?

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Drug Discovery Today

FEBRUARY 24, 2022

Global cell and gene therapy development and manufacturing business expands into Bruntwood SciTech’s Alderley Park to boost manufacturing capacity.The 17,000 sq ft high-specification lab space is currently under construction, expanding plasmid DNA and viral vector capabilities in Europe.The facility will support therapeutic developers with rapid access (..)

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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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The Pharma Data

DECEMBER 11, 2020

11, 2020 — Results from a long-term study of a gene therapy technique to prevent inherited mitochondrial disease show promise, researchers say. The researchers said the technique could break the cycle of disease passed from mother to baby through mutations in mitochondrial DNA (mtDNA). FRIDAY, Dec.

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pharmaphorum

NOVEMBER 22, 2021

This forum will specifically focus on targeting mitochondrial dysfunction to develop therapies for chronic diseases caused by mitochondrial DNA mutation/nuclear DNA mutations, muscle dystrophy diseases/muscle loss, metabolic disorders, neurodegenerative disease, and conditions linked to oxidative damage.

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XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. BacTRL Gene Therapy Platform. Related: Red Meat Allergy Test Gets FDA Clearance. “We

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Roots Analysis

JUNE 1, 2022

DNA cloning is the field of synthetic biology which can be helpful to understand the effect of mutation on a particular gene. Many diseases that can be treated using DNA cloning, such as leukemia and sickle cell anemia, involving the replacement of defected gene. DNA Cloning Kit Providers – Current Market Landscape.

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The Pharma Data

JANUARY 27, 2021

(Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. FTD is a debilitating form of early onset dementia that currently has no approved disease-modifying therapies.

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Advarra

JUNE 12, 2023

Gene therapy research is exciting and full of promise, but because of the risks involved, it’s also highly regulated, requiring an institutional biosafety committee (IBC) to provide additional oversight and risk assessment. What Does an IBC Review?

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Delveinsight

DECEMBER 17, 2020

Following that hypothesis, scientists designed oral inhibitors, which could target mitochondrial DNA (mtDNA). Instead of directly targeting existing mitochondria, the team went after mitochondrial DNA transcription that is vital to the formation of new mitochondria and the production of energy.

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XTalks

JANUARY 21, 2025

The Shield test provides a non-invasive approach to colorectal cancer screening by analyzing blood for key DNA changes linked to cancer. There, plasma is separated, and tumor DNA is identified by detecting genetic mutations, methylation patterns and fragmentation signals. The Onclarity HPV reagent pack extraction combo.

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BioPharma Reporter

MAY 18, 2021

Charles River Laboratories International, Inc is to acquire US based Vigene Biosciences, a cell and gene therapy (CGT) contract development and manufacturing organization (CDMO) providing viral vector-based gene delivery solutions for research and clinical applications.

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BioTech 365

AUGUST 17, 2020

– Patent Application Relates to Production of Adeno-Associated Virus (AAV) via Use of LinearDNA™ Amplicons; AAV Viral Vectors Used in Gene and Redirected Cell Therapies; Current Production of AAV Relies Heavily on Use of Plasmid-derived DNA – STONY BROOK, N.Y.–(BUSINESS

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pharmaphorum

OCTOBER 3, 2022

A gene therapy, it permits the body to read over the DNA mutation and still continue to produce dystrophin. Last week, Sarepta Therapeutics filed for accelerated FDA approval of its DMD gene therapy developed with Roche, SRP-9001 (delandistrogene moxeparvovec). Eventually, they will need assistance with breathing.

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