Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . If successful, this novel approach for treating Huntington’s Disease may impact how we treat many other neurodegenerative diseases in the future. . “If

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XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio. As such, rare disease patients and their families often face little hope for effective treatments.

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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.

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STAT News

MARCH 11, 2023

The Food and Drug Administration on Friday approved the first treatment for Rett syndrome, a genetic disease mostly affecting girls that causes severe neurologic impairments, robbing them of the ability to communicate or control muscle movement. The new drug, called Daybue, is made by Acadia Pharmaceuticals.

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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).

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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.

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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.

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Pharmaceutical Technology

OCTOBER 25, 2022

TSHA-102 is the first gene therapy in the clinical development stage for Rett syndrome currently while TSHA-120 is being analysed in a Phase I/II clinical trial for treating GAN. Astellas will also receive specific rights linked to any possible change of Taysha’s control.

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Pharma Times

MARCH 27, 2024

The progressive and incurable brain disorder currently affects approximately 100 patients worldwide

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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.

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Drug Discovery World

MAY 3, 2024

Importantly, these studies also indicated that emactuzumab has good tolerability and a manageable safety profile. SynOx is initiating a Phase III trial (TANGENT) to assess the efficacy and safety of emactuzumab in patients with locali s ed and diffuse TGCT.

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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.

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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.

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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.

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Drug Discovery World

JANUARY 24, 2023

By combining human movement data gathered from wearable tech with a powerful new medical AI technology, the team were able to significantly increase the efficiency of clinical trials in two very different rare disorders, Duchenne muscular dystrophy (DMD) and Friedreich’s ataxia (FA).

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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.

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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

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Drug Discovery World

DECEMBER 5, 2022

This lack of comprehensive data increases the risk of program failure in clinical trials. . A rare genetic disease caused by variation in the SYNGAP1 gene, common symptoms include intellectual disability, low muscle tone, speech delays, and epilepsy. .

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pharmaphorum

JANUARY 27, 2023

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 We talk a lot in clinical trials and drug development about benefit,” Dr Mullen said. But who is benefitting?

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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

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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.

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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. All clinical trials rely on a well-oiled machine of motivated professionals, but nowhere is this more true than in advanced-therapy studies in rare diseases where knowledge is sparse. Collaboration.

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pharmaphorum

OCTOBER 4, 2020

A few weeks ago, Bluebird reported new data from the phase 2/3 STARBEAM trial of Lenti-D which showed that 87% of CALD patients were still alive and free of major functional disabilities after at least two years’ follow-up. Around 40% of patients develop the cerebral form of ALD, which in turn affects around one in 17,000 live births.

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Drug Discovery World

JANUARY 11, 2024

Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification. Dr Baruteau said: “We have shown that mRNA holds an unprecedented therapeutic potential for incurable genetic diseases, in particular liver conditions.

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The Pharma Data

NOVEMBER 27, 2020

Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity. BOSTON, Nov.

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pharmaphorum

DECEMBER 9, 2021

An FDA advisory committee has delivered a blow to Reata Pharma, after voting unanimously that the drugmaker’s data on bardoxolone – a drug for kidney disease – did not show it is effective. Advisors also had some concerns about the safety of the drug and the design of the pivotal trial used to support the marketing application.

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XTalks

NOVEMBER 27, 2023

Pompe disease primarily manifests as progressive muscle weakness, particularly affecting the skeletal muscles in regions such as the hips, legs, shoulders, arms and diaphragm. Sanofi also offers another ERT for Pompe disease known as Myozyme, which received FDA approval in 2006, and Lumizyme in 2010 for late-onset disease.

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pharmaphorum

SEPTEMBER 28, 2022

Clinical trial results reported at this year’s European Haematology Association (EHA) congress showed that a one-time treatment with exa-cel had a significant benefit in both SCD and thalassaemia patients. HbF produces normal, healthy red blood cells, rather than the misshapen cells produced by faulty haemoglobin in the two disorders.

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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?

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Drug Discovery World

FEBRUARY 27, 2023

However, the researchers urge caution as the majority of patients have not reached the age of four to five years where the most severe stages of disease progression typically present. Patients with MPS-IIIA have a mutation in the SGSH gene, causing them to lack an enzyme which normally breaks down large sugar molecules.

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XTalks

SEPTEMBER 22, 2022

Clinical Trial Behind Spevigo. The efficacy of Spevigo (spesolimab) was determined based on the results of the Effisayil 1 double-blind, randomized, Phase II clinical trial. GPP occurs in individuals with specific gene variations as it is a genetic disease , and variations in the genes IL36RN and AP1S3 are known to cause it.

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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.

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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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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. Streamlining trial designs and regulatory pathways can help. Optimising these processes and exploring alternative delivery methods can bring down costs.

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XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.” FDA officials said it had not seen such cases in Vertex patients, but would look out for side effects.

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Delveinsight

AUGUST 26, 2021

Shape’s RNA editing technology could potentially modify the amount of a key regulatory protein in the body or treat genetic diseases. The company then utilizes the de-identified records to improve the design of clinical trials and quality of life measurements. and Leila Zegna, director of the Kabuki Syndrome Foundation.

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Drug Discovery World

AUGUST 28, 2023

DDW’s Diana Spencer speaks to Peter Hamley , Chief Scientific Officer of Samsara Therapeutics, to understand the role autophagy plays in neurodegenerative diseases and how boosting this process could increase healthspan. DS: Samsara’s autophagy inducing candidate SAM001 will be the first agent of its class to enter clinical trials.

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