Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.

Gene Therapy 64

Gene Therapy Gene Clinical Trials Clinical Trials 64

XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). DMD is caused by the absence of dystrophin, a protein that helps maintain the integrity of muscle cells.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

pharmaphorum

MAY 13, 2021

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. The first patient was recruited in The Newcastle upon Tyne Hospitals NHS Foundation Trust, one of three UK sites for the clinical trial and part of 55 globally, across 15 countries.

Gene Therapy 80

Gene Therapy Gene Trials Protein 80

pharmaphorum

DECEMBER 22, 2021

Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a young male patient. AAV vectors are well-suited to gene therapies for DMD because they are effective at targeting muscle tissue.

Gene Therapy 57

Gene Therapy Gene Trials Clinical Trials 57

XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

Gene Therapy 98

Gene Therapy Gene FDA Approval Antibody 98

pharmaphorum

DECEMBER 2, 2021

Astellas has signed another bolt-on deal to build its gene therapy pipeline, agreeing a deal with Dyno Therapeutics to tap into its adeno-associated virus (AAV) vector platform for delivering genes to skeletal and cardiac muscle. Those safety issues have hit Astellas’ aspirations in gene therapy directly.

Gene Therapy 82

Gene Therapy Gene Manufacturing Clinical Trials 82

Drug Discovery World

DECEMBER 19, 2022

Researchers have found that a topical gene therapy gel helps heal wounds in patients with life-threatening blistering skin disease epidermolysis bullosa (EB). . One trial participant was Vincenzo Mascoli, aged 22. This treatment made a huge difference in quality of life for Vincenzo and other trial participants.” .

Gene Therapy 52

Gene Therapy Gene Dermatology Clinical Trials 52

pharmaphorum

AUGUST 1, 2022

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making it available in sometime around the middle of 2023.

Gene Therapy 100

Gene Therapy Gene Trials Genetics 100

BioPharma Reporter

OCTOBER 22, 2020

Scientists have used gene therapy and a newly developed light-sensing protein to restore sight in mice: with the technology to start clinical trials in the US later this year.

Gene Therapy 98

Gene Therapy Gene Clinical Trials Clinical Trials 98

pharmaphorum

JANUARY 12, 2022

The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file for regulator approval. point increase on the NSAA scale with the gene therapy, while the control group saw a 0.7-point

Gene Therapy 94

Gene Therapy Gene Regulation Trials 94

XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. Related: Is $2 Million Too Much For FDA-Approved SMA Gene Therapy? With a price tag of over $2.5

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

pharmaphorum

JULY 7, 2022

New data on Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety. ” The post Myocarditis case mars Sarepta DMD gene therapy readout appeared first on.

Gene Therapy 57

Gene Therapy Gene Protein Trials 57

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.

Gene Therapy 72

Gene Therapy Gene Genetic Disease DNA 72

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.

Gene Therapy 52

Gene Therapy Gene Trials Gene Expression 52

pharmaphorum

NOVEMBER 5, 2020

An experimental gene therapy developed by Texas biotech Genprex will be paired with AstraZeneca’s Tagrisso and Merck & Co’s Keytruda – both leading their respective drug classes in the treatment of non-small cell lung cancer (NSCLC). Tagrisso is the top-selling EGFR inhibitor, with sales of almost $3.2

Gene Therapy 98

Gene Therapy Gene Drugs Contract Manufacturing 98

XTalks

DECEMBER 27, 2022

Adstiladrin is an adenoviral vector-based gene therapy that is non-replicating, so it cannot multiply in human cells. Boorjian, MD, Carl Rosen Professor and Chair of the Department of Urology at Mayo Clinic, and lead investigator on the recent clinical trial of Adstiladrin, in the company’s press release.

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52

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.

Engineer 113

Engineer Trials Genetic Disease Gene Therapy 113

pharmaphorum

OCTOBER 4, 2020

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review. CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein.

Gene Therapy 89

Gene Therapy Gene Genetic Disease Protein 89

pharmaphorum

DECEMBER 10, 2020

Shares in Rocket Pharmaceuticals have been living up to their name, shooting up following encouraging early-stage clinical trial results from a gene therapy for a serious inherited rare heart disease. As of November, three patients have been treated with a low dose of the therapy and two have been treated with a high dose.

Gene Therapy 58

Gene Therapy Gene Protein Trials 58

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. billion, compared to $19.9

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

Pharmaceutical Technology

JUNE 9, 2023

RP-A601 is an adeno-associated virus-based gene therapy currently being evaluated as a one-time, potentially curative treatment to improve survival and quality of life for PKP2-ACM patients. The existing standard of care for the disease includes medical therapy, ablation procedures and implantable cardioverter defibrillators (ICDs).

Gene Therapy 147

Gene Therapy Gene FDA Approval Trials 147

Pharmaceutical Technology

MARCH 27, 2023

Regenerative medicines in early-stage development (preclinical, discovery, or investigational new drug [IND]/ clinical trial application [CTA] filed status) have seen a change in drug targets compared to therapies in late-stage development (Phase II to pre-registration stage).

Medicine 246

Medicine Gene Therapy Gene Dermatology 246

Delveinsight

DECEMBER 22, 2020

Vorasidenib is an investigational, brain-penetrant, dual inhibitor of mutant IDH1 and IDH2 in a Phase III trial in patients with IDH-mutant low-grade glioma, AG-270 is an investigational first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor while AG-636 is a novel inhibitor of dihydroorotate dehydrogenase.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

pharmaphorum

MARCH 8, 2021

million one-off gene therapy for Spinal Muscular Atrophy (SMA) NICE has said, the most expensive treatment ever approved for funding. This is the second gene therapy recommended for funding by NICE – in 2019 Novartis’ Luxturna (voretigene neparvovec) was approved for NHS funding for a rare inherited eye disease.

Gene Therapy 74

Gene Therapy Gene Marketing Genetics 74

BioSpace

DECEMBER 10, 2020

In a shining moment that signaled hope for hemophilia B patients, uniQure presented data on Tuesday showing that its gene therapy treatment, etranacogene dezaparvovec (AMT-061), substantially increased production of the blood-clotting protein factor IX in nearly all pivotal Phase III HOPE-B trial.

Gene Therapy 84

Gene Therapy Trials Protein Gene 84

Pharmaceutical Technology

SEPTEMBER 22, 2022

Avrobio has received rare pediatric disease designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, AVR-RD-04, designed to treat cystinosis. AVR-RD-04 works by genetically modify a patient's hematopoietic stem cells (HSCs) to express the gene that encodes the cystinosin protein.

Gene Therapy 147

Gene Therapy Gene Protein Clinical Trials 147

Pharmaceutical Technology

APRIL 21, 2023

CARsgen chief medical officer Dr Raffaele Baffa said: “Pancreatic cancer remains a deadly disease with a dismal prognosis and lack of effective therapies. As indicated in previous clinical trials, CT041 has shown promising efficacies in treating pancreatic cancer. It targets CLDN18.2

Gene Therapy 147

Gene Therapy Gene Trials Clinical Trials 147

The Pharma Data

JUNE 28, 2021

Parkinson’s disease is most common neurodegenerative movement disorder / It impacts more than 10 million people worldwide/ No function-restoring therapy is currently available / Bayer is pursuing a two-pronged approach to deliver transformative therapies with one cell and one gene therapy candidate in clinical trials.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

OCTOBER 27, 2020

Dry AMD is one of the leading causes of vision loss globally and gene therapies hold tremendous promise for this disease. Delivering gene therapy directly to the site of disease through a subretinal injection is a proven approach; however, the current method requires a vitrectomy. at Ophthalmic Consultants of Boston.

Gene Therapy 40

Gene Therapy Gene Trials Clinical Trials 40

XTalks

NOVEMBER 6, 2020

Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinical trials this week. The first healthy volunteer was dosed with the vaccine in Australia as part of the bacTRL-Spike COVID-19 Phase I clinical trial. BacTRL Gene Therapy Platform.

Clinical Trials 75

Clinical Trials Clinical Trials Vaccination Vaccine 75

Pharmaceutical Technology

OCTOBER 10, 2022

A clinical trial of the vaccine is currently underway in individuals aged 12 years and above. The data from the trial will be provided to the authorities in the coming months. g of mRNA expressing the wild-type spike protein of SARS-CoV-2, and 15-?g 5 subvariants’ spike protein. 5-adapted bivalent vaccine.

Vaccination 147

Vaccination Vaccine Gene Therapy Protein 147

pharmaphorum

JULY 22, 2021

Bluebird bio has its first approval for Skysona (formerly Lenti-D), its gene therapy for children with the rare disease cerebral adrenoleukodystrophy (CALD), after getting the nod from the EU regulatory authority. The modified cells are reinfused and can produce a functional version of the ALD protein that is lacking in CALD.

Gene Therapy 97

Gene Therapy Gene Marketing Trials 97

Drug Discovery World

APRIL 3, 2024

In the last few years, immunotherapies like immune checkpoint inhibitors, vaccines and cell and gene therapies have revolutionised the treatment of cancer, and targeted drugs like monoclonal antibodies (mAbs), poly-ADP ribose polymerase (PARP) inhibitors and targeted protein degraders are also playing an important role.

Research 52

Research Drugs Gene Therapy Genetics 52

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

Pharmaceutical Technology

NOVEMBER 7, 2022

5 Omicron variants’ spike protein and 25µg encoding for the virus’ initial strain. The latest authorisation is based on the pre-clinical findings for mRNA-1273.222 and results from a Phase II/III clinical trial of mRNA-1273.214 (SPIKEVAX Bivalent), which acts on the Omicron BA.1 1 subvariant. 5 versus mRNA-1273.

Vaccination 130

Vaccination Vaccine Gene Therapy Clinical Trials 130