In this randomized phase 3 trial, patients with treatment-naive stage III–IV nonsmall cell lung cancer who received sintilimab or pembrolizumab in combination with chemotherapy early in the day (before 15:00 h) experienced longer progression-free survival compared with those receiving late ... In this randomized phase 3 trial, patients with treatment-naive stage III–IV nonsmall cell lung cancer who received sintilimab or pembrolizumab in combination with chemotherapy early in the day (before 15:00 h) experienced longer progression-free survival compared with those receiving late time-of-day infusions. Retrospective studies suggest that early time-of-day (ToD) infusions of immunochemotherapy may improve efficacy. However, prospective randomized controlled trials are needed to validate it. In this randomized phase 3 LungTIME-C01 trial, 210 patients with treatment naive stage IIIC–IV nonsmall cell lung cancer (NSCLC) lacking driver mutations were randomly assigned in a 1:1 ratio to either an early or late ToD group, defined by the administration of the first four cycles of an anti-PD-1 agent before or after 15:00 h. The primary endpoint was progression-free survival (PFS), while secondary endpoints included overall survival (OS) and objective response rate (ORR). Nature Medicine - In this randomized phase 3 trial, patients with treatment-naive stage III–IV nonsmall cell lung cancer who received sintilimab or pembrolizumab in combination with... Extended Data Table 1 Information about the clinical trial ... Extended Data Table 4 Baseline demographics and disease characteristics of patients included in the flow cytometry analysis by study group

Original title: “Time-of-day immunochemotherapy in nonsmall cell lung cancer: a randomized phase 3 trial | Nature Medicine”

Patients with rare cancers, including aggressive and hard‑to‑treat brain cancers, will benefit from faster access to breakthrough treatments under ...

Original title: “Transforming rare cancer treatment and clinical trials access”

The EMA’s positive opinion on Kygevvi marks a turning point for patients with TK2d, an ultra-rare genetic muscle disease previously managed only with supportive care. TK2 deficiency is a rare genetic disorder affecting fewer than 1 in 1,000,000 people and represents a rare, life-threatening genetic disorder with no previously authorized therapy in Europe. Worldwide clinical experience has been limited, and until now there have been no disease-modifying treatments approved anywhere to address the underlying cause beyond supportive care. The recommendation relied on data from a retrospective chart review study (MT-1621-101) and a phase 2 single-arm clinical study (TKO102), involving 39 patients with TK2d whose disease onset occurred by age 12. The effect of the therapy on motor function was assessed by comparing motor milestones before and after therapy in each patient. This recommendation, if followed by the European Commission’s approval, would make Kygevvi the first therapy authorized in the European Union specifically for TK2d, an ultra-rare, life-threatening genetic mitochondrial disorder characterized by progressive muscle wasting and respiratory failure. The positive CHMP opinion covers both pediatric and adult patients whose disease symptoms began by age 12. Cite this: Edited by Zeel Mehta. EMA Backs First Therapy for Rare Genetic Muscle Disease - Medscape - February 02, 2026.

Original title: “EMA Backs First Therapy for Rare Genetic Muscle Disease”

The cancer case was reported in ... for the rare enzyme deficiency Hurler syndrome, Regenxbio announced on Jan. 28. The inherited disease results in the buildup of compounds in tissue, leading to organ dysfunction and central nervous system problems. Enzyme replacement therapy is a standard treatment, but engineered enzymes do not cross the blood brain barrier. RGX-111 delivers a gene to central nervous system cells to bring ... The cancer case was reported in a Phase 1/2 test of RGX-111, a potential treatment for the rare enzyme deficiency Hurler syndrome, Regenxbio announced on Jan. 28. The inherited disease results in the buildup of compounds in tissue, leading to organ dysfunction and central nervous system problems. Enzyme replacement therapy is a standard treatment, but engineered enzymes do not cross the blood brain barrier. RGX-111 delivers a gene to central nervous system cells to bring production of the deficient enzyme to the CNS. Tests of two Regenxbio gene therapies have been placed under an FDA clinical hold after a patient in one of the studies developed cancer. Our recap of recent regulatory news also includes one clinical hold removed, several complete response letters, and drug approvals in the U.S. and Europe. —The ultra-rare inherited neurodegenerative disorder Menkes disease has its first FDA-approved therapy, a drug developed by Sentynl Therapeutics. In Menkes, a genetic defect impairs the ability to absorb and transport copper, which is essential for certain bodily processes. —Intellia Therapeutics has FDA permission to resume Phase 3 testing of the gene therapy nexiguran ziclumeran (nex-z) as a potential treatment for polyneuropathy associated with the rare disease hereditary transthyretin amyloidosis (ATTR). Last October, the agency placed a clinical hold on this study and a separate Phase 3 test in ATTR cardiomyopathy after a patient in the cardiomyopathy study developed liver complications.

Original title: “Clinical Trial Holds Cast Doubt on Approval Chances of Regenxbio Gene Therapy for Rare Disease - MedCity News”

The decision comes more than a year after disappointing initial Phase 3 results and casts further doubt on a class of autoimmune drugs thought to be challengers to treatments like Dupixent.

Original title: “Amgen gives up on its once-prized eczema drug”

EMA has recommended granting a marketing authorisation in the European Union for Kygevvi (doxecitine and doxribtimine) in patients with genetically confirmed thymidine kinase 2…

Original title: “First treatment for rare thymidine kinase 2 deficiency”

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

Original title: “Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 26-29 January 2026”

EMA has recommended extending the therapeutic indication of Zynyz (retifanlimab) to include treatment of adults with squamous cell carcinoma of the anal canal (SCAC) that cannot…

Original title: “First immunotherapy-based treatment recommended for advanced anal cancer”

CER‑T therapy shows initial safety signals and prompts protocol expansion

Original title: “CERo reports encouraging early data from phase 1 trial of CER‑1236”

Trial to assess whether once‑weekly treatment can reduce symptomatic viral respiratory infections

Original title: “ENA Respiratory begins dosing in phase 2 study of INNA‑051 nasal spray”

Conditions: Recurrent CNS Tumors; Brain Cancer; Gliomas; IDH-wildtype Gliomas; IDH-mutant Gliomas; Rare CNS Tumor Interventions: Other: Tumor sample collection Sponsors: National Cancer Institute (NCI) Not yet recruiting

Original title: “Observational Study of Responses to Treatments in Advanced Central Nervous System (CNS) Tumors”

A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy. A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy. ... The clinical hold has been placed on trials of both RGX-111 and RGX-121. RGX-121 presents an opportunity to address the urgent, significant unmet medical need in this ultra-rare disease community, and continued delay means continued neurodevelopmental decline in boys with MPS II.” · REGENXBIO added that it has not yet received the full clinical hold letter and awaits additional details from the FDA. The company’s stock price fell 28.4% on the update; from a 27 January close of $13.41 to a 28 January open of $9.60. The company has a market cap of $566m. This news came on the same day that Intellia Therapeutics had a clinical hold of its gene therapy nex-z lifted after a patient died in a Phase III trial. The US Food and Drug Administration (FDA) has placed a clinical hold on trials of two of REGENXBIO’s investigational gene therapies, for the treatment of mucopolysaccharidosis (MPS) type I, after a patient dosed was diagnosed with a brain tumour. The hold has been lifted in the MAGNITUDE-2 trial but remains in place on the MAGNITUDE study. Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.

Original title: “FDA puts REGENXBIO gene therapy trials on hold after brain tumour”

Jan 28 (Reuters) - Regenxbio (RGNX.O), ... for rare childhood diseases, sending its shares plummeting 30% premarket. The company's experimental gene therapy RGX-111 was placed on clinical hold after a brain tumor was found in one trial participant, the company said. Sign up here. The tumor was discovered during a routine brain scan of a five-year-old child who had received the treatment about four ... Jan 28 (Reuters) - Regenxbio (RGNX.O), opens new tab said on Wednesday the U.S. Food and Drug Administration has placed a clinical hold on two of its experimental gene therapy programs for rare childhood diseases, sending its shares plummeting 30% premarket. The company's experimental gene therapy RGX-111 was placed on clinical hold after a brain tumor was found in one trial participant, the company said. Sign up here. The tumor was discovered during a routine brain scan of a five-year-old child who had received the treatment about four years earlier, the company said. Regenxbio said on Wednesday the U.S. Food and Drug Administration has placed a clinical hold on two of its experimental gene therapy programs for rare childhood diseases, sending its shares plummeting 30% premarket. RGX-111 is being developed as a treatment for Hurler syndrome, a rare autosomal recessive lysosomal storage disorder. It is estimated to occur in 1 in 100,000 births. Another therapy, RGX-121, being developed as a one-time treatment of MPS II or Hunter Syndrome, was also put on clinical hold by the FDA citing similarities between the two programs and a potentially shared risk. The company said it has not yet received the full clinical hold letter and awaits additional details from the FDA.

Original title: “US FDA places clinical hold on Regenxbio's rare disease program | Reuters”

Regenxbio said the abnormal growth ... for the treatment of severe mucopolysaccharidosis type I (MPS I). Also known as Hurler syndrome, the rare disease can cause developmental delays and shorten life span. RGX-111 is designed to improve outcomes by using an AAV9 vector to deliver the IDUA gene to the CNS. While the adverse event occurred in a recipient of RGX-111, the FDA extended the clinical hold to cover RGX-121 because of similarities between the therapies, trial populations ... Regenxbio said the abnormal growth was found in a recipient of RGX-111, a gene therapy the company is developing for the treatment of severe mucopolysaccharidosis type I (MPS I). Also known as Hurler syndrome, the rare disease can cause developmental delays and shorten life span. RGX-111 is designed to improve outcomes by using an AAV9 vector to deliver the IDUA gene to the CNS. While the adverse event occurred in a recipient of RGX-111, the FDA extended the clinical hold to cover RGX-121 because of similarities between the therapies, trial populations and shared risks of the studies. The FDA put clinical holds on two Regenxbio gene therapies, including a candidate that is less than two weeks away from an approval decision. A central nervous system (CNS) tumor has prompted the FDA to REGENXBIO clinical hold FDA Adverse Events tumor Cell & Gene Therapy Biotech

Original title: “FDA puts clinical hold on Regenxbio gene therapies weeks before approval ruling”

siRNA therapeutic progresses in global study for patients with elevated Lp(a)

Original title: “Argo Biopharma doses first patient in phase 2b trial of BW‑20829”

A milestone moment for longevity as Life Biosciences gets green light to progress cellular rejuvenation trial in eye disease. The Nobel prize-winning discovery sparked a global race to harness the approach therapeutically, and today marks a historic milestone in that journey with the news that the FDA has given the go-ahead for the first ever human trial of a partial epigenetic reprogramming therapy. The FDA’s decision clears an investigational new drug application for Life Bioscience’s ER-100, a gene therapy designed to rejuvenate damaged retinal cells in people with serious, age-related eye diseases. The Boston-based biotech co-founded by Harvard professor Dr David Sinclair, who showed in 2020 that a partial epigenetic reprogramming treatment restored vision in blind mice, is now preparing to commence a Phase 1 first-in-human study to show its therapy can be delivered safely in patients with open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy (NAION). More broadly, the company views the trial as a test case for an entire therapeutic platform. “Demonstrating that partial epigenetic reprogramming can be deployed in humans opens the door to applying the same approach to other age-related diseases and organ system,” said Rosenzweig-Lipson. While the eye is seen as an ideal starting point because it is accessible and allows for localized treatment, Life Bio is already exploring other potential applications for its technology. By excluding c-Myc, a factor associated with uncontrolled growth, the strategy is intended to lower the risk of tumors that has historically concerned regulators and clinicians. From a safety perspective, Rosenzweig-Lipson told us that the company’s preclinical studies in non-human primates demonstrated that ER-100 was well tolerated, with no systemic toxicities. “The therapy also uses a doxycycline-inducible system, giving us precise control over when the genes are active and allowing treatment to be paused or stopped if needed,” she added. This shift from aspiration to evidence-based clinical development is helping move the whole field forward.” ... Danny has worked in technology communications for more than 20 years, and has been writing about developments the longevity sector since 2019. With a keen interest in the transfer of technology to market, he focuses primarily on the companies working on cutting-edge solutions to aging and age-related diseases.

Original title: “FDA clears first human trial of epigenetic reprogramming therapy”

Company strengthens pipeline with AKTX-102 as it broadens its intellectual property estate

Original title: “Akari files new patent and advances second ADC targeting CEACAM5”

Conditions: CAMK2; Calcium/Calmodulin-dependent Protein Kinase 2 Interventions: Other: No intervention Sponsors: Erasmus Medical Center Recruiting

Original title: “CAMK2-related Synapthopathies Natural History Study”

Fahed Al Marzooqi, Chief Executive ... “This trial represents a pivotal step in strengthening Abu Dhabi’s position as a global hub for biotech innovation. Through strategic partnerships and clinical excellence, we are translating scientific breakthroughs into real-world treatments for patients with urgent, unmet needs. This collaboration reflects our mission to accelerate access to transformative therapies and to shape the future of rare disease research from ... Fahed Al Marzooqi, Chief Executive Officer of M42's Integrated Health Solutions, added: “This trial represents a pivotal step in strengthening Abu Dhabi’s position as a global hub for biotech innovation. Through strategic partnerships and clinical excellence, we are translating scientific breakthroughs into real-world treatments for patients with urgent, unmet needs. This collaboration reflects our mission to accelerate access to transformative therapies and to shape the future of rare disease research from the heart of the region.” Through this trial, Opus Genetics and its partners reaffirm their commitment to advancing precision medicine, accelerating rare disease research, and bringing transformative gene therapies to patients with urgent unmet needs. ... Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs). The Company is developing durable, one-time treatments designed to address the underlying genetic causes of severe retinal disorders. The therapy is designed to deliver a functional copy of the MERTK gene to retinal cells. Mutations in MERTK impair the retina’s ability to recycle photoreceptor components, leading to progressive degeneration and vision loss. There are currently no approved treatments for MERTK-related retinitis pigmentosa. The Opus MERTK program is expected to commence clinical development activities in 2026, marking a major milestone for rare disease research and precision medicine in Abu Dhabi and the wider region, where inherited retinal diseases affect an estimated Opus Genetics (Nasdaq: IRD) launched a clinical trial for OPGx-MERTK, an AAV-based gene therapy for MERTK-related retinitis pigmentosa, with funding from Abu Dhabi’s Healthcare Research and Innovation Fund. Cleveland Clinic Abu Dhabi will serve as the clinical site and clinical development activities are expected to commence in 2026. The program targets a disease affecting an estimated 60,000 patients worldwide and addresses an area with no approved treatments.

Original title: “New gene therapy trial targets rare blindness in 60,000 people”

The researchers uncovered that, as of January 2025, <strong>the FDA has approved only 1 orphan drug class (human alpha-1-proteinase inhibitors) for AATD since 1987, out of 20 designations, while the EMA has granted 9 orphan designations but no approvals</strong> ...

Original title: “2025 FDA/EMA Update on Orphan Drugs for AATD”