Drug Discovery World

OCTOBER 17, 2023

CARsgen has published evidence of over seven years of disease-free survival in advanced hepatocellular carcinoma (HCC) following treatment with its CAR-GPC3 T cell therapy. However, both patients maintained a tumour-free status during long-term follow-up after receiving a combination of local and CAR T cell therapies.

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Drug Discovery World

JUNE 19, 2024

DDW Editor Reece Armstrong speaks to Jasminka Taleska , Director, RLT HCS Readiness and Policy at Novartis, about the opportunities radioligand therapies (RLTs) represent in treating cancer patients. The post How ready are we for radioligand therapies? RA: What are the potentials of RLTs across cancer? JT: This is very important 6.

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Drug Discovery World

FEBRUARY 15, 2024

Cell and gene therapy company Elicera Therapeutics has received approval from the Swedish Medical Products Agency to start the clinical Phase I/II study CARMA in patients with B-cell lymphoma who no longer respond to standard treatment or have relapsed.

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Drug Discovery World

JANUARY 23, 2023

Over the three days of Advanced Therapies Week (ATW) 2023 in Miami, Florida, US, from 18 to 20 January, DDW’s Megan Thomas heard from attendees about what they were most looking forward to learning from the event, as well as why they think the potential of cell and gene therapies outweighs the obstacles that must be overcome to achieve it.

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Drug Discovery World

OCTOBER 5, 2023

A research team at the Francis Crick Institute and Great Ormond Street Hospital (GOSH)/UCL Great Ormond Street Institute of Child Health have identified potential treatments for children with rare genetic conditions. Both treatments were able to improve the calcium problems, but the genetic therapy was the most effective.

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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. The modified HSCs are then infused back into the patient to engraft in the bone marrow.

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Drug Discovery World

SEPTEMBER 20, 2022

SKYSONA has become the first FDA approved therapy to slow the progression of neurologic dysfunction in boys with cerebral adrenoleukodystrophy (CALD). The post FDA approves gene therapy for rare neurological disorder appeared first on Drug Discovery World (DDW).

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Drug Discovery World

DECEMBER 12, 2023

“For people with large B-cell lymphoma who do not respond to first-line treatment or have an early relapse, outcomes are often poor,” said Professor Roch Houot, Head of Haematology Department, University Hospital of Rennes, France.

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Drug Discovery World

AUGUST 4, 2023

Sven identified the two most prominent barriers as financing, and how to get ground-breaking therapies to the patients that most need them around the world. Sven identified the two most prominent barriers as financing, and how to get ground-breaking therapies to the patients that most need them around the world.

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Drug Discovery World

MARCH 6, 2024

This second ODD allows research and development of a therapy for another form of the disease, Isolated Hypomyelination. Disease research is carried out by the world’s leading centre for leukodystrophy studies, the Children’s Hospital of Philadelphia (CHOP), under a sponsored research agreement.

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Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy Casgevy, a cell-based gene therapy, is approved for the treatment of SCD in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs).

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Drug Discovery World

FEBRUARY 28, 2023

The combination therapy of niraparib and abiraterone acetate (AA) could become the first dual action tablet (DAT) available in the European Union for metastatic castration-resistant prostate cancer (mCRPC) with BRCA1/2 mutations.

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Drug Discovery World

JANUARY 25, 2023

Vésale Bioscience, a company in the research and development of solutions and treatments using phage therapy for multi-resistant infections, has received €1.8 PhageDiag is a fast and user-friendly diagnostic technology, or phagogram, for personalised phage therapy.

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Codon

FEBRUARY 13, 2024

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.

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Drug Discovery World

FEBRUARY 26, 2024

Reece Armstrong speaks to Kathryn Golden , SVP Technical Operations and Manufacturing, bit.bio, about novel technologies, and methods to manufacture human cells for therapies so more patients can access these treatments. Cell therapies have emerged as transformative and potentially curative treatments across a range of disease areas.

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Drug Discovery World

MARCH 13, 2023

GenSight Biologics has revealed pooled safety data from five clinical studies of lenadogene nolparvovec (Lumevoq), the largest cohort of ND4-LHON (Leber hereditary optic neuropathy) patients studied after gene therapy treatment. The post Five-year data show gene therapy safe for ND4-LHON appeared first on Drug Discovery World (DDW).

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Drug Discovery World

AUGUST 11, 2023

iiCON is a consortium led by LSTM with core partners including LifeArc, Unilever, Evotec, Liverpool University Hospitals Foundation Trust, University of Liverpool, and Infex Therapeutics. LifeArc will also join the LSTM-led Infection Innovation Consortium iiCON, making its platform to progress antibody-based treatments available to partners.

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Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. The post Gene therapy for ‘childhood dementia’ shows promise appeared first on Drug Discovery World (DDW).

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Drug Discovery World

OCTOBER 6, 2022

Results from Phase I/II MGT009 study into investigational gene therapy botaretigene sparoparvovec suggest sustained vision improvement in patients with X-linked retinitis pigmentosa (XLRP). . The post Janssen shares ‘encouraging’ gene therapy study results appeared first on Drug Discovery World (DDW). Potential to preserve vision.

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Drug Discovery World

APRIL 16, 2023

Adjuvant therapy with atezolizumab (Tecentriq) and bevacizumab (Avastin) increased recurrence-free survival of patients with hepatocellular carcinoma (HCC) following surgical resection or ablation, according to the results from a Phase III trial. According to the results of an interim analysis conducted after a median follow-up of 17.4

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Drug Discovery World

JULY 25, 2022

A new consortium has been launched in the UK to help drive innovation in the manufacturing of personalised cell therapies. . The consortium was launched by the Cell and Gene Therapy Catapult (CGT Catapult) and will be led by advanced therapy medicinal products (ATMP) developer Achilles Therapeutics. Official comments .

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Drug Discovery World

MAY 16, 2023

The UK’s National Institute for Health and Care Excellence (NICE) has recommended additional uses for two CAR-T therapies from Gilead Sciences/Kite, as part of the Cancer Drugs Fund (CDF). The approval of this CAR T-cell therapy for adult patients with acute lymphoblastic leukaemia represents an important change for adult ALL patients.

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Drug Discovery World

SEPTEMBER 16, 2022

Here, DDW Editor Reece Armstrong explores the global hubs of innovation for cell and gene therapies. Since the approval of Kymirah in 2017, the cell and gene therapy sector has emerged as an exciting and innovative market, representing some of the most promising drugs the pharmaceutical industry has ever seen. .

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Drug Discovery World

FEBRUARY 24, 2023

A Phase I safety and pharmacodynamic activity trial shows IMC-M113V, the first soluble T cell receptor (TCR) therapy for people living with HIV, is well tolerated. Although people living with HIV can control their disease with antiretroviral therapies, lifelong treatment is needed as reservoirs of HIV infected cells persist.

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Drug Discovery World

NOVEMBER 6, 2023

. “Fourteen percent of people living with NSCLC harbour the KRASG12C mutation yet there are limited targeted treatment options for patients with this devastating disease,” said Dr Shobhit Baijal, Consultant Medical Oncologist of The University Hospital Birmingham.

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Drug Discovery World

JUNE 20, 2024

UK researchers have designed a new genetic therapy that could alleviate debilitating giant moles in a rare skin condition. The team used a genetic therapy called silencing RNA, which blocks the action of the mutated NRAS in mole skin cells. The therapy was delivered directly to mole cells.

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Drug Discovery World

FEBRUARY 22, 2023

Sustained and durable effect The European Commission’s decision follows the CHMP’s positive opinion in December 2022, based on findings from the pivotal HOPE-B trial, the largest gene therapy trial in haemophilia B to date. The post EU-approved gene therapy reduced haemophilia bleeds by 64% appeared first on Drug Discovery World (DDW).

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SCIENMAG: Medicine & Health

JUNE 18, 2023

A three-year-long retrospective cohort study of a single Atlanta hospital’s patient population found transgender and gender-diverse teenagers rarely chose to discontinue gender-affirming hormone therapy, according to a study being presented Sunday at ENDO 2023, the Endocrine Society’s annual meeting in Chicago, Ill.

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Drug Discovery World

NOVEMBER 14, 2023

“Of the more than three million people with HeFH in the US and Europe, very few are currently at LDL-C goal, due in part to a care model that requires lifetime therapies. The post Single-course in vivo base editing therapy proven to lower cholesterol appeared first on Drug Discovery World (DDW).

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Drug Discovery World

OCTOBER 4, 2022

This transaction increases BioIVT’s global operations by adding new clinical collection sites, which are predominantly hospital based, focused on oncology, heme-oncology, and autoimmune cases,” said Dr Richard Haigh, BioIVT Chief Executive Officer (CEO).

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Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.

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Drug Discovery World

JUNE 25, 2024

The paper was co-authored by analytics company Phesi and Dr Yi-Bin Chen, Director for the Blood and Marrow Transplant Program at Massachusetts General Hospital. A new study demonstrates that AI-powered digital twin solutions can potentially replace the standard-of-care (SOC) control arm of a clinical trial.

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Drug Target Review

JULY 31, 2023

Our inaugural report is a groundbreaking exploration of the remarkable advancements in cell and gene therapy that are revolutionising the field of drug discovery. Expert Insights We are honoured to have collaborated with renowned experts in the field of cell and gene therapy, who have generously shared their invaluable insights.

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Fierce BioTech

AUGUST 31, 2023

How Continuous Safety Monitoring Can Reduce Hospital Stays for High-Risk Oncology Patients swheeler Thu, 08/31/2023 - 14:13 Fri, 10/06/2023 - 14:00 Resource Type Webinar Sridevi Rajeeve, MD Nicole Zahradka, PhD Duration 45 Minutes Cellular therapy products such as CAR-T show promising clinical efficacy in fighting cancer.

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Drug Discovery World

MARCH 13, 2023

The cell and gene therapy (CGT) landscape has grown significantly in the past year. Between the 24 therapies already approved by the FDA 1 and a marked increase in clinical trials, widespread accessibility to precision medicine feels within reach. One example: Zynteglo, a newly approved gene therapy priced at a record-breaking $2.8M

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KIF1A

APRIL 25, 2024

She is scheduled to be the 2nd patient in the world to receive ASO therapy for KAND. March 2023 – We met with n-Lorem and found out Susannah’s ASO (1st KAND patient to receive ASO therapy for KAND) would work for Sloane. Sloane is an adorable 3 year old with KAND. Hello KIF1A Community! We are also paying for our housing.

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DrugBank

JUNE 13, 2024

  Gene Therapy: Reprogramming the Body's Cellular Code    Gene therapy is an exciting field that treats diseases at their genetic roots.    A key part of gene therapy is efficiently delivering the therapeutic genetic material directly into target cells.

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