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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BioPharma Reporter

AUGUST 18, 2022

Food and Drug Administration (FDA) has approved bluebird bioâs Zynteglo (betibeglogene autotemcel), also known as beti-cel: a one-time gene therapy custom-designed to treat the underlying genetic cause of beta thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.

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pharmaphorum

AUGUST 23, 2022

France’s BrainVectis, a subsidiary of Bayer’s Asklepios BioPharma (AskBio) unit, has been given the green light by regulators in France to start dosing patients with its gene therapy candidate for devastating neurodegenerative disorder Huntington’s disease.

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BioPharma Reporter

NOVEMBER 19, 2024

PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs the normal function of neurons.

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pharmaphorum

FEBRUARY 11, 2022

Data from the first patients enrolled into Regenxbio’s trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the first signs of clinical activity. The post Regenxbio expands MPS I gene therapy trial after first look at data appeared first on.

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BioPharma Reporter

SEPTEMBER 23, 2021

This month sees the launch of a new gene therapy player, Opus Genetics, a company backed and spun out by leading patient group Foundation Fighting Blindnessâ venture arm, the Retinal Degeneration Fund (RD Fund).

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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. The post FDA slaps clinical hold on BioMarin’s PKU gene therapy appeared first on.

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FDA Law Blog

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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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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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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XTalks

APRIL 8, 2025

There are over 10,000 rare diseases affecting an estimated 300 million people worldwide where 80% are genetic , 95% lack approved treatments and nearly half begin in childhood. Cross-border enrollment captures a wider range of genetic backgrounds, environmental influences, and cultural perspectives.

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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

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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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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Pharmaceutical Technology

MAY 22, 2023

Vyjuvek is a non-invasive, topical, re-dosable gene therapy that delivers functional human COL7A1 gene copies to offer wound healing. We offer our sincere gratitude to DEB patients, caregivers, investigators, US regulators and our employees who made this approval possible.

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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. Shares in bluebird ticked up following the announcement.

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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 do the Regulations Say about IBC Review? What Does an IBC Review?

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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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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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Pharmaceutical Technology

APRIL 5, 2023

But while companies continue studying allogeneic CAR-T therapies, including for their coveted use in solid tumours, such advancements remain challenging. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023. AZ: Cell and gene therapies often come with a high price.

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Cloudbyz

MARCH 30, 2023

Cell and gene therapies are rapidly becoming an essential component of modern medicine, offering hope to patients with previously untreatable diseases. However, the development and delivery of cell and gene therapies present significant operational challenges that must be navigated carefully to ensure successful clinical trials.

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XTalks

MARCH 13, 2023

The US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) held a recent town hall where three experts from the regulator provided guidance on how to design and conduct gene therapy clinical trials for rare diseases. It’s a very exciting time in gene therapy.

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XTalks

FEBRUARY 24, 2023

“Many rare disease communities are seeing gene therapies as potential game changers, since most rare diseases are genetically driven,” Dr. Raymond says. Hence, one conversation that needs to take place is between the sponsors developing gene therapies and the payers who in effect “gate” patient access to these novel treatments.

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Delveinsight

FEBRUARY 11, 2021

Avrobio gene therapy eradicates toxic substrate in Fabry patient. The first Fabry disease patient treated with Avrobio’s Plato gene therapy platform in a phase 2 trial has undergone the complete clearance of toxic kidney substrate. Median overall survival in those patients was 5.8

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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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pharmaphorum

FEBRUARY 28, 2022

Both the US Food and Drug Administration (FDA), through the Orphan Drug Act, and the European Medicines Agency (EMA), through the Orphan Medicinal Product Regulation, encouraged the development of orphan drugs, by providing extra support to companies working in the area, or through the offering of financial incentives to pursue rare disease R&D.

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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. billion by 2027, driven by new oral drugs such as Galafold as well as gene therapies that are still in clinical development.

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XTalks

JANUARY 21, 2025

The tricuspid valve, one of the heart’s four valves, regulates blood flow from the right atrium to the right ventricle, preventing backflow between these chambers. There, plasma is separated, and tumor DNA is identified by detecting genetic mutations, methylation patterns and fragmentation signals.

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Roots Analysis

JANUARY 27, 2025

In this form of therapy, patients are injected with living and intact human cells that are deemed to be capable of providing therapeutic benefit. Cell therapies are based on the premise that the patients own cells (autologous), or those from a healthy donor (allogeneic), can be genetically re-programmed to combat various diseases.

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XTalks

MARCH 13, 2024

There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease. The Glaucoma Foundation has funded studies focusing on two specific areas within gene therapy.

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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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Pharmaceutical Technology

APRIL 24, 2023

In an email to this news service, João Conde, PhD, professor at NOVA Medical School, Universidade NOVA de Lisboa, said advances like the mRNA vaccines serve as a testament to the breakthroughs made by science over decades of research at the junction of genetics and nanomedicine. These could feature in the next four to five years.

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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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Scienmag

APRIL 21, 2021

Suppressing a gene regulator could reduce pancreatic cancer resistance to vital chemotherapeutic treatment. Targeting an RNA to interrupt its activity could improve patient response to therapy and increase their overall survival. […].

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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. The team successfully protected HILOs from the immune system without genetic manipulation.

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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. By gene editing the patient’s own stem cells we may have the potential to make this therapy an option for many patients facing these blood diseases,” he added.

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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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