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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.
In the rapidly advancing field of cell therapies, Dr Jason Bock has emerged as a leader, known for his innovative approach to optimising the development process. With over 25 years of experience in therapeutics, Bock has played a pivotal role in shaping the future of cell therapies, particularly through his work at CTMC.
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.
By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Below, we discuss some of these challenges in cell therapy trials.
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.
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
In the rapidly evolving field of cell and gene therapy (CGT), the ability to manage complex biological data, optimise manufacturing processes, and accelerate drug discovery is crucial. How AI is solving CGTs biggest challenges Cell and gene therapy is inherently complex. Faster time-to-market and reduced costs. The result?
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.
Although it is currently only used in veterinary therapy, carprofen proves to be a molecule with a versatile therapeutic potential for human pharmacotherapy. Moreover, the molecule is a target in the drug discovery process for the development of new bioactive compounds.
One analyst thinks the collaboration “bodes well” for Gossamer, which has had trouble convincing investors its drug seralutinib can compete on the market.
What distinguishes Alder Therapeutics’ approach to regenerative cell therapy development from traditional methods, and how does it aim to reduce risks in the preclinical phase? Traditional approaches to regenerative cell therapy development are defined by several challenges. For manufacturing, it’s no different.
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.
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.
While new gene therapies for the blood disease grabbed headlines at ASH, Pfizer presented fresh data for an oral drug meant to build on its marketedtherapy Oxbryta.
Elsewhere, Vertex got an FDA decision date for its “vanza triple” and Dupixent won a marketing green light in Europe for COPD. Forbion led the startup’s Series B round.
market, bladder cancer treatment Adstiladrin earned about $77 million. In its first full year on the U.S. Elsewhere, Lilly backed a cancer drug startup and Bristol Myers won a new Opdivo approval.
Commercializing Gene Therapies, Part 4 – Market Entry This paper outlines three basic market archetypes for gene therapies and the key considerations leaders must address when prioritizing new potential markets.
The announced job cuts are the latest in a series of steps Bluebird has taken to preserve cash and break even financially amid slow uptake of its marketed gene therapies.
In an era where biotherapeutics are transforming the healthcare landscape, the organisation, Vitalant , is uniquely positioned to bridge the gap between cutting-edge therapies and community access. Building new services and treatment options for patients are a passion for me.
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.
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.
As a result, biopharma and biotech companies working to bring their drug pipelines to the market require deep expertise from trusted partners to help deliver critical therapies for their patients. billion in 2023 to 1.2 billion in 2035.
The FDA’s clearance could double Kisqali’s breast cancer market. Elsewhere, Novo is exploring new ways to deliver genetic therapies and Bain is funding a growing CDMO.
The gene therapy developer’s demise has given Tectonic Therapeutic, a GPCR-focused startup founded by the prolific biotech entrepreneur, an alternate path to the public markets.
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.
Unlocking European market access for rare disease therapies The European orphan drug market presents a significant opportunity for biotech firms. Learn more about early access programs (EAPs), and how they are emerging as a critical strategy for successfully launching rare disease therapies in this complex and diverse market.
Accelerate customer speed to market With a modern and integrated user experience, AI solutions put the right data and insights into the right hands in real time. As a result, drug developers make better decisions more quickly (removing 50% of study timeline whitespace) to bring new therapies to market faster.
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).
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.
Oncology Market Outlook 2024 This paper assesses the current state of the oncology market, reviewing key expectations for 2024. It addresses CAR-T therapies, KRAS, radiopharmaceuticals, & targeted protein degradation. It addresses CAR-T therapies, KRAS, radiopharmaceuticals, & targeted protein degradation.
The agency needs more time to settle with Valneva the design of a post-marketing study for the shot, which could become the first preventive therapy for chikungunya available in the U.S.
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.
Addressing these underlying factors is essential; only by tackling the complexities of recruitment, timelines and regulatory compliance can sponsors achieve cost-effective and efficient trials, paving the way for long-term success in todays demanding market. Nearly 39% of sponsors cite these costs as primarily driven by complex protocols.
The antibiotic market has long faced significant barriers to innovation, with structural issues that make it difficult for new antibiotics to gain traction. As the demand for novel antibiotics remains limited due to the preference for cheaper generics, the market has struggled to incentivise much-needed breakthroughs.
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).
MDT helps bridge the gap in preference heterogeneity to ensure that rare disease therapies are developed with the diverse needs of patients in mind. Challenge #4: Regulatory and market access hurdles The regulatory and health technology assessment (HTA) pathways for rare disease therapies are complex and vary by region.
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.
Given the complexity and widespread impact of autoimmune and bone health conditions, developing new therapies is essential. These therapies represent a significant shift from traditional treatments, offering patients a higher chance of long-term symptom relief and improved quality of life.
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