Drug Target Review

NOVEMBER 8, 2023

Problem w/ CTs and foundational understanding of Vittoria: can you explore the current limitations of cell therapies and the challenges faced by patients and providers? Currently, only a small percentage of cancers can be effectively treated with cell therapies, and there is little diversity in the currently approved products.

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PPD

OCTOBER 17, 2024

Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.

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BioPharma Drive: Drug Pricing

OCTOBER 5, 2023

Kyowa will pay nearly $400 million to acquire Orchard, which sells the gene therapy Libmeldy in Europe but has struggled to find paths to market for other experimental treatments.

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PLOS: DNA Science

MARCH 21, 2024

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. She passed away in 2013.

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Drug Target Review

SEPTEMBER 6, 2024

What trends are driving the increased use of in vivo delivery methods in gene therapy? How is the market demand for lentiviral vectors evolving, and what factors are influencing this growth? There is a very significant increased market demand for lentiviral vectors. New therapeutic areas are emerging in a powerful way.

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The Premier Consulting Blog

NOVEMBER 12, 2024

Recently, we convened a panel of national payer pharmacy decision-makers to ask them about how they evaluate 505(b)(2)s and whether an approved FDA label is enough to secure strong payer market access. This blog is the first installment in a series summarizing our findings. Non-preferred coverage.

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ASPET

OCTOBER 20, 2023

However, Pramlintide an adjunct to insulin therapy, enhances glucagon suppression and thereby offers improved glycaemic control. Although clinical benefits of Pramlintide are well reported, there still exists a high patient resistance for the therapy as separate injections for Pramlintide and insulin are required to be administered.

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Fierce BioTech

JULY 17, 2024

Accelerate your cell therapy to clinic with a flexible CGMP-ready process The cell therapy market holds great promise, yet faces challenges like material variability and the need for standardization. Download this whitepaper to discover how to de-risk these challenges and meet the evolving needs of the cell therapy market.

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Fierce BioTech

JUNE 13, 2024

Discover how this innovative digital design system is helping increase patient medication adherence and the length of therapies, by moving beyond segmentation to truly personalized experiences. Listing Image AllazoHealth_ListingLogo_250x190.png On Demand Start Date Wed, 08/07/2024 - 12:00

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BioPharma Drive: Drug Pricing

JUNE 27, 2023

The treatment could become Pfizer’s first marketed gene therapy, an area the pharma has poured significant resources into in recent years.

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The Pharma Data

JUNE 22, 2023

Kite Announces Completion of Marketing Authorization Transfer for Yescarta® CAR T-cell Therapy in Japan SANTA MONICA, Calif.–(BUSINESS in December 2022 about changes to their initial 2017 partnership whereby Daiichi Sankyo obtained the Marketing Authorization for Yescarta ®. to Gilead Sciences K.K.,

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Drug Channels

MARCH 28, 2024

Bill discusses specialty therapy access barriers, including payer utilization management techniques and “no coverage” policies. Today’s guest post comes from Bill Dupere, SVP of Product Development & Partnerships at Mercalis. Contact Mercalis to learn about their Patient Support Services. Read on for Bill’s insights.

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Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. In some cases, cell therapy can be curative for the patient.

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BioPharma Drive: Drug Pricing

DECEMBER 6, 2023

The funds are meant to boost Fujifilm’s capacity to manufacture cell therapies, a market it expects to grow substantially in the coming years.

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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 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 Patent Watch

JANUARY 21, 2021

The post Market access of gene therapies across Europe, USA, and Canada: challenges, trends, and solutions appeared first on DrugPatentWatch - Make Better Decisions. Highlights….

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Agency IQ

NOVEMBER 3, 2023

Probiotics, a type of microbiome-based therapy containing live micro-organisms, are broadly available on drug store and grocery store shelves, but the regulatory classifications for products containing the same micro-organism can differ dramatically. Fill out the form to read the full article.

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Fierce BioTech

OCTOBER 5, 2023

Discover Strategies for Combatting Disruptions in Gene Therapy Development Cell and gene therapy development has exploded, with Q4 2022 showing more FDA approvals than over the past five years combined.[1] 1] How can sponsors keep pace with this rapidly growing market and avoid costly delays that can threaten critical timelines?

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Common Sense for Drug Policy Blog

JUNE 6, 2024

The Market for Image and Performance Enhancing Drugs (IPEDs) "The market for image and performance enhancing drugs has undergone seismic changes in the last two decades and, as has been alluded to above, its partial digitisation has created something of a dual space of commerce (Gibbs, Forthcoming ). Fincoeur et al.

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Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.

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Advarra

JUNE 13, 2023

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% As more therapies are moving out of the lab and into human clinical trials, this paradigm is changing.

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PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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LifeSciVC

NOVEMBER 28, 2023

Market conditions like this are demoralizing in the worst way. Furthermore, to make matters even more complicated, that same summer, markets had already turned sour, and the sentiment was darkening by the day. The biotech equity markets had effectively shut off the idea of an IPO (which was one of several paths we had mapped out).

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Advarra

MARCH 20, 2023

The Food and Drug Administration (FDA) recently released new guidance regarding cellular and gene therapy products, one of which may significantly impact early-phase clinical trials of such products. This article explores the implications of this guidance, including new approaches for studies focused on cellular or gene therapies.

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ASPET

OCTOBER 12, 2023

The CET RAIDR program conserves both time-to-market and funds by leveraging previous conventional development work as a launch point for repurposing efforts. CBRN MCMs).

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BioPharma Drive: Drug Pricing

MARCH 20, 2024

million list price, the highest of any genetic medicine to come to market. Orchard is counting on the long-term data it’s accrued to convince insurers to cover Lenmeldy’s $4.25

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ASPET

SEPTEMBER 7, 2023

Avalglucosidase alfa has received marketing authorization in several countries for infantile-onset and/or late-onset Pompe disease. This recently approved enzyme replacement therapy (ERT) was glycoengineered to maximize CIMPR binding through high-affinity interactions with ~7 bis-M6P moieties.

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The Pharma Data

SEPTEMBER 10, 2021

– Abrocitinib is a once-daily oral JAK1 inhibitor indicated in Great Britain for the treatment of moderate to severe atopic dermatitis in patients aged 12 years and over, who are candidates for systemic therapy-. This is the first marketing authorization globally for abrocitinib-.

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DS in Pharmatics

JUNE 10, 2023

On April 12, 2023, BeNeLuxA1 announced that negotiations failed to secure reimbursement for Libmeldy, a gene therapy developed by Orchard Therapeutics for the treatment of metachromatic leukodystrophy, on grounds of price. In the UK, an initial application to NICE was rejected as well back in 2020. This is a huge […]

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BioPharma Drive: Drug Pricing

JULY 3, 2024

Multibillion-dollar buyouts from Bristol Myers Squibb and Gilead could yield new drugs for brain and liver diseases, while a new cell therapy may reach market.

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Drug Target Review

AUGUST 6, 2024

Liver Fibrosis : Targeted therapies using ADCs could help reduce liver fibrosis and prevent the progression to cirrhosis. Expedited programs like the FDA’s Breakthrough Therapy Designation and Fast Track can shorten development timelines and reduce risk. Oncology drugs often command high prices, potentially leading to higher ROI.

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Codon

APRIL 9, 2023

” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. Read Ocular stress enhances contralateral transfer of lenadogene nolparvovec gene therapy through astrocyte networks. Molecular Therapy. Gene Therapy. McGrady N.R.

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BioPharma Drive: Drug Pricing

MAY 13, 2024

Goldman Sachs and Eli Lilly are among those backing the startup, which claims its prospect could be more potent than the myelofibrosis therapies that have come to market in recent years.

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LifeSciVC

OCTOBER 13, 2022

This summer, approvals on both sides of the Atlantic – for beta thalassemia and cerebral adrenoleukodystrophy in the United States and for severe hemophilia A and aromatic L-amino acid carboxylase deficiency in Europe – show that regulators are likely open to green-lighting a range of emerging gene therapies.

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BioPharma Drive: Drug Pricing

MAY 6, 2024

One analyst thinks the collaboration “bodes well” for Gossamer, which has had trouble convincing investors its drug seralutinib can compete on the market.

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BioPharma Drive: Drug Pricing

OCTOBER 23, 2024

Elsewhere, Sangamo plotted a much faster path to market for its Fabry gene therapy. Yale spinout Modifi agreed to sell to Merck for $30 million upfront.

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