July 24, 2025

The need-to-know this morning

Roche reported second-quarter earnings.
Rocket Pharmaceuticals announced a restructuring that will include layoffs affecting 30% of its employees. The company is reducing its cash burn by 25% to focus on gene therapies for cardiovascular conditions. It will no longer pursue the approval of a gene therapy for Fanconi anemia.

cancer

FDA delays GSK's Blenrep decision again

From my colleague Andrew Joseph: The wait for a Food and Drug Administration approval decision on GSK's blood cancer drug Blenrep is going to take a little longer.

The agency had been set to decide on Blenrep as a multiple myeloma treatment by yesterday, but the company that the FDA had extended the review period, with a new target decision date of Oct. 23. The move, GSK said, "provides the FDA with time to review additional information provided in support of the application."

"GSK is confident in the data supporting Blenrep combinations and looks forward to ongoing constructive conversations with the FDA as they continue their review," the company said.

FDA advisers last week concluded that the risks tied to the drug outweighed the benefits it had demonstrated in trials, with concerns focused on sometimes serious eye-related side effects. The agency often follows its advisers' recommendations, but does not have to.

policy

Alnylam CEO: The IRA needs a policy fix

A technical glitch in the Inflation Reduction Act is threatening the future of genetically targeted therapies, opines Alnylam CEO Yvonne Greenstreet. She's referring to a cutting-edge class of RNA- and DNA-based drugs designed to tackle the root causes of disease.

While biologics get 11 years before facing Medicare price negotiations, some genetic medicines are mistakenly classified as small-molecule drugs and given just seven, Greenstreet argues in a new First Opinion piece. This abbreviated window undermines the financial feasibility of these complex, costly treatments, she writes, potentially stalling progress on dozens of promising medicines.

A bipartisan bill called the MINI Act could fix this oversight by extending the exemption to 11 years. Passing it "would signal that Congress values innovation, supports patients, and is committed to fostering the next wave of medical breakthroughs," she writes.

gene editing

Tessera cuts jobs amid financial challenges

From my colleague Jason Mast: Tessera Therapeutics, the Flagship Pioneering gene-editing startup, laid off 17% of its staff this week, a spokesperson said.

It's Tessera's second layoff in the last year and a half, amid a continued downturn for the gene-editing field. The company has raised over $500 million but last announced a new round in April 2022.

It faces some of the same hurdles as other gene-editing firms — including challenges around delivery, indication-picking, and reimbursement, as well as a general sinking investor interest in new genetic technologies — alongside intellectual property questions, as its been accused of copying a competitor's technology. (Tessera denies having done so.)

The layoffs, the spokesperson said, will give Tessera the resources it needs as it pushes two different treatments toward clinical trials. Among other diseases, the company is developing gene-editing treatments for sickle cell disease and alpha 1 antitrypsin deficiency. In both cases, it is competing against other gene-editing companies, as well as companies developing other technologies.

genomics

'Landmark' study fills gaps in human genome

Scientists have decoded some of the most complex, previously unsequenced regions of the human genome, STAT's Veronica Paulus writes. This could potentially pave the way for more precise diagnoses and treatments.

"This is a landmark paper," one cell biologist not involved in the research told STAT. "It opens the door to potentially solving cases that have been inaccessible to diagnosis for a long time."

The study, published in Nature, mapped 92% of the remaining gaps using advanced sequencing technology from Oxford Nanopore and PacBio, and used a diverse set of 65 individuals from across 28 population groups.

Researchers fully resolved regions tied to diseases like type 2 diabetes, spinal muscular atrophy, and chromosomal disorders, while highlighting key structural variants — especially among individuals of African ancestry — that have long been overlooked in Eurocentric reference genomes.