Drug Discovery World

JULY 22, 2024

Keir Loiacono , CEO of BlueSphere Bio, discusses the goals of the company’s lead programme, the opportunities and potential of T-cell receptor (TCR) T cell therapies, and how to make oncology therapies tailored to individuals a reality. The goals of TCX-101 BlueSphere’s lead programme, TCX-101, includes two goals.

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Drug Discovery World

AUGUST 20, 2024

The company says the data support the potential clinical use of opaganib for the prevention and therapy of Type 2 diabetes and other obesity-related disorders. The studies showed the benefit of opaganib therapy in suppression of HFD-induced body weight gain, loss of glucose tolerance and fat deposition.

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Drug Discovery World

JULY 30, 2024

Tecvayli (teclistamab) has been recommended for relapsed and refractory multiple myeloma (RRMM) in England and Wales, leading analysts to predict Johnson & Johnson (J&J) will dominate the bispecific T-cell engagers (BiTEs) market by 2030. Talvey is estimated to become the second top-selling BiTE, with $2 billion.

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Drug Discovery World

JANUARY 18, 2024

Market opportunity estimated at over US$1 billion Largely due to the CRS risk (which can be life threatening), administration of cancer immunotherapies, such as bispecific antibodies, is currently restricted to specialist cancer centres which limits uptake of these therapies. million cases of cancer by 2030 1,2.

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Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The accelerated approval follows a vote of support from the FDA Cellular, Tissue and Gene Therapies Advisory Committee and the granting of priority review status.

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Drug Discovery World

JANUARY 30, 2024

Viral vector manufacturing At the Advanced Therapies Awards ceremony, Dr Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy, was awarded a Lifetime Achievement Award in recognition of ‘extraordinary contributions to gene therapy’, a field he has helped shape through his pioneering work on lentiviral vectors.

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Drug Discovery World

JULY 12, 2024

AB-1005 has also been awarded the innovative medicine designation, the Innovation Passport by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of Parkinson’s disease.

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Drug Discovery World

JUNE 1, 2023

According to NCBI, most of the ADCs developed so far are for use in the treatment of cancer, but there is plenty of potential for using ADCs to treat other diseases 1. A Nature publication confirmed that there are currently 12 FDA-approved ADCs on the market, and nine of these secured FDA approval in the past six years 2.

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Drug Discovery World

NOVEMBER 22, 2023

The European Commission (EC) has granted marketing authorisation for YORVIPATH (palopegteriparatide) as replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. YORVIPATH is Ascendis Pharma’s prodrug of parathyroid hormone (PTH 1-34), administered once daily.

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Drug Discovery World

MAY 20, 2024

Glucagon-like peptide-1 (GLP-1) receptor agonist sales for the type 2 diabetes and obesity markets are forecast to reach over $125 billion in the seven major markets (7MM) by 2033, according to analysts GlobalData. But there is an opportunity for new players to join the race, as the market is vast.

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Drug Discovery World

FEBRUARY 22, 2024

market capitalisation growth to $3.67 The top 20 global biopharmaceutical companies experienced varied year-on-year (YoY) market capitalisation shifts in 2023 amid the macroeconomic headwinds, steep patent cliffs and the commencement of US drug price negotiations under the Inflation Reduction Act (IRA). billion for 2023.

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Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

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Drug Discovery World

JULY 11, 2022

Biotechnology company Awakn Life Sciences has received funding from Innovate UK to help it advance its ketamine-assisted therapy programme to market in the UK and US. . Awakn Life Sciences has developed a ketamine-assisted therapy programme for treating alcohol use disorder (AUD). The addiction treatment market?opportunity

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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. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

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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. This means having GMP-trained operators and high-quality process development.

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Drug Discovery World

NOVEMBER 27, 2023

Hailed as a revolution in the treatment of many diseases, cell and gene therapy is the fastest growing area of therapeutics. The post New eBook: The latest innovations in cell and gene therapy appeared first on Drug Discovery World (DDW).

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Drug Discovery World

FEBRUARY 29, 2024

For Rare Disease Day 2024, Diana Spencer provides a round-up of the latest breakthroughs in the treatment of rare disease. Read more Funds advance gene therapy for cerebrotendinous xanthomatosis Vivet Therapeutics has received €4.9

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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

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

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

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

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Drug Discovery World

MAY 15, 2024

Dr Catherine Beech , CEO at Exonate and Dr Pete Adamson , CSO at Breye Therapeutics discuss the need for better therapies to treat diabetic retinal disease and current progress in the field. Despite affecting such a large subset of diabetic patients, treatment options are limited to repeated, invasive intravitreal injections to the eye.

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Drug Discovery World

APRIL 18, 2023

While the global proteasome inhibitors market is expected to reach nearly $2.3 billion by 2026, these drugs are primarily indicated for the treatment of multiple myeloma. ” The post Data shows new therapy successfully targets solid tumours appeared first on Drug Discovery World (DDW). .

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Drug Discovery World

FEBRUARY 21, 2024

Dr Mark Payton, CEO of Mercia, said: “The broader CGT market is a rapidly growing multi-billion-dollar market and its next stage of evolution is to focus down on technologies that reduce the cost of production and purification to ensure this therapeutic benefit is available for the many.

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Drug Discovery World

JANUARY 30, 2023

Register to attend Cell and gene therapy: sustaining growth and maximising opportunities, a free-to-attend DDW webinar, supported by Benchling and BPS Bioscience, on February 17, 2023 at 8am PST / 11am EST / 4pm GMT / 5pm CET. Cell and gene therapies (CGT) are transforming the way diseases are treated.

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Drug Discovery World

NOVEMBER 15, 2023

New trial evidence has shown that Kite’s CAR-T cell therapy Yescarta (axicabtagene ciloleucel: axi-cel) reduces the risk of death by 27.4% It is the only treatment in nearly 30 years to show statistically significant OS, with a median follow-up of 47.2 Type II variations require formal approval by the European Medicines Agency.

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Drug Discovery World

SEPTEMBER 14, 2023

Patients with mUC, including FGFR-driven tumours, face a particularly poor prognosis and the need for innovative therapies remains high. Patients with mUC, including FGFR-driven tumours, face a particularly poor prognosis and the need for innovative therapies remains high.

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Drug Discovery World

SEPTEMBER 4, 2024

Companies that obtain orphan designation benefit from protocol assistance, a type of scientific advice specific for designated orphan drugs, and market exclusivity once the drug is on the market. Fee reductions are also available depending on the status of the sponsor and the type of service required.

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Drug Discovery World

SEPTEMBER 28, 2023

The drug is currently being investigated in a pivotal study in cutaneous T-cell lymphoma (CTCL) as maintenance treatment by 4SC in Europe and by Yakult Honsha in Japan. Maintenance  Maintenance treatment – a unique innovative treatment approach in CTCL – is intended to prolong the period patients are stable and not progressing.

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Drug Discovery World

FEBRUARY 15, 2024

DDW Editor Reece Armstrong speaks to Tracy M Woody , President and Chief Executive Officer of Cessation Therapeutics, about the company’s goal to develop an antibody therapy to prevent fentanyl overdoses. RA: Naloxone is currently widely available on the market and can reverse an opioid overdose. Yet opioid deaths are high in the US.

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Drug Discovery World

JUNE 1, 2023

Using marketed treatments as a proxy, BI is expecting ex-US markets to have a 30-40% contribution within five years of launch. Shah added: “Unprecedented demand in obesity coupled with the strong relaunch of Wegovy, which serves as a benchmark for future launches, underpins our upgraded sales view for the market.

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Drug Discovery World

DECEMBER 19, 2023

Despite VMS, or hot flushes, being a common symptom of menopause, until now, hormone replacement therapy (HRT) was the only treatment. Veoza was also approved by the European Commission on 7 December 2023 at a dose of 45mg once daily for the treatment of moderate to severe VMS.

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Drug Discovery World

MAY 23, 2023

DDW’s Megan Thomas caught up with George White , General Manager, Product Management, Cell & Gene Therapy at Cytiva, to learn more about the company’s insight into the CGT market. Key challenges for cell and gene therapies In order for cell and gene (CGT) therapies to succeed, George White identifies several challenges to overcome.

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Drug Discovery World

NOVEMBER 21, 2022

Upstaza is the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency. It is also the first marketed gene therapy directly infused into the brain. The MHRA marketing authorisation is for treatment of patients 18 months and older. .

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Drug Discovery World

JULY 24, 2023

The EU Committee for Medicinal Products for Human Use (CHMP) has recommended the granting of conditional marketing authorisation for epcoritamab (Tepkinly) as a monotherapy for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.

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Drug Discovery World

MAY 16, 2023

What are the global innovations in cell and gene therapy? What opportunities and challenges are emerging and can therapies get to market faster? Curbishley explains that as the wave of new therapies in development continues to increase, global pressures on manufacturing availability will become more acute.

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Drug Discovery World

MAY 22, 2023

Ryan Leahy , a science communication expert in cell and gene therapy, at Phacilitate, says there is a lot to celebrate but also much to assess, question and challenge in this sector. Its subsequent success with approval in the US shows us that some drugs will be unavailable to some markets and patients for now.

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