July 10, 2024

The Dutch biotech said a higher dose of the gene therapy slowed progression by 80% compared to a matched control group, measured using the Unified Huntington’s Disease Rating Scale (cUHDRS), 24 months after it was dosed, in a pair of […]

July 9, 2024

Qarziba (dinutuximab beta) is on the market in 35 ex-US markets for the treatment of high-risk neuroblastoma in patients aged 12 months and above. It is sold by Recordati in all markets except for China, where BeiGene has rights, with both […]

July 8, 2024

Genezen, a gene therapy contract development and manufacturing organization (CDMO), and uniQure, a gene therapy company, announced on July 1, 2024 that they have entered into an agreement under which Genezen will acquire uniQure’s commercial gene therapy operations in Lexington, […]

July 8, 2024

Lilly is launching a tender offer to acquire all outstanding shares of Morphic for a purchase price of $57 per share in cash, prompting shares in the biotech to rocket 77% from $31.84 to $56.40 at the time of writing. […]

July 5, 2024

In a Securities & Exchange Commission (SEC) filing, Pieris said that Servier will exit the agreement between the two companies – first agreed in 2017 and focusing on the development of several Anticalin-based therapeutics for cancer – before the end of the […]

July 4, 2024

The award from the Biomedical Advanced Research and Development Authority (BARDA) will be used to accelerate the development of pandemic flu vaccines, according to Moderna, and specifically “support late-stage development for an mRNA-based vaccine to enable the licensure of a […]

July 3, 2024

The US regulator has cleared donanemab as Kisunla for use in patients with mild cognitive impairment or the mild dementia stage of Alzheimer’s with confirmed amyloid plaques, becoming the third drug in the class after Eisai/Biogen’s short-lived Aduhelm (aducanumab) and current rival Leqembi (lecanemab). The Alzheimer’s […]

July 3, 2024

The Series B – led by Forbion and joined by other investors, including Syncona, Oxford Science Enterprises, and Oxford University Innovation – takes the total raised by Beacon since its formation last year to $290 million, making it a rising […]

July 2, 2024

For patients with gout who cannot get relief using standard therapies, daily life can be a painful, debilitating experience. The disease is a painful form of inflammatory arthritis caused by elevated levels of uric acid in the blood resulting in […]

July 1, 2024

Like many of its cell therapy peers, Artiva reshuffled its strategy hoping to ride a recent wave of investor interest in autoimmune disease research. The company is one of a number of biotechs to emerge in recent years to develop […]

July 1, 2024

The Lexington plant is a central production hub for UniQure, which describes it as “one of the world’s leading, most versatile, gene therapy manufacturing facilities.” The company has for years pitched it as a competitive advantage in the field, affording […]

June 28, 2024

The agency’s positive opinion, which specifically applies to adults with severe or moderately severe haemophilia B without a history of factor IX (FIX) inhibitors, reverses its previous decision to reject the therapy. Affecting more than 2,000 people in the UK, […]

June 27, 2024

On June 24, 2024, FDA posted a conversation on its website with Yoni Tyberg, associate director of the Special Program Staff in the Office of New Drugs, regarding the progress of the modernization of the New Drug Regulatory Program (NDRP). […]

June 26, 2024

The analysis found that historical abuses in clinical trials and negative experiences patients may have experienced with their own medical care have generated a reluctance – particularly among communities with lower socioeconomic status – to participate in studies. One example […]

June 25, 2024

New Zealand-based contract manufacturing organisation Douglas Pharmaceuticals – which sponsored the 329-patient BEDROC trial and supplied the ketamine tablets – is now planning a phase 3 programme that could support regulatory filings. The results have been published in the journal Nature Medicine […]

June 24, 2024

The FDA has given accelerated approval to Krazati (adagrasib) as a combination with Eli Lilly’s anti-EGFR-antibody Erbitux (cetuximab) to treat patients with KRAS G12C-mutated, locally advanced, or metastatic CRC who have previously received chemotherapy. At the moment there are two KRAS inhibitors on the […]

June 24, 2024

The new data from Vertex’s phase 1/2 trial of VX-880, presented at the American Diabetes Association (ADA) annual congress in Florida, suggest that the therapy can restore the physiological activity of the insulin-producing islet cells of the pancreas and restore glucose […]

June 21, 2024

FDA published a final guidance document on June 18, 2024 that provides applicants with information on FDA’s policy for assigning a goal date for a facility’s inspection readiness as certified on Form FDA 356h, submitted as part of an original […]

June 20, 2024

Skyrizi (risankizumab) was previously already cleared by the FDA for adults with moderately to severely active Crohn’s disease, active psoriatic arthritis, and moderate to severe plaque psoriasis. The approval in UC is based on the results of a 12-week induction study called […]

June 20, 2024

The Food and Drug Administration has substantially loosened limits on the first gene therapy for Duchenne muscular dystrophy in a decision that could greatly expand its use even as questions remain about its effectiveness. The agency on Thursday made the therapy, called […]