Pharmaceutical Technology

APRIL 24, 2023

In 1974, Dr. Robert Langer was part of the first cohort of researchers to begin nanoparticle research , as his lab at Cornell University developed tiny particles to deliver large molecules for angiogenesis. The next wave came from early research into liposomes and lipid nanoparticles, both of which are used for drug delivery.

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Drug Delivery Vaccine Vaccination Research 147

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. Under the deal, both companies will partner on the research and preclinical studies, which will be funded by Moderna.

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Gene Editing Gene In-Vivo Gene Therapy 147

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. Beyond the liver, ReCode’s SORT LNP genetic medicines technology enables the delivery to target cells and organs. By Cytiva Thematic.

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Gene Editing Gene Gene Therapy Genetics 130

Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences has entered a strategic research collaboration with Phlox Therapeutics to expedite the development of new therapies for rare cardiac diseases. Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM).

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RNA Gene Therapy Genetics Genetic Disease 130

Pharmaceutical Technology

MARCH 1, 2023

Tevard Biosciences and Vertex Pharmaceuticals have announced a four-year global research partnership to develop new tRNA-based therapies to treat Duchenne muscular dystrophy (DMD) patients. The deal offers access to the platforms of Tevard to discover and develop tRNA-based therapies.

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Development Gene Therapy Genetics Genetic Disease 130

Delveinsight

FEBRUARY 11, 2021

Verily is partnering with Johnson & Johnson’s Janssen division to witness the body’s earliest immune responses to a coronavirus infection, with people participating in the research from within their homes. . Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease.

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Vaccine Vaccination Gene Therapy RNA 98

XTalks

FEBRUARY 25, 2025

CTX is a progressive multisystemic disorder that significantly impacts patients and previously lacked approved treatments, stated Dr. Janet Maynard, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine at the FDAs Center for Drug Evaluation and Research in the FDA news release.

FDA Approval 59

FDA Approval Gene Therapy Gene Genetics 59

pharmaphorum

AUGUST 12, 2020

The enzyme replacement therapy (ERT) – also known as PRX-102 – has been granted a priority review by the US regulator, and is the top prospect in Chiesi’s recently formed rare diseases division. Market research firm Optima Insights has predicted that sales of Fabry disease drugs will more than double from around $1.8

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Drugs Genetic Disease Gene Therapy Trials 98

The Pharma Data

JANUARY 9, 2021

(NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Scienmag

JANUARY 13, 2021

Study demonstrates the efficacy and therapeutic potential of select ionizable lipid nanoparticles for delivering mRNA to treat genetic diseases before birth Credit: Children’s Hospital of Philadelphia Philadelphia, January 13, 2021–Researchers at Children’s Hospital of Philadelphia and the School of Engineering and Applied Science (..)

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Genetic Disease Research Engineer Genetics 40

pharmaphorum

OCTOBER 4, 2022

US-based LogicBio is a clinical-stage genetic medicine company focused on genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. As per the agreement, Alexion will initiate a cash tender offer through a subsidiary to acquire all outstanding shares of LogicBio for $2.07

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Gene Editing Gene Genome Genomics 64

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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Gene Therapy Gene Gene Editing Genetics 238

The Pharma Data

JULY 6, 2021

Data will include the final analysis from the phase IIIb STASEY study of Hemlibra® (emicizumab) and updated data from the phase I/II study of SPK-8011, an AAV-based gene therapy in development by Spark Therapeutics (a member of the Roche Group). Roche’s Chief Medical Officer and Head of Global Product Development.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

pharmaphorum

AUGUST 24, 2021

It seems likely that the type 1 diabetes programme will be focused on developing cells that can secrete insulin in response to blood glucose levels – acting as a replacement for pancreatic beta cells that are destroyed in the autoimmune disease – which is already a focus of Vertex’ research.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine.

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Marketing Gene Sequencing DNA Genome 52

pharmaphorum

DECEMBER 6, 2020

The trials are the first to test a CRISPR/Cas9 gene editing therapy in humans for a genetic disease, according to the partners. What we have been able to do through this study is a tremendous achievement,” said investigator Haydar Frangoul of the Sarah Cannon Research Institute, who presented the results at ASH. “By

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

XTalks

NOVEMBER 10, 2023

Related: Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio The FDA approval of Adzynma was based on its demonstrated safety and efficacy in a Phase III global cross-over study, the first of its kind for cTTP.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

The Pharma Data

OCTOBER 27, 2020

Former Sarepta Therapeutics executive Bo Cumbo left to launch his new gene therapy company, AavantiBio, with a $107 million Series A. AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. Source link.

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Gene Gene Therapy Medicine Development 52

XTalks

AUGUST 7, 2024

In this episode, Ayesha spoke with Lawreen Asuncion, a patient advocate who works to raise awareness for the rare disease Usher syndrome. She began her career as a research scientist, working for more than eight years in this role before transitioning into product management and marketing.

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Life Science Scientist Production Research 52

The Pharma Data

DECEMBER 13, 2020

Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies. Dr. Zhang completed his postdoctoral fellowship at Harvard Medical School/Boston Children’s Hospital.

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Business Development Development Gene Editing In-Vivo 40

The Pharma Data

NOVEMBER 19, 2020

He has pioneered the establishment of diagnostic evaluation algorithms for children with sensorineural hearing loss and developed a next generation sequencing platform to determine the genetic causes of hearing loss in children. He has 92 peer review articles published with the majority related to hearing loss. www.sensorion-pharma.com.

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Genetics Containment Genetic Disease Gene 52

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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Gene Therapy Gene Trials Clinical Trials 95

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. Here are four life sciences trends to look out for in 2023.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

XTalks

APRIL 1, 2025

However, in 2018, the companies concluded the research and option phase of their partnership. Among these are three gene therapies. Alnylam took the reigns on Amvuttra and Sanofi assumed full ownership of Qfitlia. Both companies met with some setbacks during Qfitlias development, including clinical holds in 2017 and 2020.

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FDA Approval RNA Clinical Trials Clinical Trials 59

XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S. “They called him ‘The Boy.’ Who wasn’t supposed to sit up.

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Development Gene Therapy Clinical Trials Clinical Trials 98

STAT News

FEBRUARY 22, 2023

The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham.

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Gene Editing Gene Genetic Disease Research 126

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”.

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DNA Gene Gene Silencing Genome 98

The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. This route of administration has the potential to open up access for older patients to all the benefits of gene therapy.

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Clinical Trials Clinical Trials Gene Therapy Gene 52

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

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Clinical Development Gene Therapy Development Gene 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. The research was published earlier this month in the journal Cell. pyogenes dCas9.

DNA 52

DNA Gene Gene Silencing Genome 52

pharmaphorum

JANUARY 27, 2023

In the morning, I can be talking about gene therapy for hearing loss, for example, [and] in the afternoon, I can be talking about the treatment of a very specific tumour. Appropriate site selection [is] really important to engage with enthusiastic investigators that want to be involved in pioneering research and innovations.”

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Clinical Trials Clinical Trials Trials Development 111

The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Additionally, Parag V.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

Delveinsight

JANUARY 12, 2021

Bluebird spins off to two companies, cleaving off its gene therapy, and cancer units. Bluebird bio recently announced its plans to split its genetic disease and oncology businesses. The company has decided to prioritize its severe genetic disease unit and form its oncology business into a new company.

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Licensing Licensing Antibody Gene Therapy 52

XTalks

FEBRUARY 26, 2025

Regenerons CHORD study evaluating investigational gene therapy DB-OTO for otoferlin-related hearing loss a rare condition caused by variants in the OTOF gene has revealed that 10 of 11 children with at least one post-treatment hearing assessment experienced notable improvements, including dramatic gains in speech perception.

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Gene Therapy Gene Clinical Trials Clinical Trials 87

XTalks

JANUARY 6, 2025

Whether its the integration of nanotechnology in medicine, the evolution of point-of-care (POC) diagnostics or the transformative impact of CRISPR and regenerative medicine, these biotech trends are pushing scientific boundaries and creating new opportunities for businesses and researchers alike. billion in 2021, is projected to reach $42.56

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