Alkeus brought to late-stage testing a Stargardt disease therapy that former executive chairman and Vertex founder Josh Boger once called, “if it works, the most perfect drug I’ve ever seen.”

Original title: “Alkeus, an unorthodox eye drug startup, acquired by Tarsus for up to $800M”

Co-founded by longtime Alnylam CEO John Maraganore, City has brought a clotting disorder drug into early human testing. An experimental therapy for Stargardt disease could soon follow.

Original title: “City banks nearly $100M for next-gen RNAi drugs”

determinants of vision related quality of life in recessive stargardt disease

Original title: “Determinants of vision-related quality of life in recessive Stargardt disease.”

BIO-Europe Spring 2026 brought together more than 3,700 innovators in the life sciences industry all under the same roof. Another biotech at BIO-Europe Spring 2026 that was a part of the discussion was Milan-based AAVantgarde Bio, which scored $141 million in a series B funding round in November 2025 to fund two gene therapies AAVB-039 for Stargardt disease and AAVB-081 for Usher syndrome, both of which are rare diseases, the latter characterized by combined hearing and vision loss as well as balance issues. As a space for partnerships to forge, most, if not all the biotechs at BIO-Europe Spring 2026 were there seeking licensing deals and funding. While mergers and acquisitions (M&As) were part of the biopharma scene in 2025, albeit not thriving, the funding landscape in general has not been considered favourable over the past few years. Another biotech in the neurology space at BIO-Europe Spring 2026 was Prosperodes, but both its R&D and partnering goals were different from that of Vesper’s. To end the need for better biomarkers to diagnose neurodegenerative diseases, Prosperodes has built a technology platform over the past three years. Meanwhile, in women’s health and gynaecological care, Hungarian pharmaceutical company Gedeon Richter is eyeing innovation and collaborations to address female fertility, uterine fibroids, endometriosis, female contraception, infectious disease, and hormone replacement therapy (HRT). It has tapped Swiss biotech FimmCyte to potentially begin first-in-human trials to treat the root cause of endometriosis, a chronic, often painful disease where tissue similar to the lining of the uterus grows outside the uterus.

Original title: “BIO-Europe Spring 2026: partnership event brings funding hope to biotechs”

According to a recent LinkedIn post from AAVantgarde Bio, the company is using Rare Disease Day to spotlight the unmet needs of patients with rare inherited retinal... According to a recent LinkedIn post from AAVantgarde Bio, the company is using Rare Disease Day to spotlight the unmet needs of patients with rare inherited retinal diseases, or IRDs. The company’s LinkedIn post highlights that its sole focus is on rare diseases, with an emphasis on developing next‑generation gene therapies for large gene‑related IRDs such as Usher Syndrome type 1B and Stargardt disease. AAVantgarde Bio suggests it is pushing AAV vector technology to enable delivery of large genes, aiming to address the genetic root causes of targeted diseases and improve patient quality of life. For investors, this focus on technically challenging large‑gene delivery may position the company within a high‑risk, high‑reward segment of the gene therapy landscape, where successful proof‑of‑concept could be a differentiating asset. The post further frames Rare Disease Day as a reminder of the role of patients, families, clinicians, researchers, and advocates in driving progress, and it reiterates the company’s commitment to translating scientific innovation into meaningful outcomes. While the post does not provide concrete milestones, timelines, or financing details, its emphasis on underserved IRD populations may signal continued strategic focus on orphan indications that could qualify for regulatory incentives and premium pricing if therapies reach market.

Original title: “AAVantgarde Bio Emphasizes Gene Therapy Focus on Inherited Retinal Diseases - TipRanks.com”

Encelto uses a small, semi-permeable ... disease treatment.8 · VG801 (VeonGen Therapeutics, formerly ViGeneron) is a dual-AAV gene therapy developed to treat Stargardt disease. The therapy is under investigation in a phase 1/2 clinical trial. VG801 received FDA Rare Pediatric Disease ... Encelto uses a small, semi-permeable capsule implanted in the eye that contains allogeneic retinal pigment epithelium cells genetically engineered to produce specific therapeutic proteins for targeted disease treatment.8 · VG801 (VeonGen Therapeutics, formerly ViGeneron) is a dual-AAV gene therapy developed to treat Stargardt disease. The therapy is under investigation in a phase 1/2 clinical trial. VG801 received FDA Rare Pediatric Disease Designation and is progressing in a first-in-human phase 1/2 trial for Stargardt disease, with functional endpoint development underway through the FDA’s Rare Disease Endpoint Advancementprogram.9,10 Gene and cell therapies plus AI imaging tools accelerate breakthroughs for rare retinal diseases, spotlighting new trials for RP and Stargardt patients. HORA-PDE6b has demonstrated clinically meaningful benefits in visual function and a good safety profile in patients with PDE6b at the 24-month follow-up from the ongoing phase 1/2 trial.5 · MCO-010 (Nanoscope Therapeutics) demonstrated durable 3-year clinically meaningful vision improvements in the REMAIN Study for treating RP. REMAIN results showed sustained, three-line visual gains vs baseline through 152 weeks for MCO-010 in patients having severe vision loss from RP, and a favorable safety profile,6 according to the company press release. OCU410ST (Ocugen) is a possible treatment of ABCA4-associated retinopathies, including Stargardt disease, an inherited macular degeneration. AAVB-039 (AAVantgarde Bio), an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Stargardt disease, has received orphan drug designation from the FDA and clearance of a clinical trial application (CTA) from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).11 AAVB-039, which is currently being evaluated in the first-in-human, phase ½ CELESTE clinical trial (NCT07161544) in the US, is intended to deliver a functional copy of the disease-targeted ABCA4 gene.

Original title: “Rare Disease Day: An abundance of research Is underway to address rare eye diseases | Ophthalmology Times - Clinical Insights for Eye Specialists”