Today Stories

Sanofi announced today that the U.S. FDA has granted orphan drug designation to rilzabrutinib for the treatment of sickle cell disease, marking its fourth orphan status for this investigational Bruton's tyrosine kinase (BTK) inhibitor. The oral, reversible BTK inhibitor targets immune system modulation and aims to reduce vaso-occlusive crises—a major source of pain and complications in sickle cell patients. Rilzabrutinib is also under regulatory review in the US, EU, and China for immune thrombocytopenia (ITP), with an FDA decision expected by August 29, 2025. Previous designations include approvals for ITP, warm autoimmune hemolytic anemia (wAIHA), and IgG4-related disease. Preclinical data presented at ASH 2024 demonstrated rilzabrutinib’s potential to reduce inflammation and blood vesse…

The University of Hull is establishing a £48m internationally leading translational wound research centre in East Yorkshire.  With a consortium of industry partners, the Wound Innovation Centre (WIC) will be at the forefront of global wound research. Research carried out at the centre will transform the quality of life for those impacted by chronic wounds.  The University has been awarded £16m from the UK Research Partnership Investment Fund. The centre will be match funded by industry partners including Polaroid Therapeutics (PTx) and Reckitt, creating a £48m research facility in East Yorkshire.  Currently the NHS spends approximately £8.3 billion per year on wound care, more than both obesity and cancer. There is a real and increasing need for effective wound care treatment, regionally,…

Sanofi announces the completion of its acquisition of DR-0201, a targeted bispecific myeloid cell engager, from Dren Bio, Inc., a private clinical-stage biopharmaceutical company. The acquisition bolsters Sanofi’s ambition to become the foremost immunology company and broadens the company’s leading immunology pipeline. DR-0201, now named SAR448501, has shown robust B-cell depletion in pre-clinical and early clinical studies. The potential first-in-class targeted bispecific myeloid cell engager targets and engages specific tissue-resident and trafficking myeloid cells to induce deep B-cell depletion via targeted phagocytosis. Recent pre-clinical and early clinical study data in autoimmune diseases suggest that deep B-cell depletion has the potential to reset the adaptive immune system, lead…

21 May 2025 – Ramsey, Isle of Man – Juvenescence Ltd., a clinical-stage biotech leveraging AI to target age-related diseases, has announced the successful first close of its Series B-1 financing round, raising $76 million. The round was led by Abu Dhabi-based M42, which also enters a strategic partnership with Juvenescence to advance AI-driven therapeutics. The fresh capital will propel Juvenescence’s pipeline of age-related disease therapies into later-stage clinical trials, with the second tranche of the Series B-1 round expected to close in Q3 2025. This financing follows the announcement of a landmark collaboration between Juvenescence and M42, aimed at establishing a cutting-edge drug development hub in Abu Dhabi. The initiative will merge M42’s capabilities in digital health, genomic…

Paris, May 22, 2025. Sanofi announced today that it has entered into an agreement to acquire Vigil Neuroscience, Inc. (“Vigil”), a publicly traded clinical-stage biotechnology company focused on developing novel therapies for neurodegenerative diseases. This acquisition in neurology, one of Sanofi’s four strategic disease areas, enhances Sanofi’s early-stage pipeline and includes VG-3927, which will be evaluated in a phase 2 clinical study in Alzheimer’s disease. VG-3927 is an oral small molecule TREM2 agonist. Activating TREM2 is expected to enhance the neuroprotective function of microglia in Alzheimer’s disease. Houman Ashrafian, MD, PhD Head of Research and Development, Sanofi “This acquisition is fully supporting Sanofi’s strategic focus on neurology and on advancing science and lever…

Researchers at the University of Turku, Finland, have taken a step towards more personalised and effective cancer treatments. A new study has identified the conditions under which the antibody drug bexmarilimab can activate the body's own defence system against cancer, and how to identify patients who will benefit from the drug. Bexmarilimab has the ability to “wake up” macrophages, a type of immune cell, to act against cancer. The study found that this activation occurs especially when the tumour microenvironment is immunologically silent. Moreover, in the healthy tissue adjacent to the tumour, bexmarilimab was shown to trigger B cell–mediated immune responses, regardless of how sensitive the neighbouring tumour was to the treatment. This suggests that the drug may have immunological effe…

Mulhouse, France, April 20, 2025 – CellProthera, a regenerative cell therapy developer specializing in ischemic diseases, has chosen CELLforCURE by SEQENS, as its contract development and manufacturing organization (CDMO) partner for its planned Phase 3 trial for ProtheraCytes®, its autologous expanded CD34+ stem cell-based therapy aimed at improving heart failure event-free survival following a severe heart attack. CellProthera will tech transfer to CELLforCURE by SEQENS this year, with plans to produce clinical batches starting in 2026.  With promising results from a Phase 1/2b study of its lead ProtheraCytes®, CellProthera has begun preparations for its late stage of clinical development. Last year, the company presented its Phase 3 design to FDA and received favorable advice. Tech tran…

London, 15 May 2025 – Hikma Pharmaceuticals PLC (Hikma, Group), the multinational pharmaceutical group, is today hosting sell-side analysts and investors at its US manufacturing and R&D facility in Columbus, Ohio. The senior leadership teams from our Generics and Injectables segments will present on their respective businesses, strategies, and plans to deliver long-term, sustainable growth. Attendees will tour the Group's state-of-the-art, 1,000,000 sq. ft. facility in Columbus, which produces oral, respiratory and other generic and specialty products. It is one of Hikma's four US manufacturing facilities producing a broad range of medicines for patients across the US, where Hikma is one of the largest suppliers of generic medicines. Attendees will also tour the Company's newly acquire…

GSK to acquire efimosfermin, a phase III-ready potential best-in-class specialty medicine to treat and prevent progression of steatotic liver disease (SLD) GSK's acquisition of efimosfermin represents a significant strategic move for GSK in the hepatology space. The promising Phase II data and unique properties of efimosfermin, coupled with the substantial unmet medical need in SLD, position this asset as a potential new standard of care. The acquisition strengthens GSK's pipeline and offers opportunities for both monotherapy and combination approaches, with a potential launch in 2029. This investment underscores GSK's commitment to developing precision interventions for fibrotic liver diseases.

Simris Group's Q1 2025 report released today, highlights a period of significant focus on advancing their ADC payload development, particularly with promising early data for their microcystin variants. While revenue has decreased, operational efficiency shows improvement. The company is actively managing its financial position through various loan agreements and is streamlining operations through the sale of the Hammenhög facility. The positive preclinical data, coupled with the emphasis on further studies and optimisation, positions the company for potential future milestones in targeted cancer therapy. The exploration of broader applications and the strength of their IP portfolio also indicate potential for diversification and long-term growth.

Lundbeck has reported a strong start to 2025, leading the company to raise its full-year financial guidance. This performance is primarily driven by significant growth in its strategic brands, particularly Rexulti® and Vyepti®, across all key geographical regions. The company is also making substantial investments in its R&D pipeline, with key late-stage assets like bexicaserin and amlenetug progressing according to plan. While overall profitability is strong, reflected in increased EBITDA margins, the company is facing higher administrative and R&D costs, as well as increased net financial expenses due to recent acquisition-related debt. Legal proceedings and environmental remediation efforts continue to be managed.

FDA’s two designations demonstrate KER-0193’s potential as a treatment for FXS Follows the successful completion of Phase 1 clinical trial Fragile X syndrome is the most common cause of inherited autism London, UK, 14 May 2025 – Kaerus Bioscience (“Kaerus” or “the Company”), a clinical stage biopharmaceutical company created by Medicxi for the development of therapeutics for rare genetic syndromes of neurodevelopment, today announces that its lead candidate KER-0193 has been granted both Orphan Drug Designation and Rare Pediatric Drug Designations for the treatment of Fragile X syndrome (FXS) by the U.S. Food and Drugs Administration (FDA). It follows the recent successful completion of a Phase 1 trial of KER-0193 in healthy volunteers, which confirmed the drug to be safe, well tolerated…

Aviva Capital Partners (“ACP”), Aviva’s in-house capital unit that invests in a range of infrastructure and real estate projects, and mixed-use developer Socius have unveiled plans for a new £1 billion development to create the world’s leading centre for cancer research and treatment in Sutton, London.  A planning application has been submitted to London Borough of Sutton for the development which will be delivered on a 12-acre site at the London Cancer Hub, adjacent to The Institute of Cancer Research, London, one of the world’s leading cancer research organisations, and The Royal Marsden NHS Foundation Trust’s Sutton site, Europe’s leading centre for cancer treatment. Working with London Borough of Sutton as the landowner, ACP and Socius plan to deliver c. 1 million sq ft of state-of-the…

France, 30 April 2025 – It is official: Opella is now a standalone company. Sanofi announced today the closing of the sale to CD&R of a 50.0% controlling stake of Opella, Sanofi keeping a significant shareholding with a 48.2% stake and Bpifrance owning a 1.8% stake. Opella is stepping into its next chapter with powerful backing, and a clear mission - “Health in Your Hands” - making self-care as simple as it should be.This is not just a change of ownership. This is a bold move. As the purest and third-largest global player in the €190 billion Over-The-Counter and Vitamins, Minerals and Supplements space, Opella is set to lead in one of the most dynamic,  resilient corners of healthcare – where megatrends like aging populations, digital access, and self-care demand are rewriting the rule…

Asimov, the synthetic biology company advancing the design and manufacture of therapeutics, is collaborating with Cytiva, a global life sciences leader, to provide customers with an integrated offering for optimized biologics production. The new offering will provide seamless access to Asimov’s CHO Edge cell line development platform, Cytiva's HyClone media and feed solutions, and Cytiva’s Fast Trak process development services. Together, these services and technologies enable the expression and development of protein biologics, including complex modalities, that hold the potential to significantly impact human health.  Alec Nielsen, co-founder and CEO of Asimov, says: “Central to this collaboration is the extensive optimization of our CHO Edge System in Cytiva’s HyClone media and feed, en…

Lund, Sweden, 24 April 2025. Hansa Biopharma AB, “Hansa” (Nasdaq Stockholm: HNSA) today announced the appointment of Renée Aguiar-Lucander as Chief Executive Officer (CEO), effective immediately. Søren Tulstrup will be stepping down from his position by mutual agreement as CEO after seven years of dedicated service to the Company. “On behalf of the Board of Directors, I would like to extend our gratitude to Søren for his leadership and significant contribution to the company during his tenure. Under his guidance, Hansa has evolved from a clinical-stage company into a commercial-stage biopharmaceutical organization, navigating both transformative progress and substantial challenges. We deeply appreciate Søren’s commitment to the Company and wish him every success in his future endeavors,” s…

In-licensing transforms Mosaic from a research and platform company to a clinical-stage oncology business Mosaic will develop proprietary, targeted combination products, supported by proprietary biomarkers, to realise the full potential of the assets As part of the agreement Astex has taken an equity stake in Mosaic Cambridge, UK, 24 April 2025 – Mosaic Therapeutics, Ltd (‘Mosaic’, or ‘the Company’) a targeted oncology therapeutics company dedicated to resolving cancer’s complexity with new combination therapies for patients, today announces the in-licensing of two clinical-stage oncology programs from Astex Pharmaceuticals (‘Astex’), a wholly owned subsidiary of Otsuka Pharmaceutical Co. Ltd. Mosaic has identified proprietary combinations incorporating the licensed products and their ta…

A new report reveals disparities in chimeric antigen receptor (CAR) T-cell therapy delivery across Europe, with healthcare system utilisation rates of the therapy as low as 11% for eligible patients.  The independent research ‘Achieving CAR T-Cell Therapy Health System Readiness: An assessment of barriers and opportunities’ by the IQVIA Institute for Human Data Science, funded by Gilead and Kite, highlights challenges across countries that can lead to uneven access to CAR T-cell therapy. The report, which assessed seven countries (Australia, Canada, France, Germany, Italy, Spain, the United Kingdom), found that factors such as complex referral pathways, limited treatment centers and reimbursement challenges are hindering access to this potentially life-saving treatment for eligible patient…

Roche has announced a $50 billion investment in the United States over the next five years, aiming to create over 12,000 jobs. This strategic move is in response to potential tariffs from the Trump administration targeting Swiss pharmaceutical imports. The investment will expand manufacturing and distribution centers in Kentucky, Indiana, New Jersey, and California. New facilities include a gene therapy plant in Pennsylvania, a continuous glucose monitoring site in Indiana, a weight loss drug factory at an undisclosed location, and a cardiovascular, renal, and metabolism research center in Massachusetts. CEO Thomas Schinecker emphasized that this initiative will position Roche to export more medicines from the U.S. than it imports, reinforcing the company's commitment to U.S. innovation an…

Jeanette Rigby is Director Human Resources at Valneva Scotland Ltd Scotland’s life sciences sector is a crucial part of the nation’s economy, contributing groundbreaking innovation in areas such as biotechnology, pharmaceuticals, and vaccine manufacture. Recent data shows the sector now contributes almost £10.5bn to the Scottish economy, with life sciences exports worth £4.3bn, and the potential is building. Its strong academic foundation, coupled with a robust talent pool and the Scottish Government’s life sciences strategy, makes Scotland a prime destination for innovation and investment. However, if we are to remain competitive globally, Scottish companies must invest in their people. Read the full article here