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”

12 new medicines recommended for approvalEMA’s human medicines committee (CHMP) recommended 12 medicines for approval at its February 2026 meeting.The committee recommended…, CHMP statistics Key figures from the February 2026 CHMP meeting are represented in the graphic below. , CHMP statistics:…, Positive recommendations on new medici

Original title: “Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 23-26 February 2026”

EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Ojemda (tovorafenib) to treat patients aged 6 months and older with paediatric…

Original title: “New medicine to treat paediatric low-grade glioma”

On track to complete dosing in Embolden™ registrational trial of ... NEW YORK--(BUSINESS WIRE)-- Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the · U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to · NGN-401, an investigational gene therapy in late-stage clinical development as a potential best-in-class treatment ... On track to complete dosing in Embolden™ registrational trial of ... NEW YORK--(BUSINESS WIRE)-- Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the · U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to · NGN-401, an investigational gene therapy in late-stage clinical development as a potential best-in-class treatment for Rett syndrome. NGN-401 is the latest of several regulatory designations granted by FDA, including Regenerative Medicine Advanced Therapy (RMAT) and Rare Pediatric Disease designations, and selection for FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program. NGN-401 is being evaluated in the Embolden™ registrational clinical trial. Interim data from the Phase 1/2 trial (as of October 30, 2025) have shown that participants experienced multidomain, durable gains with continued skill acquisition observed over time, and · NGN-401 at the 1E15 vg dose has been generally well-tolerated. NGN-401 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations and selection for the START Pilot Program from the NGN-401; the potential for success of the Embolden registrational clinical trial of · NGN-401 for Rett Syndrome; the clinical benefit of delivering · NGN-401 via intracerebroventricular administration; and expected future interactions with or positions of the FDA, including the timing and outcome of any such interaction and anticipated benefits of any regulatory designation for · NGN-401, including the FDA's Breakthrough Therapy designation, Rare Pediatric Disease designation, RMAT designation, and participation in the FDA's START program.

Original title: “Neurogene gets FDA Breakthrough Therapy nod for NGN-401 | NGNE Stock News”

The U.S. Food and Drug Administration today issued an approval for the lung cancer drug Hernexeos (zongertinib) as a part of the new Commissioner's National Priority Voucher (CNPV) pilot program.

Original title: “FDA Grants Second Approval under the National Priority Voucher Pilot Program”

Orforglipron, which could be approved for obesity next quarter, proved superior at cutting blood sugar and body weight — but at the expense of higher side effect rates.

Original title: “Lilly’s GLP-1 pill tops Novo’s Rybelsus in head-to-head trial”

Ty Sperle says he felt “insane shock” after learning he’d been cured of a rare genetic disease through a clinical trial using a new gene-editing treatment.

Original title: “Rare disease cured by gene editing treatment”

Boehringer’s lung cancer medicine Hernexeos is the second “national priority” voucher winner to gain a speedy approval. Elsewhere, a landmark gene therapy was withdrawn and a Pfizer deal paid dividends.

Original title: “Boehringer drug approved in 44 days; BioMarin shelves Roctavian”

At the 2026 Conference on Retroviruses and Opportunistic Infections, Gilead and Merck demonstrated that their respective daily oral HIV drugs can match current therapies in keeping the virus at bay.

Original title: “Gilead, Merck Clear Path to Market for Daily HIV Pills With New Late-Stage Data”

Phase 2 study reports significantly accelerated debridement and wound repair in venous leg ulcers

Original title: “SolasCure trial shows faster healing with Aurase Wound Gel”

New phase 3 results highlight potential alternative for adults switching from complex regimens

Original title: “Gilead data show investigational HIV combination maintains viral suppression”

"It is really crazy to think about," says 18-year-old Ty Sperle. "We're definitely at a turning point in medicine." “It’s amazing to see a young person from B.C. cured of a life-threatening genetic disease through innovation from clinical teams,” she said. “This gives hope to families facing rare conditions and shows how smart investment in science can lead to life-changing care.” ... Sperle said he isn’t sure how to feel about being part of a groundbreaking moment in medicine. He is in his second year at UBC after taking half of last year off to travel to Montreal for the treatment. Ty Sperle, 18, is the first person to receive and be cured by a gene modification treatment for a rare disease called chronic granulomatous disease. The immunologist had been keeping up on advances in gene editing, so when American company Prime Medicine announced a clinical trial that was being run out of CHU Sainte-Justine in Montreal, he spoke to Dr. The disease impacts the immune system, specifically the white blood cells that engulf and destroy bacteria by releasing a substance similar to bleach. In someone with CGD, the cells don’t make the chemical, severely reducing the body’s ability to fight infection. ... “You can think of it like there was a big hole in his protective armour,” said Dr. Stuart Turvey, a pediatric immunologist at B.C. Children’s Hospital and a clinician at the B.C.

Original title: “B.C. teen first in world to be cured of rare disease through gene editing treatment | Vancouver Sun”

Sarepta to present new long-term ... slowing disease progression and supporting informed treatment decisions. ... U.S. Recommended Uniform Screening Panel (RUSP): The inclusion of Duchenne on the RUSP represents a pivotal advancement for the Duchenne community, encouraging broader newborn screening at the state level and empowering more families with early diagnosis and timely information to pursue earlier care, including access to available therapies and clinical trials... Sarepta to present new long-term and safety data across gene therapy and exon-skipping programs at 2026 Muscular Dystrophy Association Clinical & Scientific Conference: Sarepta will present new clinical and real‑world evidence at MDA 2026 showing long‑term efficacy, safety, and caregiver‑reported impact of its gene therapy and exon‑skipping treatments for Duchenne muscular dystrophy, reinforcing dystrophin’s role in slowing disease progression and supporting informed treatment decisions. ... U.S. Recommended Uniform Screening Panel (RUSP): The inclusion of Duchenne on the RUSP represents a pivotal advancement for the Duchenne community, encouraging broader newborn screening at the state level and empowering more families with early diagnosis and timely information to pursue earlier care, including access to available therapies and clinical trials. Net product revenue reached $1.86B in 2025 as Sarepta posted a $713M GAAP net loss but exited the year with $954M cash and expects 2026 profitability. At the same time, our PMO exon-skipping therapies continue to demonstrate durable clinical value, exceptional safety, extraordinary real‑world outcomes, and unwavering support from families and physicians. Finally, with five clinical‑stage RNAi programs and multiple readouts ahead, we are poised for meaningful growth while advancing therapies that can profoundly change the lives of patients living with rare disease.” Feb 4, 2026 Sarepta Therapeutics Announces Approval of Clinical Trial Application for SRP-1005, Its Investigational Treatment for Huntington’s Disease

Original title: “Inside Sarepta’s tumultuous $2.2B year in Duchenne gene therapy”

More than 200 participants enrolled as company targets topline data by end of 2026

Original title: “Verdiva Bio completes enrolment for phase 2b study of oral GLP‑1 candidate VRB‑101”

Oral therapy authorised for ER‑positive, HER2‑negative metastatic disease with ESR1 mutations

Original title: “MHRA approves imlunestrant tosylate as new breast cancer treatment”

A trial of a MacroGenics' drug temporarily paused enrollment after several safety events, including a fatality. The deceased patient had developed a severe case of neutropenia and concurrent septic shock.

Original title: “Patient Death Forces Partial Freeze on MacroGenics’ Gynecologic Cancer Study”

Following a disappointing Phase 3 performance and given Gossamer Bio’s balance sheet, seralutinib’s path to the market for pulmonary arterial hypertension has become unclear, according to analysts at Guggenheim Partners.

Original title: “Gossamer Nosedives as Late-Stage Failure Plunges PAH Program Into Uncertainty”

Erythromelalgia treatment gains regulatory support following EMA decision

Original title: “ACD440 granted orphan drug status in the EU”

Novo’s answer to a closely watched Lilly drug spurred roughly 20% weight loss after 24 weeks in a Phase 2 trial, results one of its executives claimed to be “differentiating.”

Original title: “Novo, searching for a spark, spotlights new data for three-pronged obesity drug”

PASO DOBLE sub‑analysis indicates metabolic differences between 2‑drug and 3‑drug HIV regimens

Original title: “ViiV Healthcare data show lower rates of steatotic liver disease after switching to Dovato”