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

AUGUST 30, 2022

The Phacilitate Advanced Therapies Europe 2022 conference will take place from 30 August to 1 September at the Royal Lancaster in London, UK. The conference is the European twin to Phacilitate Advanced Therapies Week, an event held in January in Florida, US. Creating a sustainable cell & gene therapy company.

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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 Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

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Drug Discovery World

JANUARY 31, 2024

Breye Therapeutics, a clinical-stage biopharmaceutical company developing novel oral therapies for retinal vascular diseases within ophthalmology, has started a Phase Ib/IIa clinical trial to investigate danegaptide, following oral administration, in patients suffering from diabetic macular oedema (DMO).

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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, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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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). Nearly half of patients who do not receive treatment die within five years of symptom onset. Today, parents whose boys receive a CALD diagnosis can have renewed hope for the future.”.

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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. months and overall survival (OS) of 15.6

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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

MARCH 6, 2024

This second ODD allows research and development of a therapy for another form of the disease, Isolated Hypomyelination. The technology has been proven in the treatment of other dystrophies, including Duchenne muscular dystrophy, and is quick and cost-effective to develop.

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Drug Discovery World

MARCH 21, 2024

It found that patients with rare kidney diseases are 28 times more likely to experience kidney failure than those with chronic kidney disease (CKD), but are less than half as likely to die before kidney failure treatment is needed. Rare kidney diseases are a group of conditions that each affect fewer than one in 2,000 people.

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Drug Discovery World

AUGUST 4, 2023

Dr Christian K Schneider , Head of Biopharma Excellence at PharmaLex, presents the findings of an industry panel debate on barriers to commercialisation of novel treatments, and the most effective strategies to bring them to market. Other challenges involve talent gaps, particularly now that cell and gene therapy manufacturing is maturing.

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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. A lethal disease Prostate cancer is the most common cancer in men in Europe.

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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 The project is a phagogram using artificial intelligence (AI) that enables decentralised diagnostics and personalised treatment.

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Drug Discovery World

AUGUST 11, 2023

million into the fund, which will support the progression of new technologies and treatments for emerging viral threats and neglected tropical diseases. 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

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. The positive results of IMBrave050 address this huge and urgent unmet clinical need in HCC.”

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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. Phesi created a digital twin for chronic graft versus host disease (cGvHD) in the primary treatment setting.

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Drug Discovery World

NOVEMBER 6, 2023

The UK regulator has approved a new treatment for non-small cell lung cancer (NSCLC) in adults that has a mutation and produces a rare protein called KRAS G12C. “The expansion of treatment options for NSCLC benefits patients and clinicians alike.

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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 treatment could be used to reverse moles, and therefore prevent cancer. The team used a genetic therapy called silencing RNA, which blocks the action of the mutated NRAS in mole skin cells.

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KIF1A

MAY 29, 2024

Fortunately, Sloane does not currently have epilepsy, and Megan hopes this treatment will prevent it’s onset as her disease progresses. On the day of the treatment, they checked into the hospital where Sloane was administered sedation medication. The post Sloane’s 1st ASO treatment appeared first on KIF1A.

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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. Three additional patients are currently within the normal cognitive development range at nine to 18 months post-treatment, but require longer follow-up to assess outcomes.

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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. The bispecific soluble TCR therapy built on the company’s ImmTAX technology which is being developed for the treatment of people living with HIV (PLWH).

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Drug Discovery World

OCTOBER 10, 2022

A new clinical trial, led by the University of Glasgow and NHS Greater Glasgow and Clyde (NHSGGC), hopes to improve treatment options for patients with Crohn’s disease. . Unfortunately, no currently available treatment provides an effective solution for all patients.

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Drug Discovery World

NOVEMBER 14, 2023

Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments. Verve-101 is an investigational, in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower blood LDL-C. “Of

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Drug Discovery World

SEPTEMBER 15, 2022

A new combination therapy could represent a significant advancement in the treatment of metastatic colorectal cancer. million per year,” said Professor Josep Tabernero, Head of Medical Oncology, Vall d’Hebron University Hospital, Barcelona, Spain, and Primary Investigator for the SUNLIGHT trial. .

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Drug Discovery World

SEPTEMBER 13, 2022

The findings were presented at the European Society for Medical Oncology (ESMO) Congress 2022 by Professor Robin Jones, MD, Head of the Sarcoma Unit at The Royal Marsden Hospital, London. The post New treatment hope for patients with rare tumours appeared first on Drug Discovery World (DDW).

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Drug Target Review

JANUARY 23, 2024

What innovative approaches and technologies are anticipated to reshape immuno-oncology treatments in 2024? Immuno-oncology, notably through checkpoint inhibitors, has significantly reshaped cancer treatment over the past decade. Espen integrates clinical and research expertise as Director of Medical Affairs.

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Drug Discovery World

DECEMBER 16, 2022

The treatment of multiple myeloma becomes more complex with each relapse, so it is critical for frontline therapy to achieve deep treatment responses and extend survival,” said Edmond Chan, EMEA Therapeutic Area Lead Haematology, Janssen-Cilag Limited. Phase Ib MajesTEC-2 trial . of patients. . Phase III GLOW study .

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Drug Discovery World

SEPTEMBER 5, 2022

A pathway to better therapies. The findings suggest a pathway to better therapies for one of the least treatable forms of cancer. The problem is that these tumours respond to treatment initially, but then they come back. But the nice thing about this strategy is that it may work where current therapies have failed.

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Drug Discovery World

JULY 11, 2022

Pharmaceutical company Novo Nordisk has announced positive results for its prophylactic treatment targeting people living with haemophilia A or B with inhibitors. . The company’s concizumab treatment is an anti-tissue factor pathway inhibitor (TFPI)? The treatment of haemophilia is complex and no one treatment fits all, ” ?

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Drug Discovery World

OCTOBER 21, 2022

While advances in treatment over the last few years have resulted in good overall survival rates for patients with non-metastatic disease (90% five-year survival rate in the US 1 ), outcomes are still poor in certain cancers that do not respond to standard therapies. Advances in understanding treatment resistant cancer cells (part 1).

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DrugBank

JUNE 13, 2024

These innovative technologies are changing how medications reach their targets within the body, leading to more effective treatments with fewer side effects.   Take cancer treatment, for example. Implantable insulin pumps equipped with continuous glucose monitoring (CGM) sensors can update old diabetes treatment options.

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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. Her mother, Megan, has offered to share their experience and journey with everyone to help us understand the process and to share in their hopes, fears, challenges, and successes that come along with ASO treatment. Hello KIF1A Community!

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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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Drug Discovery World

AUGUST 2, 2023

Renaissance Pharma has announced its first development programme focused on Hu14.18, a humanised anti-GD2 monoclonal antibody (mAb), licensed from St Jude Children’s Research Hospital for the treatment of newly diagnosed high-risk neuroblastoma. A novel Phase II trial incorporating Hu14.18 and three-year event-free (EFS) of 73.7%.

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Drug Discovery World

APRIL 22, 2024

The treatment becomes only the second CAR-T cell therapy to secure routine funding for use in the NHS, and the only one for treating ALL in children and young adults under 26 years old. It is approved in cancers that are refractory, in relapse post-transplant or in second or later relapse.

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Drug Discovery World

JULY 13, 2022

The first new treatment in 20 years that prevents a common, painful side-effect in patients with sickle cell disorder (SCD) has been issued interim acceptance by the Scottish Medicines Consortium (SMC). . Many people with sickle cell disease experience recurrent painful crises which can result in hospital admission.

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