BioPharma Reporter

SEPTEMBER 3, 2020

AskBio, which develops adeno-associated virus (AAV) gene therapies for genetic disorders, has won an R&D grant valued at Â2m (around US$2.7m) from Scottish Enterprise.

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Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. It has been estimated that by 2025 the FDA will be approving 10 to 20 cell and gene therapy products per year 2.

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pharmaphorum

NOVEMBER 8, 2021

French drugmaker Sanofi has made a $40 million investment in Gyroscope Therapeutics, with another $20 million potentially on offer as the UK biotech advances its clinical-stage gene therapies for ophthalmic diseases. At the moment there is no approved therapy for the disease, which affects around five million people globally.

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Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

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BioPharma Reporter

FEBRUARY 27, 2023

AbbVie and Capsida Biotherapeutics have expanded their strategic collaboration to develop genetic medicines for eye diseases with high unmet need.

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BioPharma Reporter

FEBRUARY 27, 2023

AbbVie and Capsida Biotherapeutics have expanded their strategic collaboration to develop genetic medicines for eye diseases with high unmet need.

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Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

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BioPharma Reporter

JANUARY 2, 2023

Last week saw Opus Genetics announce it had acquired the rights to two preclinical-stage adeno-associated virus (AAV)-based gene therapy product candidates for inherited retinal diseases (IRDs) from Iveric Bio.

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pharmaphorum

AUGUST 8, 2022

As we reported this morning, the deal gives Pfizer already-approved SCD therapy Oxbryta (voxelator) – which industry watchers reckon could see a dramatic uptick in sales with Pfizer’s marketing muscle – plus a phase 3 antibody candidate, a phase 1 follow-up to Oxbryta that could offer improved dosing.

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The Pharma Data

OCTOBER 26, 2020

The agreement covers the gene therapy program targeting OTOF (OTOF-GT), the gene encoding the otoferlin protein. Sensorion is currently advancing two preclinical gene therapy programs conducted under a broad multi-program research partnership with the Genetics and Physiology of Hearing Unit at Institut Pasteur (Paris).

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Pharmaceutical Technology

APRIL 5, 2023

More than a decade after the first patient was treated with a CAR-T therapy, six therapies relying on the same principles have been approved by the US Food and Drug Administration (FDA) and marketed to thousands of patients. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023.

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pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

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The Pharma Data

NOVEMBER 19, 2020

MD, FAAP, Cincinnati Children’s Hospital, who will discuss genetic forms of hearing loss in children. Dr. John Greinwald is a tenured professor of Otolaryngology and Pediatrics with over 20 years of experience with a focus on the genetic causes and treatment of deafness. To register for the call, please click here.

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Drug Discovery World

JANUARY 30, 2024

Viral vector manufacturing At the Advanced Therapies Awards ceremony, Dr Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy, was awarded a Lifetime Achievement Award in recognition of ‘extraordinary contributions to gene therapy’, a field he has helped shape through his pioneering work on lentiviral vectors.

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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.

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The Pharma Data

JANUARY 27, 2021

FDA Also Grants Two Orphan Drug Designations for the Company’s Gene Therapy Programs for Gaucher Disease and Mucolipidosis –. In addition, the FDA granted two orphan drug designations (ODDs) for its gene therapy programs for Gaucher disease and mucolipidosis. Currently, there are no cures for any of these disorders.

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Drug Discovery World

APRIL 17, 2024

Recently, research published by Dr Richard (Rick) Young (Whitehead Institute) describes the key mechanism of how these regRNAs regulate gene expression. CAMP4 is exploiting these regulatory interactions to specifically control the expression of genes tied to disease. How have you navigated a difficult investment climate?

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pharmaphorum

AUGUST 12, 2020

The FDA has started its review of Israeli biotech Protalix BioTherapeutics and partner Chiesi’s Fabry disease therapy pegunigalsidase alfa, setting up a possible approval by 27 January. Market research firm Optima Insights has predicted that sales of Fabry disease drugs will more than double from around $1.8 billion last year to $3.8

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Advarra

JANUARY 11, 2024

Personalized medicine using genetic resequencing techniques, such as cell and gene therapies, enables researchers to create more customized therapies for combatting cancer. Adoptive T Cell Therapies Adoptive T cell therapy is another form of cancer treatment leveraging the patient’s immune system.

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pharmaphorum

JANUARY 12, 2022

EMA “lags behind” other international regulators when it comes to approval timelines, finds EFPIA analysis. The EC’s upcoming review of pharmaceutical regulation, part of the EU Pharmaceuticals Strategy, comes as the region faces substantial unmet need in disease areas from cancers to Alzheimer’s, liver disease to CNS disorders.

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XTalks

MARCH 13, 2024

Related: Transforming Eye Care: Innovating New Glaucoma and Dry Eye Disease Treatments — Featuring Pierre Simon, VP Marketing, Sight Sciences – Xtalks Life Science Podcast Ep. There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease.

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XTalks

NOVEMBER 10, 2023

cTTP is a very rare, inherited and life-threatening blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13) gene, which encodes the ADAMTS13 enzyme that regulates blood clotting by cleaving the von Willebrand factor (VWF) protease.

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Delveinsight

AUGUST 20, 2020

FDA rejects BioMarin’s gene therapy and Gilead’s filgotinib over concerns. The FDA refused to approve BioMarin’s hemophilia A gene therapy valoctocogene roxaparvovec. Gilead is not likely to be able to refile until the next year, pushing it further behind its competitors for the JAK inhibitor market.

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Drug Discovery World

MARCH 13, 2023

The cell and gene therapy (CGT) landscape has grown significantly in the past year. Between the 24 therapies already approved by the FDA 1 and a marked increase in clinical trials, widespread accessibility to precision medicine feels within reach.

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The Pharma Data

NOVEMBER 22, 2020

As the Pfizer and BioNTech COVID-19 vaccine goes to regulators and the Moderna vaccine approaches the end of its Phase III trials, AstraZeneca and the University of Oxford announced high-level results from an interim analysis of their COVID-19 vaccine, AZD1222. It contains the genetic materials of the spike protein. Source link.

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Roots Analysis

JANUARY 14, 2024

Like all drugs, biologics are regulated by the FDA. Since its genetic and physiological profiles are well characterized, genetic manipulation in E. Additionally, novel modalities, including recombinant proteins, vaccines, cell and gene therapies, are growing faster than any other segment of medicine.

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Pharmaceutical Technology

FEBRUARY 3, 2023

With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more. 1] BioNews. Hemophilia News Today. Biores Open Access.

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Drug Discovery World

SEPTEMBER 28, 2022

In fact, the market research firm, Bekryl, predicts that AI has the potential to offer over $70 billion in savings for the drug discovery process by 2028. . According to Hampleton Patners’ latest Healthtech M&A market report2, digital health companies raised a total of $57.2 Some recent highlights from market-leaders include: .

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pharmaphorum

JUNE 11, 2021

These trends combine to increase the pace of innovation and the precision of therapies. Leading academic research institutions and pharma/biopharma players exploring therapeutic areas such as immuno-oncology or cell and gene therapy no longer start ‘analogue only’ projects because of time and productivity shortcomings.

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XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

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Delveinsight

JANUARY 18, 2021

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. The present Angelman syndrome treatment market revolves around the management of symptoms rather than curing the condition. . GeneTx and Ultragenyx are co-developing the therapy.

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Drug Discovery World

JANUARY 26, 2023

DDW Editor Reece Armstrong takes a look at the pharmaceutical research and development market in 2023 and asks key opinion leaders what challenges and trends will emerge throughout the year. billion showed the pharma giant taking further steps into the oncology market and developing its speciality medicines vaccines portfolio.

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Drug Discovery World

AUGUST 10, 2022

By Mark White, Associate Director of Biopharma Product Marketing at Bio-Rad and Marwan Alsarraj, Biopharma Segment Manager, Digital Biology Group. Exactly three decades later, the first CAR T cell therapy hit the market. For example, it is challenging to regulate transgene copy numbers in each T cell, posing safety risks.

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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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pharmaphorum

SEPTEMBER 1, 2020

Previously a centre of the trade in textiles and dyes, Swiss manufacturers gradually began to realise their dyestuffs had antiseptic and other properties and began to market them as pharmaceuticals, in contrast to the origin in pharmacies of other enterprises. Thalidomide and the development of drug safety regulation and monitoring.

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Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. For instance, consider how at the end of 2018, the mRNA therapies market was valued at $3.43

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Drug Discovery World

MAY 11, 2023

Kerstin Pohl , Senior Global Marketing Manager, Gene Therapy & Nucleic Acid at SCIEX, looks at the application of liquid chromatography-mass spectrometry for analysing host cell proteins. Novel gene-based drugs can be severely impacted when HCPs are not distinguished and quantified individually. 2017): 1-12.

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