Gene Therapy and Trials - Clinical Research Informer

FDA panel backs bluebird’s CALD gene therapy, despite safety worries

pharmaphorum

JUNE 10, 2022

The FDA may have safety concerns abut bluebird bio’s gene therapy for rare, fatal disease cerebral adrenoleukodystrophy (CALD), but its advisors believe its benefits far outweigh the risks. bluebird is also conducting the phase 3 ALD-104 trial of eli-cel in CALD, which is due to generate results in 2024.

Gene Therapy

Gene Therapy Gene FDA Approval Trials

Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA

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

Gene Therapy Gene Protein Trials

Ophthalmic Drugs Conference

pharmaphorum

JULY 15, 2022

SAE Media Group’s 5th Annual Ophthalmic Drugs Conference will explore advancements in ophthalmic treatments and variety of novel drugs, which are showing good clinical trial data. Further key areas will be gene therapy and Artificial Intelligence with developments on the latest drug pipelines and clinical trials.

Gene Therapy

Gene Therapy Drugs Drug Delivery Clinical Trials

BioMarin Announces Positive Phase 3 Gene Therapy Trial Results in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set

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

Gene Therapy Gene Trials Clinical Trials

Chairs from Aerie Pharmaceuticals & Santen Inc Invitation to join Ophthalmic Drugs

pharmaphorum

NOVEMBER 7, 2022

The conference will explore advancements in ophthalmic treatments and variety of novel drugs, which are showing great clinical trial data. Further key areas will be gene therapy and Artificial Intelligence with developments on the latest drug pipelines and clinical trials. The conference is co-chaired by Mitchell A.

Drugs

Drugs Clinical Trials Clinical Trials Gene Therapy

Second unanimous FDA adcomm vote boosts bluebird bio

pharmaphorum

JUNE 12, 2022

bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern. By the end of the week, it was two for two, with both recommended for approval by a unanimous vote.

Gene Therapy

Gene Therapy Gene Marketing Trials

Chiesi’s flagship Fabry drug heads for FDA verdict in early 2021

pharmaphorum

AUGUST 12, 2020

Earlier this year, Protalix and Chiesi reported results from the phase 3 BRIDGE trial showing that a year’s treatment with pegunigalsidase alfa was well-tolerated and seemed to slow kidney disease progression more effectively than Replagal, whilst also reducing biomarkers of Fabry disease. billion last year to $3.8 billion last year to $3.8

Drugs

Drugs Genetic Disease Gene Therapy Trials

Sickle Cell Disease: The Importance of Amplifying Patient Voices

Advarra

MARCH 10, 2023

To add even more complexity to this rare disease, there are different types : Hemoglobin SS Hemoglobin SC Hemoglobin SB+(beta) thalassemia Hemoglobin SB 0 (Beta-zero) thalassemia Currently, the only cure for SCD is accomplished through a bone marrow transplant. It’s not just a lack of specialists in the U.S.

Clinical Trials

Clinical Trials Clinical Trials Trials Doctors

FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

Pharmaceutical Technology

AUGUST 18, 2022

A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis. The latest approval is based on findings from the Phase III clinical trials, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up LTF-303 study. 0 genotypes.

Gene Therapy

Gene Therapy Gene Genotype Genetics

Expectations rise for gene therapy to change treatment landscape

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

Gene Therapy

Gene Therapy Gene Gene Editing DNA

Register for the Oligonucleotide Therapeutics and Delivery Conference

pharmaphorum

JUNE 28, 2022

Listen to case studies presenting on pre-clinical and clinical research in areas such as oncology and what can be learned for future clinical trials. For sponsorship enquiries contact Luke Teachen on +44 (0) 20 7827 6002 or email: lteachen@smi-online.co.uk. By attending the conference attendees will have the opportunity to.

RNA

RNA Engineer Development Clinical Trials

Hansa Biopharma provides business update incl. certain key financials ahead of the JP Morgan Global Healthcare Conference

The Pharma Data

JANUARY 10, 2021

However, the global COVID-19 pandemic may still adversely impact Hansa Biopharma’s operational business and trial activities. Randomized Controlled Trial in the US Hansa is in ongoing discussions with the U.S. Imlifidase as pre-treatment ahead of gene therapy in DMD and LGMD.

Gene Therapy

Gene Therapy Gene Antibody Trials

positive result of phase III study with radioligand therapy 177Lu-PSMA-617 in patients with advanced prostate cancer

The Pharma Data

MARCH 22, 2021

VISION trial findings to be presented at upcoming medical meeting, with regulatory submissions in the US and EU anticipated in 2021. Novartis is committed to reimagining prostate cancer through targeted radioligand therapy with 177 Lu-PSMA-617. The safety profile was consistent with data reported in previous clinical studies 1.

Hormones

Hormones Gene Therapy Trials Medicine

Artificial intelligence could be new blueprint for precision drug discovery

The Pharma Data

JULY 12, 2021

. “In head-to-head comparisons, we demonstrated the superiority of this approach over existing methodologies to accurately predict ‘winners’ and ‘losers’ in clinical trials,” said Ghosh. “The ‘phase 0’ trial concept was developed because most drugs fail somewhere between phases I and III.

Drugs

Drugs Gene Expression Big Data Gene

Pfizer Investor Day Features Significant Number of Pipeline Advances for COVID-19 Programs and Across Numerous Therapeutic Areas

The Pharma Data

SEPTEMBER 15, 2020

Limited blinded tolerability data from the ongoing Phase 3 trial, confirming the mostly mild to moderate tolerability profile as was observed in Phase 1. In the blinded data presented, 50% of trial participants received placebo and 50% received BNT162b2. A Phase 3 trial is ongoing. Protease Inhibitor Program. Rare Disease.

Gene Therapy

Gene Therapy Vaccination Vaccine Trials

Pfizer and BioNTech Complete Submission to European Medicines Agency for Omicron BA.4/BA.5-Adapted Bivalent Vaccine Booster in Children 5 Through 11 Years of Age

Pfizer

SEPTEMBER 28, 2022

This study is consistent with regulatory guidance and follows the design of the previous Phase 1/2/3 trial to evaluate further options for protection in this age group. . 5-adapted bivalent COVID-19 vaccine in children 6 months through 11 years of age.

Vaccination

Vaccination Vaccine Medicine Clinical Trials

Pfizer and BioNTech Receive Positive CHMP Opinion for COMIRNATY® in Children 6 Months to less than 5 Years in the European Union

Pfizer

OCTOBER 19, 2022

Today’s recommendation is based on data from a Phase 2/3 randomized, controlled trial that included 4,526 children aged 6 months to less than 5 years. In the trial, children received a third 3-µg dose of the original Pfizer-BioNTech COVID-19 Vaccine at least two months after the second dose at a time when Omicron BA.2

Vaccination

Vaccination Vaccine Clinical Trials Clinical Trials

Top 30 Pharma and Biotech Companies in 2023: Statistics and Trends

XTalks

AUGUST 2, 2023

Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. This is largely due to failed trials as a prophylaxis resulting in the European Committee for Medical Products (CHMP) recommending against the approval of Lagevrio. But all three drugs have new competitors. billion ($1.17

Sales

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Clinical Research Informer

FDA panel backs bluebird’s CALD gene therapy, despite safety worries

Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA

Trending Sources

Ophthalmic Drugs Conference

BioMarin Announces Positive Phase 3 Gene Therapy Trial Results in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set

Chairs from Aerie Pharmaceuticals & Santen Inc Invitation to join Ophthalmic Drugs

Second unanimous FDA adcomm vote boosts bluebird bio

Chiesi’s flagship Fabry drug heads for FDA verdict in early 2021

Sickle Cell Disease: The Importance of Amplifying Patient Voices

FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

Expectations rise for gene therapy to change treatment landscape

Register for the Oligonucleotide Therapeutics and Delivery Conference

Hansa Biopharma provides business update incl. certain key financials ahead of the JP Morgan Global Healthcare Conference

positive result of phase III study with radioligand therapy 177Lu-PSMA-617 in patients with advanced prostate cancer

Artificial intelligence could be new blueprint for precision drug discovery

Pfizer Investor Day Features Significant Number of Pipeline Advances for COVID-19 Programs and Across Numerous Therapeutic Areas

Pfizer and BioNTech Complete Submission to European Medicines Agency for Omicron BA.4/BA.5-Adapted Bivalent Vaccine Booster in Children 5 Through 11 Years of Age

Pfizer and BioNTech Receive Positive CHMP Opinion for COMIRNATY® in Children 6 Months to less than 5 Years in the European Union

Top 30 Pharma and Biotech Companies in 2023: Statistics and Trends

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