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Pharmaceutical
Swiss pharma giant Novartis today announced an upgrade to its mid-term guidance, in advance of its Meet Novartis Management event for investors and analysts in London. 21 November 2024
Biotechnology
US biotech major Amgen has announced that Dr Howard Chang will join the company as senior vice president of research, effective December 16, 2024. 21 November 2024
Biotechnology
Australia’s CSL Limited plans to shut down its Californian R&D facility focused on cell and gene therapies by January 2025, signaling a shift away from ex vivo lentiviral-based technology. 19 November 2024
Biotechnology
Cellular immunotherapy specialist Fate Therapeutics soared as much as 73%, and closed up 12.45 at $2.27 yesterday, after it released early-stage data on its FT819 in lupus. 19 November 2024
Pharmaceutical
US antiviral giant Gilead Sciences on Friday unveiled two-and-a-half-year interim analysis from the ongoing Phase III ASSURE study of its Livdelzi (seladelpar). 16 November 2024
Pharmaceutical
French drugmaker Ipsen announced late-breaking data for Iqirvo (elafibranor 80mg tablets) from an interim analysis of the ongoing open-label extension of the Phase III ELATIVE study at the American Association for the Study of Liver Disease (AASLD) congress. 16 November 2024
Biotechnology
Oxford, UK-based rare respiratory diseases focused AlveoGene today revealed it has been granted a Rare Pediatric Disease designation (RPDD) by the US Food and Drug Administration (FDA) for AVG-002, its novel, inhaled gene therapy for lethal neonatal surfactant protein B (SP-B) deficiency. 15 November 2024
Biotechnology
Shares of US genetic meds company MeiraGTx Holdings were up 7% at $7.12 in early trading after it announced important developments along with its third-quarter 2024 financials, reporting a $0.54 per share loss. 13 November 2024
Biotechnology
Dutch firm Argenx and China’s Zai Lab have received an approval from the National Medical Products Administration (NMPA) for Vyvgart Hytrulo (efgartigimod alfa). 13 November 2024
Pharmaceutical
AstraZeneca and Merck & Co announced that their jointly developed drug Koselugo (selumetinib) has shown positive outcomes in the KOMET Phase III trial. 12 November 2024
Biotechnology
Geron Corporation, a biopharma company focused on telomerase inhibition therapies, has announced a $375 million financing agreement aimed at supporting the commercial rollout of Rytelo (imetelstat). 11 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program, according to Sarah Wicks and James Valentine at US law firm Hyman, Phelps & McNamara. 11 November 2024
Biotechnology
Sarepta Therapeutics has announced it will cease development of SRP-5051, an experimental drug for Duchenne muscular dystrophy, citing safety issues, feedback from the Food and Drug Administration, and the shifting Duchenne treatment landscape. 11 November 2024
Pharmaceutical
Research results featured heavily in the news last week, first with Viking Therapeutics releasing promising early-stage data for its investigational medicine VK2738 at the ObesityWeek meeting. US biotech Beam Therapeutics released early-stage data on its sickle cell disease candidate BEAM-101. Also of note, Arcus Biosciences and Gilead Sciences announced promising Phase III results for their TIGIT inhibitor domvanalimab in combination with zimberelimab. Denmark’s Novo Nordisk released financial results that attracted interest. 10 November 2024
Pharmaceutical
Italian pharma major Recordati saw its shares dip 3.75% to 50.10 euros this morning, despite posting a set of strong financial results. 8 November 2024
Biotechnology
Asklepios BioPharmaceutical (AskBio) today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). 7 November 2024
Biotechnology
The global market for Duchenne muscular dystrophy (DMD) therapeutics is projected to grow by $4.4 billion between 2024 and 2028, fueled by artificial intelligence and strong demand for new treatments. 7 November 2024
Pharmaceutical
US drugmaker Sarepta Therapeutic saw its shares gain 2.4% to $127.97 as it reported financial results for the third quarter 2024. 7 November 2024
Pharmaceutical
USA-based Acadia Pharmaceuticals has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million upon the closing of the transaction. 6 November 2024
Biotechnology
UK health technology assessor the National Institute for Health and Care Excellence (NICE) issued final guidance recommending reimbursement of Ayvakyt (avapritinib) from USA-based Blueprint Medicines. 6 November 2024
Biotechnology
A new antibody from Brussels-based UCB has been approved by the US Food and Drug Administration, for the treatment of certain adults with generalized myasthenia gravis (gMG). 27 June 2023
Biotechnology
Swiss pharma giant Roche will not begin any new trials of rugonersen (RO7248824) in Angelman syndrome, a rare genetic condition that affects the nervous system and causes severe physical and learning disabilities. 26 June 2023
Biotechnology
Japanese pharma major Takeda has presented positive interim results from a global Phase III trial of TAK-755. 26 June 2023
Pharmaceutical
The European Medicines Agency’s (EMA) human medicines committee (CHMP) recommended two novel medicines for approval at its June 2023 meeting. 23 June 2023
Biotechnology
The US Food and Drug Administration (FDA) yesterday approved Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for the treatment of pediatric patients aged four through five years of age with Duchenne muscular dystrophy (DMD). 23 June 2023
Biotechnology
The US Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for lovotibeglogene autotemcel (lovo-cel) for priority review, said the drug’s developer, bluebird bio. 22 June 2023
Biotechnology
uniQure lost 40% of its market value on Wednesday. 22 June 2023
Biotechnology
Promising clinical results from the 12-week portion of the PIVOT-HD Phase II study of PTC518 in Huntington's disease (HD) lifted US pharma group PTC Therapeutics. 22 June 2023
Pharmaceutical
The appointment of a new chief executive at immune-mediated disease specialist Veralox Therapeutics coincides with a fresh investment of $24 million from new and existing backers. 20 June 2023
Pharmaceutical
CDKL5 deficiency disorder (CDD), which has an incidence of 1 in 40,000 to 60,000 newborns, is a developmental disorder caused by either the presence of a non-functional cyclin-dependent kinase-like 5 (CDKL5) protein known as serine-threonine kinase (STK9), or its complete absence.1,2 20 June 2023
Biotechnology
Oxford University, UK, biotech spinout Evox Therapeutics has purchased USA-base Codiak Biosciences’ engEx-AAV technology platform, including all intellectual property rights therein, as well as secured exclusive access to certain associated engineering and manufacturing rights. 20 June 2023
Pharmaceutical
Shares of Santhera Pharmaceuticals were up almost 8% at 0.97 francs in early trading, after the Swiss rare diseases company revealed a lucrative licensing deal with Catalyst Pharmaceuticals for its vamorolone in the North America. 20 June 2023
Pharmaceutical
Texas, USA-based drug developer Reata Pharmaceuticals says that the US Food and Drug Administration (FDA) accepted for filing the Prior Approval Supplement (PAS) to update the drug substance specification for Skyclarys (omaveloxolone). 16 June 2023
Biotechnology
Japanese drugmaker Chugai Pharmaceutical said today it has filed a regulatory application with the Ministry of Health, Labor and Welfare (MHLW) for the anti-C5 antibody crovalimab for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). 14 June 2023
Biotechnology
Israel-based NeuroSense Therapeutics announced in a Securities and Exchange Commission that it has entered into a collaborative evaluation agreement with a unit of US biotech major Biogen. 14 June 2023
Pharmaceutical
Just months after France’s Ipsen bought liver disease specialist Albireo (Nasdaq: ALBO), the company’s main product Bylvay (odevixibat) has won a new US nod. 14 June 2023
Biotechnology
Soliris (eculizumab), from the AstraZeneca subsidiary Alexion, has won a new approval in China. 13 June 2023
Biotechnology
Shares of US gene and cell therapy company Abeona Therapeutics closed down 6.5% at $4.01 on Friday, when it announced receipt of feedback from the US Food and Drug Administration (FDA) regarding EB-101. 12 June 2023
Biotechnology
As usual, the American Society of Clinical Oncology (ASCO) meeting held last week attracted a great deal of attention, among which were presentations related to AstraZeneca’ Tagrisso in lung cancer and Blueprint Medicines new VELA trial of BLU-222 in breast and lung cancer. Outside of ASCO, Mereo BioPharma and partner Ultragenyx released new data showing their setrusumab induced rapid bone production in patients with osteogenesis imperfecta. US biotech FibroGen released disappointing Phase III result for its Duchenne muscular dystrophy (DMD) candidate pamrevlumab. Also of note last week, US pharma giant Merck & Co filed a lawsuit challenging the USA’s Inflation Reduction Act. 11 June 2023
Biotechnology
San Diego, USA-base next-gen gene therapy developer Kate Therapeutics (KateTx) has emerged from stealth mode with a $51 million Series A financing. 9 June 2023
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