Regenerative Medicine

A venture capital syndicate has mobilised $141 million in Series B financing for an Italian biotech which is developing two gene therapies for retinal disorders. The company, AAVantgarde Bio Srl of Milan, is developing therapies for Stargardt disease and retinitis pigmentosa due to Usher syndrome type 1B. Both conditions are inherited retinal diseases for which there are no approved therapies. AAVantgarde is a spin-off of the Telethon Institute for Genetics and Medicine, an international research institute based in Naples.

Bristol Myers Squibb Co has stepped up its commitment to cell therapies with plans to spend $1.5 billion to acquire a Cambridge, Massachusetts biotech which is developing a medicine to reprogramme the immune system and treat autoimmune diseases. Orbital’s lead product, OTX-201, is an in vivo chimeric antigen receptor (CAR) T cell therapy that is being developed using ribonucleic acid (RNA) technology. The preclinical product consists of a circular RNA that encodes a CAR which targets cells expressing CD19, a B cell specific antigen.

Restructuring across the biotech industry has resulted in decisions by three companies to end programmes in cell therapy after investing heavily in the sector for several years. On 30 September, Heartseed Inc of Japan announced that partner Novo Nordisk A/S, was ending a collaboration to create a stem cell-based therapy for heart failure. In separate announcements on 1 October, Takeda Pharmaceutical Co Ltd of Japan and Galapagos NV of the Netherlands announced plans to discontinue or divest cell therapy progammes in cancer.

A gene therapy intended as a one-time treatment for Huntington’s disease significantly slowed progression of the disorder in a pivotal Phase 1/2 trial, paving the way for a regulatory submission in the first quarter of 2026. The developer, uniQure NV, showed that it is possible to safely impact the disease by targeting both the disease-causing huntingtin protein and the normal protein using a vector-based gene therapy and a gene encoding a microRNA (miRNA).

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.