Pharmaceutical Technology

MAY 4, 2023

It is based on data from a cohort in a Phase III, 24-week, open-label trial assessing the safety, pharmacokinetics and pharmacodynamics of ivacaftor in CF patients with an ivacaftor-responsive CFTR mutation who are under 24 months old. This cohort showed a safety profile identical to that found in older children and adults.

In-Vitro 246

In-Vitro Genetic Disease Medicine Genetics 246

Pharmaceutical Technology

MAY 22, 2023

It is claimed to be both the first re-dosable gene therapy and the first and only FDA-approved treatment for both recessive and dominant types of DEB, a rare and serious genetic disease affecting the skin and mucosal tissues. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinical trials.

FDA Approval 130

FDA Approval Gene Therapy Genetic Disease Gene 130

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. Gene-altering and other advanced medicines are radically rewriting the future for people with some of the rarest, and most devastating, diseases on earth. “They called him ‘The Boy.’

Development 98

Development Gene Therapy Clinical Trials Clinical Trials 98

Drug Discovery World

JANUARY 24, 2023

DMD and FA are rare, degenerative, genetic diseases that affect movement and eventually lead to paralysis. This is particularly important for rare diseases where it can be hard to identify suitable patients. In the FA study, teams worked with patients to identify key movement patterns and predict genetic markers of disease.

Development 52

Development Clinical Trials Clinical Trials Drugs 52

Drug Discovery World

DECEMBER 5, 2022

A new partnership between industry and three rare disease foundations has formed with the goal of accelerating the development of precision drugs to treat rare neurodevelopmental disorders. . This lack of comprehensive data increases the risk of program failure in clinical trials. .

Development 52

Development Genome Genomics Drugs 52

Drug Discovery World

JUNE 22, 2023

According to ARM, all seven gene therapies for rare genetic diseases would have been rejected under the baseline methodologies proposed for the EU joint clinical assessment (JCA) starting in 2025. Single-arm trials are often medically, scientifically, and ethically necessary in the case of ATMPs.

Gene Therapy 94

Gene Therapy Genetic Disease Gene Clinical Trials 94

Drug Discovery World

FEBRUARY 28, 2024

The first 20 participants have been enrolled in a multicentre trial to test whether ImmunityBio’s Nant Cancer Vaccine (NCV) could potentially prevent colon and other cancers in individuals with Lynch syndrome. The post Combination cancer vaccine to be assessed in Phase II trial appeared first on Drug Discovery World (DDW).

Vaccination 52

Vaccination Vaccine Trials Protein 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.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

pharmaphorum

OCTOBER 25, 2022

The Japanese firm has agreed to make a $50 million investment in Dallas-based Taysha in exchange for a 15% stake in the company, plus exclusive options to license two clinical-stage, single-gene therapies for rare genetic diseases. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on.

Gene Therapy 84

Gene Therapy Gene Genetic Disease Genetics 84

Drug Discovery World

JULY 11, 2022

However, the speed at which CGT products can impact healthcare depends on how the industry will navigate novel challenges and risks from the nascent field of CGT development. These challenges exist across the translation cycle from early research to clinical trials, manufacturing, and ultimately reimbursement. Regulatory requirements.

Gene Therapy 52

Gene Therapy Gene Development Production 52

XTalks

APRIL 15, 2021

million people in the US, yet there is no treatment for the most common form of the disease: heart failure with preserved ejection fraction (HFpEF). It’s not just one disease,” says Dr. New advances in heart failure genomics are helping to address this challenge. Heart failure affects about 6.2 HFrEF vs. HFpEF.

Development 98

Development Drugs Genome Genomics 98

XTalks

SEPTEMBER 17, 2020

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Verifying the biomarker’s clinical validity for use as a surrogate endpoint in rare disease research is another hurdle which is generally a longer-term goal.

Trials 98

Trials Gene Hormones Clinical Trials 98

Drug Discovery World

MARCH 19, 2024

Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system. This is the first FDA-approved treatment option for children who have this rare genetic disease,” said Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research (CBER). “We

Gene Therapy 52

Gene Therapy Gene FDA Approval Genetic Disease 52

Pharmaceutical Technology

JULY 29, 2022

CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year. Ultragenyx Pharmaceuticals has an orthinine transcarbamylase (OTC) activator drug in Phase III trials. Future Possibilities.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial. James Miessler.

Genetic Disease 52

Genetic Disease Drugs Licensing Licensing 52

Drug Discovery World

JANUARY 30, 2024

“Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Developing more efficient and affordable alternatives is key. Streamlining trial designs and regulatory pathways can help.

Gene Therapy 71

Gene Therapy Manufacturing Gene Clinical Trials 71

pharmaphorum

JULY 20, 2022

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on. .

RNA 52

RNA Genetic Disease Drugs Protein 52

XTalks

MARCH 21, 2024

XTALKS WEBINAR: Cell and Gene Therapy Development: Characterization of Cellular Source Material Live and On-Demand: Tuesday, May 14, 2024, at 11am EDT (4pm BST/UK) Register for this free webinar to learn about characterizing cellular source material for developing and manufacturing cell and gene therapy-based products.

Gene Therapy 111

Gene Therapy Gene Drugs FDA Approval 111

XTalks

MAY 26, 2023

The regulatory nod “ushers in a whole new paradigm to treat genetic diseases” and is a major milestone for DEB patients and their families, Krystal Biotech CEO Krish Krishnan said in a statement from the company. Results of the trials are published in the journals Nature Medicine and The New England Journal of Medicine.

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

pharmaphorum

JANUARY 27, 2023

Late last year, pharmaphorum caught up with Dr Karen Mullen, chief medical officer and VP of clinical & medical affairs at global drug development consultancy Boyds. Clinical trials design and patient input The definition of patient centricity, in fact, and its benefits are now – finally – being defined by patients themselves. “We

Clinical Trials 106

Clinical Trials Clinical Trials Trials Development 106

The Pharma Data

SEPTEMBER 22, 2020

Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. This guidance provides clarity on the path to registration for AVXS-101 IT.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

Pharmaceutical Technology

JUNE 19, 2023

Pharmaceutical companies are putting their trust in the immense potential this new generation of medicine has for treating individuals with rare genetic diseases, which currently affect an estimated 280 million patients worldwide. The majority of these are in Phase I (68%) and Phase II (24%), with just 8% in Phase III.

Gene Therapy 130

Gene Therapy Gene Packaging Packaging 130

XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

RNA 105

RNA Genetics Genetic Disease Medicine 105

pharmaphorum

OCTOBER 4, 2020

If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD that usually emerges in boys during early childhood and causes physical and mental disabilities as well as behavioural problems.

Gene Therapy 97

Gene Therapy Gene Genetic Disease Protein 97

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. While most gene therapy clinical studies are ongoing, a number of products are in advanced clinical development, and several are approved by FDA. PK Planning for Gene Therapy Development Programs. Exposure-Response Considerations.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

pharmaphorum

OCTOBER 7, 2020

Axovant has said it plans to continue developing its Parkinson’s Disease gene therapy after reporting supportive data from a small cohort of patients from a phase 2 trial. The data came from a second cohort of just four patients in the phase 2 clinical trial SUNRISE-PD, although the improvement in symptoms was dramatic.

Gene Therapy 117

Gene Therapy Gene Genetic Disease Trials 117

Drug Discovery World

FEBRUARY 27, 2023

The study found that four out of five patients diagnosed with Sanfilippo have continued to gain cognitive skills in line with development in healthy children after being given the investigational gene therapy, OTL-201.

Gene Therapy 52

Gene Therapy Gene Genetics Genetic Disease 52

Drug Discovery World

NOVEMBER 7, 2022

In 2018, the UK’s department of health announced an NHS Genomic Medicine Service, which allows patients with rare diseases to have their entire genetic code read in the hope of providing a much-needed diagnosis. That said, some people with rare genetic diseases remain without a molecular diagnosis after their genome is analysed. . “We

Genome 52

Genome Genomics Genetics Genetic Disease 52

Drug Discovery World

MARCH 6, 2024

The prevalence of these diseases has grown substantially in recent decades due to an increasing global population, longer life expectancies and increasing morbidity with predisposing risk factors. This now represents one of the most challenging and costly burdens to health and social care systems and societies worldwide.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

pharmaphorum

SEPTEMBER 28, 2022

The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market. “We continue to work with urgency to bring forward the first CRISPR therapy for a genetic disease,” said Nia Tatsis, Vertex’ chief regulatory and quality officer, in a statement.

Gene Editing 57

Gene Editing Gene In-Vivo Clinical Trials 57

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

Drugs 97

Drugs Genetic Disease Gene Therapy Trials 97

XTalks

FEBRUARY 28, 2022

Related: UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD. Below are some facts and information about rare diseases, including rare disease clinical trials and orphan drugs. How is a Rare Disease Defined? Are Most Rare Diseases Genetic?

Clinical Trials 98

Clinical Trials Clinical Trials Genetics Trials 98

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. mRNA exists in every cell within the human body and this, along with its role in the development of proteins, are the reasons why mRNA therapies have such potential in treating many diseases.

RNA 52

RNA Protein Vaccination Vaccine 52

Delveinsight

AUGUST 26, 2021

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. Shape’s RNA editing technology could potentially modify the amount of a key regulatory protein in the body or treat genetic diseases.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

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. Five of the mice had adenomas and one had a hepatocellular carcinoma (HCC).

Gene Therapy 85

Gene Therapy Gene Genetic Disease DNA 85

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. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

pharmaphorum

OCTOBER 16, 2020

In the age of artificial intelligence, no trial data should be going to waste. Findacure’s Rick Thompson looks at how these technologies could bring us closer to treatments for underserved rare diseases. The repurposing of drugs is becoming more common, especially in the field of rare diseases.

Clinical Trials 101

Clinical Trials Clinical Trials Trials Life Science 101