Pharmaceutical Technology

MARCH 10, 2023

On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UK’s ability to develop and manufacture cell and gene therapies. Personalised medicines will also be developed at the centre.

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Pharmaceutical Technology

MAY 16, 2023

The Foundation for the National Institutes of Health (FNIH) has announced its plans to prioritise eight rare diseases to provide industry standards for manufacturing, preclinical testing and product analytical testing for gene therapy development.

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Pharmaceutical Technology

AUGUST 25, 2022

The European Commission (EC) has granted conditional marketing authorisation (CMA) for BioMarin Pharmaceutical ’s gene therapy, Roctavian (valoctocogene roxaparvovec), to treat adults with severe haemophilia A (congenital Factor VIII deficiency). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

FEBRUARY 28, 2024

AGTC-501 is in Phase II development for retinitis in the SKYLINE trial (NCT03316560) and addresses unmet needs within the retinitis space

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Bio Pharma Dive

MAY 16, 2023

Backed by nearly $100 million, the public-private consortium aims to create a standard development roadmap for gene therapies using AAV viral vectors.

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Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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Pharmaceutical Technology

JUNE 8, 2023

The use of 4D Molecular Therapeutics’ (4DMT) aerosolised gene therapy 4D-710 has improved the quality-of-life and spirometry-measured outcomes in three cystic fibrosis patients , based on early results from a Phase I/II study presented at this year’s annual meeting of the European Cystic Fibrosis Society (ECFS).

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Camargo

DECEMBER 20, 2021

One of the biopharmaceutical industry’s most closely watched forthcoming regulatory developments is the FDA’s imminent reauthorization of the Prescription Drug User Fee Act (PDUFA). Many of the advancements included in PDUFA VII are driven in large part by an increase in the development of innovative products such as cell and gene therapies.

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Pharmaceutical Technology

JULY 7, 2022

For many decades, investigators have been working on innovative therapeutic modalities known as cell and gene therapies, which use modified versions of the body’s own cellular and genetic material to treat and potentially cure these diseases. A new frontier in cancer research. Securing the supply chain.

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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.

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Pharmaceutical Technology

JULY 22, 2022

The spotlight was placed on many up-and-coming pharmacotherapies for retinal diseases, one of which was AbbVie’s/Regenxbio’s gene therapy RGX-314. Clinical trial results in DR patients have shown that 47% of eyes treated with RGX-314 have experienced diabetic retinopathy severity score (DRSS) improvements of two or more steps.

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pharmaphorum

SEPTEMBER 26, 2022

Pfizer and Sangamo have restarted recruitment of patients into their registration trial for haemophilia A gene therapy giroctocogene fitelparvovec, after the FDA lifted a clinical hold on the study. The post Pfizer, Sangamo resume haemophilia gene therapy trial appeared first on.

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Bio Pharma Dive

SEPTEMBER 13, 2022

The development comes three weeks after Roctavian was approved in Europe and ahead of a planned regulatory submission in the U.S. Drug regulators have not ordered a trial hold, however.

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Pharma Mirror

APRIL 28, 2021

Catalent will provide process development and CGMP manufacturing of AavantiBio’s adeno-associated viral (AAV) vector-based therapeutic candidate for use in clinical trials in the U.S. and Europe. Catalent will further support process optimization and look to reduce material.

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XTalks

JUNE 8, 2023

Over the past few years, there has been a significant expansion in the cell and gene therapy landscape, with an increasing number of therapies entering clinical trials and receiving regulatory approvals. “I think if we all go into these relationships with this mindset, we’ll achieve great things together.”

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Pharmaceutical Technology

MAY 24, 2023

The RMAT designation programme is intended to accelerate the drug’s development and review processes for products, including gene therapies. The regulator granted RMAT designation based on the data obtained from the ongoing Phase I RP-L301 clinical trial.

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Pharmaceutical Technology

MAY 12, 2023

Editas Medicine will release new efficacy and safety results of its gene therapy EDIT-301 in severe sickle disease as part of an oral presentation at the European Hematology Association’s (EHA) Hybrid Congress. In December 2022, the company presented data from the first two patients treated in the Phase I/II RUBY (NCT04853576) trial.

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XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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Drug Discovery World

DECEMBER 18, 2023

University College London (UCL) researchers have developed a new gene therapy to cure focal cortical dysplasia, which a new study shows can significantly reduce seizures in mice. The researchers plan to conduct a first-in-human clinical trial in the next five years.

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Drug Discovery World

OCTOBER 9, 2023

Positive Phase I trial data indicates that Reqorsa (quaratusugene ozeplasmid) has shown early efficacy for the treatment of non-small cell lung cancer (NSCLC). Developed by gene therapy company Genprex, Reqorsa is a non-viral gene therapy that leads to expression of the TUSC2 tumour suppressor gene in cancers.

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Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years. According to GlobalData’s clinical trials database, there are currently 1,231 planned and ongoing trials for gene therapies and gene-modified cell therapies alone.

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Pharmaceutical Technology

MAY 25, 2023

Pushing back an initial deadline, the US Food and Drug Administration (FDA) has proposed a new regulatory action date of 22 June, by which time the agency will assess the logistics of a possible approval for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy.

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pharmaphorum

SEPTEMBER 14, 2021

There has been a fourth patient fatality in Astellas’ clinical trial of its AT132 gene therapy for the rare disease X-linked myotubular myopathy (XLMTM), which has been halted twice due to safety concerns. It said it was “committed to the development of AT132 and the XLMTM patient community.”

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XTalks

JUNE 14, 2023

Courtney Silverthorn who is an Associate Vice President at the Foundation for the National Institutes of Health (FNIH) and the Director of the Accelerating Medicines Partnership (AMP) program including the Bespoke Gene Therapy Consortium. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

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Worldwide Clinical Trials

JUNE 15, 2022

At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Around 40% of clinical holds are for gene therapy programs.

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Pharmaceutical Technology

JULY 14, 2022

Avrobio has received orphan drug designation for its gene therapy, AVR-RD-05, from the US Food and Drug Administration (FDA) to treat mucopolysaccharidosis type II (MPSII) or Hunter syndrome. The company noted that this gene therapy is the fourth one to receive orphan drug designation.

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Drug Discovery World

OCTOBER 31, 2023

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The drug was developed over eight years by Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester.

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Drug Discovery World

APRIL 19, 2024

Askbio’s AB-1005 (also known as AAV2-GDNF), an investigational gene therapy for the treatment of Parkinson’s disease, was well tolerated with no serious adverse events in a Phase Ib clinical trial. mL) were well tolerated, with no SAEs associated with the investigational gene therapy or contrast agent.

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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.

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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.

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FDA Law Blog

MARCH 13, 2023

Valentine — Incorporating patient and caregiver experiences into every phase of drug development has become increasingly prioritized during both development and review ( see, e.g. , previous coverage here ). At least some (if not all) of the panelists will have had experience participating in gene therapy trials.

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pharmaphorum

DECEMBER 16, 2022

CSL’s gene therapy for haemophilia B has been recommended for approval by the EMA’s human medicine committee, setting up a decision by the European Commission early next year. The post CSL closes on EU approval for haemophilia B gene therapy appeared first on.

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BioPharma Reporter

AUGUST 24, 2022

BrainVectis, a subsidiary of AskBio, has received clearance from the French authorities to start a Phase 1-2 clinical trial for its novel Huntingtonâs Disease gene therapy, BV-101.

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BioPharma Reporter

DECEMBER 8, 2022

Emmes has announced the creation of a dedicated center for cell and gene therapy research: which will focus on supporting clinical trials for the Clinical Research Organizationâs (CRO) clients.

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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).

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Cloudbyz

MARCH 30, 2023

Cell and gene therapies are rapidly becoming an essential component of modern medicine, offering hope to patients with previously untreatable diseases. However, the development and delivery of cell and gene therapies present significant operational challenges that must be navigated carefully to ensure successful clinical trials.

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Bio Pharma Dive

SEPTEMBER 11, 2023

The decision follows a review by a trial monitoring committee, which concluded data for the geographic atrophy treatment didn't support further development.

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