Gene Therapies

The drug maker said the program would allow for continued reimbursement when patients change insurance plans. A Sangamo-partnered gene therapy for hemophilia A is also in the works.

UK-based Ochre Bio has signed its first major deal with Boehringer Ingelheim. Scrip talked to its co-founder and CEO, Jack O’Meara, about its human tissue-based drug discovery platform, its resulting RNA platform for liver disease and how the fledgling drug company's early work mirrored that seen in diagnostics.

The company has pledged to continue developing its Duchenne in vivo gene editing therapy without Lilly, but will remain focused on its lead program in hepatitis B.

Merck & Co leads a market expected to exceed $10bn by 2028, but others are aiming to muscle in.

The company told Scrip that the sixth patient with elevated liver enzymes and low platelets recovered after hospital observation and oral steroids, and it may still continue VERVE-101’s development.

Orchard, which Kyowa Kirin bought in October, gave Lenmeldy a $4.25m list price, exceeding the upper bound of about $3.9m set by ICER.

As drug pricing continues its ascent among hot button issues, the multi-million-dollar price tags for one-and-done advanced therapies, their justification and reimbursement all remain unaligned.

The gene therapy company could have two products approved by the end of 2024 – with ambitions for long-term expansion beyond rare disease.

Analysts had forecast Q4 Roctavian sales of $5m-$6m, but they came in shy of $3m, and uptake has not yet come to reflect apparent demand.

The US group has had a tough year but the signs look more promising for isaralgagene civaparvovec as the FDA agrees on the design for a registrational trial for the gene therapy. Now Sangamo needs to secure a partner.

The Phase II LUNA study of ixo-vec showed a reduction in anti-VEGF use and stabilization of visual acuity and anatomic parameters that Adverum – and some analysts – called best in class.

The company claims its gene editing tech is the most potent yet – and is joining others in making established cardiovascular disease gene PCSK9 its first therapeutic target

More than 75 industry executives and experts gazed into the crystal ball and shared with Scrip their predictions around transformative technologies. Perhaps unsurprisingly, AI was the dominant theme – but gene editing, cell therapies and new targeting modalities were also among the highlights.

A Phase II study testing a next-generation exon skipping medicine vesleteplirsen in Duchenne muscular dystrophy amendable to exon 51 outpaced Sarepta’s Exondys 51.

In this week's podcast edition of Five Must-Know Things: Bayer’s pipeline refresh; J&J’s selective pharma M&A approach; silver lining for Gilead’s Trodelvy miss?; paying for obesity drugs; and the story behind Vertex and CRISPR’s gene therapy breakthrough. 

Looking ahead on manufacturing and supply-related issues in 2024, respondents told Scrip to expect companies to bring their supply chains closer to home, improvements for delivery of cell and gene therapies, a focus by contract manufacturers and developers on sustainability and continued emphasis on access to critical drugs.