Pharmaceutical Technology

OCTOBER 25, 2022

Additionally, Astellas will obtain an exclusive option for licencing two clinical-stage programmes of Taysha, namely TSHA-102 and TSHA-120, for Rett syndrome and GAN, respectively. Astellas will also receive specific rights linked to any possible change of Taysha’s control.

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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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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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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. .

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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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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. In order to find the specific RNA sequences for a genetic disease, the biotech is using an AI- and machine learning-based approach.

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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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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

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. Cell and gene therapies are not your typical clinical supply chain.

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Drug Discovery World

DECEMBER 5, 2022

This lack of comprehensive data increases the risk of program failure in clinical trials. . COMBINEDBrain is devoted to speeding the path to clinical treatments for people with severe rare neurodevelopmental disorders by pooling efforts, studies, and data. .

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Development Genome Genomics Drugs 52

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.

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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.

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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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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

MAY 3, 2024

Dr Incerti has more than three decades of experience in the biopharmaceutical industry and brings an extensive track record in global drug development, including from his time at Sanofi Genzyme where he played a leading role in pioneering therapies for rare and genetic diseases.

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Development Genetic Disease Genetics Drugs 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. Vector production: Viral vectors used in some CGTs are expensive to produce due to complex manufacturing processes and limited yields.

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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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Genome Genomics Genetics Genetic Disease 52

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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XTalks

NOVEMBER 27, 2023

Throughout the course of extensive clinical trials, Pombiliti and Opfolda have consistently exhibited a favorable safety profile, and patients have generally tolerated the treatment well.

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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. Spevigo is the first approved treatment option for GPP in adults.

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Drug Discovery World

MARCH 8, 2023

GNT-0003 combines normal copies of the UGT1A1 gene coding for the bilirubin metabolising enzyme with an AAV vector. In the early phases of the GNT-0003 clinical trials, the treatment of five adult patients demonstrated the safety and good tolerance of the gene therapy as well as a dose effect.

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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. ” Trading in Reata’s shares was halted ahead of the meeting, but the stock was down 38% pre-market this morning.

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Drugs Clinical Trials Clinical Trials Genetic Disease 52

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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XTalks

SEPTEMBER 14, 2022

In this episode, Ayesha discussed the FDA approval of Sanofi’s enzyme replacement therapy Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disease, in adults and pediatric patients.

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pharmaphorum

SEPTEMBER 6, 2021

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down phenylalanine, an amino acid that is commonly found in many foods. The post FDA slaps clinical hold on BioMarin’s PKU gene therapy appeared first on.

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pharmaphorum

OCTOBER 16, 2020

By definition, however, trial master files represent a much richer and more detailed source of data on a drug and how it performs. For instance, provided a drug has not failed a trial on safety, the side effects it caused in one population could constitute on-target effects in another.

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Camargo

JULY 27, 2021

Research and development in the area is currently growing at a fast rate, and the National Institute of Health reports hundreds of clinical trials to test gene therapies for different genetic diseases, immune system disorders, oncology treatments, neurogenerative diseases, infectious diseases, and more.

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The Pharma Data

DECEMBER 17, 2020

The Company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. Sarepta Therapeutics, Inc. www.lysogene.com.

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pharmaphorum

JUNE 28, 2022

Xenpozyme – developed by Sanofi’s rare disease business Genzyme – replaces an enzyme that is deficient in ASMD, reducing fatty accumulation within cells with a dose delivered intravenously every two weeks. “The approval of Xenpozyme by the European Commission represents a transformational shift in what we can offer to patients.

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Genetic Disease Clinical Trials Clinical Trials Genetics 52

pharmaphorum

JANUARY 10, 2022

The technology is being deployed as a therapeutic in two ways – either modifying cells outside the body (ex vivo), which are reinfused to treat a disease – or directly administered (in vivo) to target tissues in the body.

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The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).

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The Pharma Data

NOVEMBER 11, 2020

Received Rare Pediatric Disease and Fast Track Designations for INZ-701 for the treatment of ENPP1 deficiency. Expect to initiate INZ-701 Phase 1/2 clinical trials for ENPP1 and ABCC6 deficiencies in first half of 2021. H1 2021: Initiation of Phase 1/2 clinical trial.

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Clinical Trials Clinical Trials Trials Development 40

Delveinsight

JANUARY 12, 2021

The duo will jointly develop the therapy, conducting clinical trials globally. Bluebird bio recently announced its plans to split its genetic disease and oncology businesses. The company has decided to prioritize its severe genetic disease unit and form its oncology business into a new company. and Europe.

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Drug Discovery World

NOVEMBER 14, 2023

Adzynma provides patients with a treatment option that replaces their deficient ADAMTS13 enzyme and offers a favourable efficacy and safety profile and reduced administration time and volume compared to current plasma-based therapies.

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FDA Approval Gene Clinical Trials Clinical Trials 52

pharmaphorum

JANUARY 9, 2023

Zolgensma meanwhile is now approved in 45 countries, with over 2,500 patients treated globally across clinical trials, managed access programs, and in the commercial setting. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1

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XTalks

NOVEMBER 10, 2023

Related: Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio The FDA approval of Adzynma was based on its demonstrated safety and efficacy in a Phase III global cross-over study, the first of its kind for cTTP. Adzynma’s approval is the second for Takeda this week.

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