Regenerative Medicine

Netherlands’s- based VectorY Therapeutics BV has negotiated an option and licensing agreement with a US manufacturer of adeno-associated viral (AAV) capsids ahead of making its first regulatory filings for clinical trials of a new drug for amyotrophic lateral sclerosis. The supplier of the tool is Shape Therapeutics Inc of Seattle, Washington which is developing capsids for genetic medicines for difficult-to-reach tissues. Capsids are the protein shells of AAV viruses that can be used to carry a working copy of a gene into the nucleus of a target cell. 

Enlaza Therapeutics Inc and Vertex Pharmaceuticals Inc agreed to collaborate on drug discovery aimed at creating small format drug conjugates and T cell engagers to treat certain autoimmune diseases and to improve conditioning in sickle cell disease and beta-thalassemia. Enlaza will receive $45 million in an upfront payment and equity investment. Vertex will work with Enlaza’s covalent biologics platform, called War-Lock, which uses proprietary non-natural amino acids to precision-engineer biologic medicines, resulting in an expanded therapeutic window. 

A gene therapy being developed by Beacon Therapeutics Holdings Ltd to treat X-linked retinitis pigmentosa has shown progress in two ongoing Phase 2 trials, SKYLINE and DAWN. The product, laru-zova, was generally well tolerated by participants evaluated in the 36-month SKYLINE trial and by participants in the nine months DAWN trial, showing improvements across key measures of visual function, including low luminance visual acuity and microperimetry.

A gene therapy being developed by Beacon Therapeutics Holdings Ltd to treat X-linked retinitis pigmentosa has shown progress in two ongoing Phase 2 trials, SKYLINE and DAWN. The product, laru-zova, was generally well tolerated by participants evaluated in the 36-month SKYLINE trial and by participants in the nine months DAWN trial, showing improvements across key measures of visual function, including low luminance visual acuity and microperimetry.

YolTech Therapeutics Co Ltd, a Shanghai, China-based gene therapy company, raised  approximately $45 million in a Series B financing led by the AstraZeneca-CICC healthcare investment fund. The proceeds will support the advancement of YolTech’s clinical programmes and its strategic development plans, the company said on 11 September. Yoltech, a developer of in vivo genome editing therapies, said it is advancing four clinical-stage programmes for:

UK-based Quell Therapeutics Ltd is to out-licence a candidate cell therapy for inflammatory bowel disease to AstraZeneca Plc following the achievement of a research milestone. Further details of the research goal were not disclosed. The two companies have been collaborating since 2023 in the development of engineered T-regulatory (Treg) cell therapies for two immune-mediated disease indications. Inflammatory bowel disease is one indication and Type 1 diabetes is the other. The achievement of the milestone will trigger a payment to Quell of $10 million.

The US Food and Drug Administration has relaxed some rules for chimeric antigen receptor (CAR) T cell therapies in order to minimise the tasks that healthcare providers are required to perform. From 26 June, rules known as Risk Evaluation and Mitigation Strategies (REMS) will be eliminated for six autologous CAR T cell therapies which are on the market for cancer indications. The therapies are directed against the CD19 and B-cell maturation antigens.

A new stem cell therapy has been recommended for conditional approval by the European Medicines Agency for patients with a blood cancer who need a transplant following chemotherapy and/or radiotherapy but have no suitable donors. The allogeneic product, Zemcelpro, is derived from umbilical cord blood and contains two components. These are expanded CD34+ cells and unexpanded CD34- cells, each derived from the same cord blood unit.

Eli Lilly and Co is to acquire Verve Therapeutics Inc of Boston, US, in a share transaction valued at $1 billion upfront in order to broaden its portfolio of treatments for people at risk of cardiovascular disease. Announced on 17 June, the deal comes two years after the companies entered a collaboration to explore the use of in vivo gene editing to develop therapies for genetic diseases. With a candidate medicine for artherosclerotic cardiovascular disease now in Phase 1b, the companies have decided to merge.

CRISPR base editing has delivered a landmark n-of-1 gene therapy for an infant with carbamoyl phosphate synthetase 1 (CPS1) deficiency, in a clinical milestone that may redefine how rare genetic diseases are treated. CPS1 deficiency is a mitochondrial disorder caused by a loss-of-function mutation in the CPS1 gene that impairs the urea cycle and leads to life-threatening hyperammonaemia.