It was a low-key week for the biotech sector with no major updates. Regulatory and pipeline updates from a few players were in focus.

Recap of the Week’s Most Important Stories:

Vertex, CRSP Gene Therapy Label Expansion: Vertex Pharmaceuticals VRTX and partner CRISPR Therapeutics CRSP announced that the FDA has expanded the label of their one-shot gene therapy Casgevy (exagamglogene autotemcel) to treat transfusion-dependent beta thalassemia (TDT) in patients aged 12 years and older. This approval comes ahead of the target action date in March. The gene therapy won FDA approval last month for sickle cell disease in the same age group. Consequently, Casgevy became the first gene therapy utilizing the Nobel prize-winning CRISPR technology. This technology can selectively delete, modify or correct a disease-causing abnormality in a specific DNA segment.

Vertex and CRISPR collaborated in 2015 to focus on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. Casgevy is the first treatment to emerge from the joint research program. Under an amended agreement, Vertex now leads the global development, manufacturing and commercialization of exa-cel and splits program costs and profits worldwide in the ratio of 60:40 with CRISPR.

Allakos Plunges on Updates:  Allakos ALLK plummeted after it announced that its two mid-stage studies evaluating its lead pipeline drug lirentelimab (AK002) in two inflammatory conditions were unsuccessful. Results from top-line data of the two studies — the phase II ATLAS study and phase IIb MAVERICK study — which evaluated lirentelimab in atopic dermatitis (AD) and chronic spontaneous urticaria (CSU) indications, respectively, failed to achieve their primary endpoints. Allakos will not pursue further clinical development of lirentelimab.

Concurrently, Allakos announced a restructuring program to reduce costs.  Allakos will reduce its workforce by 50% following the failure of lirentelimab in mid-stage studies. It will now focus on clinical development of AK006 along with additional preclinical programs. The reduction is expected to extend the company’s cash runway into mid-2026.

Solid’s Gene Therapy Gets Orphan Drug Tag: Solid Biosciences Inc. SLDB announced that the FDA has granted the orphan drug designation to its next-generational investigational candidate, SGT-003, which is being developed as a gene therapy for Duchenne muscular dystrophy (DMD). The orphan drug designation will grant Solid Biosciences market exclusivity for SGT-003 in the treatment of DMD for a predefined period, along with the exemption of FDA application fees and tax credits for qualified clinical studies, all subject to approval. The investigational gene therapy already enjoys the FDA’s fast-track designation in the United States for the treatment of DMD.