The U.S. Food and Drug Administration has approved a first-of-its-kind device for the treatment of adult patients with locally advanced pancreatic cancer.

Original title: “FDA Approves First-of-Its-Kind Device to Treat Pancreatic Cancer”

Dec 18, 2025 Positive Clinical Results from Phase 2 Trial of SGX945 for the Treatment of Behçet's Disease Published in Rheumatology (Oxford) Dec 17, 2025 Soligenix Announces Top-line Results of the Phase 2a Study of SGX302 (Synthetic Hypericin) in Patients with Mild-to-Moderate Psoriasis · Dec 5, 2025 Soligenix Inc. (NASDAQ: SNGX) Advancing Rare-Disease Therapies ... Dec 18, 2025 Positive Clinical Results from Phase 2 Trial of SGX945 for the Treatment of Behçet's Disease Published in Rheumatology (Oxford) Dec 17, 2025 Soligenix Announces Top-line Results of the Phase 2a Study of SGX302 (Synthetic Hypericin) in Patients with Mild-to-Moderate Psoriasis · Dec 5, 2025 Soligenix Inc. (NASDAQ: SNGX) Advancing Rare-Disease Therapies Positioned at Pivotal Crossroad Investors may focus on timely FLASH2 execution, regulatory interactions, and future trial designs as key checkpoints for the rare-disease strategy. cutaneous t-cell lymphoma, investigator-initiated study, area under the curve (auc), dermatology life quality index, +1 more ... "clinical trial for HyBryte™ ... in the treatment of early-stage cutaneous T-cell lymphoma (CTCL)" With 2026 being an important year for us, we remain energized by the promise of our late-stage rare disease pipeline as we continue to evaluate potential strategic options, including, but not limited to, partnership and merger and acquisition opportunities. The previously publicly disclosed upcoming key clinical events and milestones are summarized below. Top-line results from the actively enrolling 80 patient confirmatory Phase 3 FLASH2 ( ... Hypericin 2) clinical trial for HyBryte™ (SGX301 or synthetic hypericin) in the treatment of early-stage cutaneous T-cell lymphoma (CTCL) are expected in the second half of 2026, with an interim analysis fast approaching in 2Q. FLASH2 interim readout in 2Q and top-line CTCL data in 2H 2026 lead Soligenix update, with Behçet's, psoriasis progress and funding runway through 2026.

Original title: “Rare cancer therapy: Soligenix targets ~$2B in potential annual sales”

FDA designation aims to speed development of new diagnostic tool

Original title: “99mtc‑maraciclatide granted fast track status for ILD imaging”

New FDA Pilot Program Will Provide Hands-On Regulatory Guidance for Cell and Gene Therapy Trials in Rare Disease · Neurogene also announced in June 2024 that NGN-401 had been selected as part of the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program.2 ... New FDA Pilot Program Will Provide Hands-On Regulatory Guidance for Cell and Gene Therapy Trials in Rare Disease · Neurogene also announced in June 2024 that NGN-401 had been selected as part of the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program.2 The program will provide sponsors with the opportunity for frequent advice and regular communication as needed with regard to clinical trial design and other development issues particular to the investigational product being evaluated. We look forward to participating in this landmark effort with the FDA as we seek to rapidly advance NGN-401 toward a potential registrational study for the patients and families who live with this devastating disease.” · NGN-401 is being evaluated in a phase 1/2 clinical trial (NCT05898620). As of June 2024, the therapy has been well-tolerated in treated study participants, with all treatment-related adverse events (AEs) being mild and transient or resolving. NGN-401 is being evaluated in an early phase clinical trial which has been selected for the FDA’s START Pilot Program. ... “We appreciate the FDA’s ongoing commitment to expedite development of our NGN-401 gene therapy for Rett syndrome, with RMAT designation following NGN-401’s selection for the FDA’s START Pilot Program, 2 synergistic initiatives designed to more rapidly advance promising treatments for patients with unmet medical needs,” Rachel McMinn, PhD, Founder and Chief Executive Officer, Neurogene, said in a statement.1 “RMAT and START designations underscore the therapeutic potential of NGN-401 for Rett syndrome and reflect our commitment to accelerate development of NGN-401. There have also been no treatment-emergent or intracerebroventricular (ICV) procedure-related serious AEs.3 · “We have met an important program milestone of initiating dosing in the high-dose cohort of our Rett syndrome gene therapy trial, and we are pleased to share that high-dose NGN-401 has been well-tolerated thus far with an early favorable safety profile,” McMinn said in a statement at that time.3 “We intentionally designed NGN-401 with the goal of creating a best-in-class treatment option by leveraging what we believe to be the optimal route of administration to deliver consistent and tightly controlled full-length MECP2 expression to key areas of the brain and nervous system, and today’s safety update underscores NGN-401’s potential to deliver on that profile.

Original title: “Rett Syndrome Gene Therapy Adds RMAT Designation to the List | CGTlive®”

ProLectin‑M achieves 100% viral elimination by day seven in placebo‑controlled study

Original title: “Bioxytran reports rapid viral clearance in phase 2 trial”

The percentage of patients achieving total clearance of eczema lesions in a Phase 2b trial increased with prolonged rezpeg treatment.

Original title: “Nektar’s Rezpeg Differentiates in Atopic Dermatitis With Durable, Deep Responses in Mid-Stage”

The FDA issued a CRL for gene therapy RGX-121 in Hunter syndrome, citing trial design and biomarker doubts; Regenxbio seeks a meeting with the agency and resubmission. A January 2026 clinical hold on CAMPSIITE followed an intraventricular CNS tumor in an RGX-111 recipient, with suspected AAV integration and PLAG1 overexpression. The hold was applied across RGX-111 and RGX-121 due to platform and population similarities, while causality remains undetermined and RGX-121 safety in >30 patients is asserted.SHOW MORE · The FDA issued a CRL for gene therapy RGX-121 in Hunter syndrome, citing trial design and biomarker doubts; Regenxbio seeks a meeting with the agency and resubmission. According to Regenxbio, the FDA’s CRL, which it received February 7, 2026, covered several reasons the gene therapy product was not approved—despite the agency’s agreement with the study protocol in principle—including concerns about the ability of the clinical trial eligibility criteria to distinguish between neuronopathic disease and attenuated disease adequately, whether the external natural history control population used is sufficiently comparable to the trial population, and if there is reasonable likelihood that the surrogate end point used—heparan sulfate D2S6 levels in the cerebrospinal fluid—can predict clinical benefit in an appropriate manner. Notably, the CRL contained several suggested pathways to a potential approval for the product, such as a new clinical trial, carrying out dosing of additional patients with longer follow-up, and implementation of an untreated control group on-study. However, Regenxbio stated that it has concerns about the difficulty of these pathways considering the ultrarare nature of MPS II. "This decision is devastating for the families of boys living with this progressive, life-threatening disease," Curran M. Simpson, the president and CEO of Regenxbio, said in a statement.1 "We are concerned about FDA’s feedback regarding the overall development path and evaluation of the data in the context of the urgent need for this irreversible ultra-rare disease. We remain confident in the quality and volume of evidence demonstrating the long-term potential of RGX-121 to positively change the trajectory of Hunter syndrome. This program has been in development for over 10 years. We are incredibly grateful to all the patients, their families, investigators, and site staff who have supported this program and our continued efforts to bring a much-needed new treatment option to the Hunter syndrome community.

Original title: “Regenxbio's MPS II Gene Therapy RGX-121 Hit With CRL | NeurologyLive - Clinical Neurology News and Neurology Expert Insights”

Signal Transduction and Targeted Therapy - Investigation of a clinical trial drug VV261 as a potent antiviral candidate against Chikungunya virus Since 2004, CHIKV outbreaks have become more frequent and widespread, affecting millions of people and establishing Chikungunya disease as a significant global public health issue.1 In Asia, the Asian lineage (CHIKV-Asian) and the East-Central-South-African lineage (CHIKV-ECSA) have become the most prevalent. In July 2025, a large-scale outbreak occurred in Guangdong Province, China, resulting in more than 9000 confirmed cases.2 To date, no drug has been approved for clinical therapy, highlighting the urgent need to develop effective treatments against CHIKV. Considering the preclinical pharmacokinetic studies in mice and monkeys,5 it is estimated that after oral administration of 10 mpk VV261 in humans, the maximum blood concentration of 4’-FlU would be approximately 2000 ng/mL, which is about 9000-fold higher than the EC50 value for inhibiting CHIKV. Given the urgent clinical need for treatments against CHIKV, clinical studies should be conducted to investigate its efficacy for Chikungunya fever therapy. VV261 was designed to enhance the chemical stability and pharmacokinetic properties of 4’-FlU, the original nucleoside that is susceptible to aqueous solutions and obscure to be druggability, and our previous study has demonstrated that VV261 has potent activity against severe fever with thrombocytopenia syndrome virus (SFTSV).5 In 2024, VV261 has been approved for Phase I clinical trials in China for the treatment of severe fever with thrombocytopenia syndrome (SFTS). The viral RNA copy reductions in the target tissues are consistent with the readouts of viral titers (supplementary Fig. 6). Additionally, the results of clinical sign scoring on muscle weakness of limbs, reduced activity, and piloerection revealed that compound VV261 (5 and 10 mpk) and T-705 (400 mpk) could significantly reduce disease severity (supplementary table 2).

Original title: “Investigation of a clinical trial drug VV261 as a potent antiviral candidate against Chikungunya virus | Signal Transduction and Targeted Therapy”

Rockville’s RegenxBio just got a rejection from regulators on a treatment for a rare disease that it'd hoped would become its first product to market. The Rockville biotech's gene therapy for Hunter syndrome faced FDA rejection over study design concerns, threatening its path to its first commercial product. The treatment for a rare neurodegenerative disease that afflicts young boys would be the company's first commercial product.

Original title: “FDA rejects RegenxBio's gene therapy for rare disease, a major setback for Rockville company - Washington Business Journal”

Rockville’s RegenxBio just got a rejection from regulators on a treatment for a rare disease that it'd hoped would become its first product to market. The Rockville biotech's gene therapy for Hunter syndrome faced FDA rejection over study design concerns, threatening its path to its first commercial product. The treatment for a rare neurodegenerative disease that afflicts young boys would be the company's first commercial product.

Original title: “FDA rejects RegenxBio's gene therapy for rare disease, a major setback for Rockville company - Washington Business Journal”

The newly public Evommune shared data showing that EVO301, an IL-18 targeting protein, cleared symptoms comparably to Regeneron and Sanofi’s mega-blockbuster in a mid-stage atopic dermatitis clinical trial.

Original title: “Evommune Soars as Dermatitis Treatment Rivals Dupixent in Mid-Stage Trial”

FDA rejects Regenxbio rare disease gene therapy RGX-121 over trial and safety concerns, halting approval under accelerated pathway. The decision follows a clinical hold the FDA placed Jan. 28 on two Regenxbio gene therapy programs — RGX-111 and RGX-121 — after a brain tumor was detected in a child who had received RGX-111 four years earlier. The FDA has rejected Regenxbio’s biologics license application for RGX-121, an experimental gene therapy for Hunter syndrome.

Original title: “FDA rejects Regenxbio’s rare disease gene therapy”

The FDA recommended that REGENXBIO run a new study, treat more patients and include a placebo arm to support a resubmission for the gene therapy RGX-121.

Original title: “REGENXBIO’s Hunter Syndrome Gene Therapy Fails to Win FDA Nod”

The rejection comes a week after the FDA placed clinical holds on RGX-121 and a separate therapy for a different rare disease, Hurler syndrome, when a treated study participant with the latter condition developed a brain tumor. The FDA extended the hold to both products because of certain similarities, as well as a “shared risk” between the two studies. Regenxbio has countered that no causal link was established and no similar issues were identified in all of the 32 trial ... The rejection comes a week after the FDA placed clinical holds on RGX-121 and a separate therapy for a different rare disease, Hurler syndrome, when a treated study participant with the latter condition developed a brain tumor. The FDA extended the hold to both products because of certain similarities, as well as a “shared risk” between the two studies. Regenxbio has countered that no causal link was established and no similar issues were identified in all of the 32 trial participants who have received RGX-121. The decision comes two weeks after the agency halted testing due to safety concerns and represents the latest regulatory setback for a gene therapy maker. However, the FDA ultimately decided that, while it agreed “in principle” to the design of the clinical trial Regenxbio built its application around, multiple issues with its results persisted. The regulator wasn’t convinced by the surrogate measure Regenxbio used to assess the treatment’s effectiveness, and was skeptical of the study’s comparisons between historical data and the group receiving the therapy. The Food and Drug Administration rejected a gene therapy Regenxbio has been developing for a rare disease called Hunter syndrome, in a decision that marks the latest blow for a field that has seen multiple recent regulatory setbacks in the U.S.

Original title: “FDA rejects Regenxbio treatment in another blow to gene therapy | BioPharma Dive”

Regulator recommends durvalumab as first perioperative IO option for muscle‑invasive disease

Original title: “AstraZeneca bladder cancer therapy wins NICE backing”

Re:Cognition Health and Cera collaborate to expand access to Alzheimer’s and neurodegenerative clinical trials

Original title: “Redefining trial access”

** Shares of drug developer Regenxbio NASDAQ:RGNX tumble 14% to $8.85 premarket** U.S. Food and Drug Administration declines to approve co's gene therapy for a rare disease called Hunter syndrome, citing uncertainty regarding the trial design** Co plans to seek meeting with the FDA on ... ** Shares of drug developer Regenxbio NASDAQ:RGNX tumble 14% to $8.85 premarket** U.S. Food and Drug Administration declines to approve co's gene therapy for a rare disease called Hunter syndrome, citing uncertainty regarding the trial design** Co plans to seek meeting with the FDA on resubmitting…

Original title: “Regenxbio drops after FDA rejects gene therapy for rare disease — TradingView News”

NeurologyLive is your direct connection to the latest neurology news and interviews with expert neurologists in multimedia formats. According to REGENXBIO, the FDA’sCRL, which it received on February 7, 2026, covered several reasons that the gene therapy product was not approved—despite the agency’s agreement with the study protocol in principle—including concerns about the ability of the clinical trial eligibility criteria to distinguish between neuronopathic disease and attenuated disease adequately, whether the external natural history control population used is sufficiently comparable to the trial population, and if there is reasonable likelihood that the surrogate end point used—heparan sulfate (HS) D2S6 levels in the cerebrospinal fluid (CSF)—can predict clinical benefit in an appropriate manner. Notably, the CRL contained several suggested pathways to a potential approval for the product, such as a new clinical trial, carrying out dosing of additional patients with longer follow-up, and implementation of an untreated control group on-study. Although, REGENXBIO stated that it has concerns about the difficulty of these pathways considering the ultrarare nature of MPS II. "This decision is devastating for the families of boys living with this progressive, life-threatening disease," Curran M. Simpson, the president and chief executive officer of REGENXBIO, said in a statement.1 "We are concerned about FDA's feedback regarding the overall development path and evaluation of the data in the context of the urgent need for this irreversible ultra-rare disease. We remain confident in the quality and volume of evidence demonstrating the long-term potential of RGX-121 to positively change the trajectory of Hunter syndrome. This program has been in development for over 10 years. We are incredibly grateful to all the patients, their families, investigators, and site staff who have supported this program and our continued efforts to bring a much-needed new treatment option to the Hunter syndrome community. Patient safety is our top priority, and we, our investigators, and the patient community remain confident in the benefit-risk ratio of RGX-121 and are highly encouraged by the meaningful efficacy profile demonstrated in the pivotal trial. RGX-121 presents an opportunity to address the urgent, significant unmet medical need in this ultrarare disease community, and continued delay means continued neurodevelopmental decline in boys with MPS II." Keep your finger on the pulse of neurology—subscribe to NeurologyLive for expert interviews, new data, and breakthrough treatment updates.

Original title: “REGENXBIO's MPS II Gene Therapy RGX-121 Hit With CRL | NeurologyLive - Clinical Neurology News and Neurology Expert Insights”

The FDA has issued a complete response ... for the treatment of mucopolysaccharidosis type 2 (MPS II), also known as Hunter syndrome.¹ · REGENXBIO reported that it received the CRL on February 7, 2026. In the letter, the FDA outlined multiple reasons for not approving the gene therapy at this time, despite noting general agreement with the study protocol. These included concerns about whether the clinical trial eligibility criteria adequately differentiate neuronopathic from attenuated disease, whether the ... The FDA has issued a complete response letter (CRL) to REGENXBIO’s biologics license application (BLA) for clemidsogene lanparvovec (RGX-121), an investigational adeno-associated virus (AAV) vector–based gene therapy being developed for the treatment of mucopolysaccharidosis type 2 (MPS II), also known as Hunter syndrome.¹ · REGENXBIO reported that it received the CRL on February 7, 2026. In the letter, the FDA outlined multiple reasons for not approving the gene therapy at this time, despite noting general agreement with the study protocol. These included concerns about whether the clin FDA objections centered on study population misclassification risk, external control validity, and surrogate end point evidentiary strength. The CRL also described several potential paths toward approval, including conducting a new clinical trial, dosing additional patients with longer follow-up, and incorporating an on-study untreated control group. REGENXBIO noted, however, that these approaches present challenges given the ultrarare nature of MPS II. "This decision is devastating for the families of boys living with this progressive, life-threatening disease," Curran M. Simpson, the president and chief executive officer of REGENXBIO, said in a statement.1 "We are concerned about FDA's feedback regarding the overall development path and evaluation of the data in the context of the urgent need for this irreversible ultra-rare disease. We remain confident in the quality and volume of evidence demonstrating the long-term potential of RGX-121 to positively change the trajectory of Hunter syndrome. This program has been in development for over 10 years. We are incredibly grateful to all the patients, their families, investigators, and site staff who have supported this program and our continued efforts to bring a much-needed new treatment option to the Hunter syndrome community.

Original title: “FDA Slams REGENXBIO's MPS II Gene Therapy RGX-121 With CRL | CGTlive®”

The Food and Drug Administration has rejected a rare-disease gene therapy from Regenxbio, the company said Monday. The Food and Drug Administration has rejected a rare-disease gene therapy from Regenxbio, the company said Monday. The one-time treatment, called RGX-121, is designed to replace a malfunctioning gene that causes mucopolysaccharidosis type II, also known as Hunter syndrome, an ultra-rare disorder that causes physical and cognitive impairments. RGX-121 is designed to replace a malfunctioning gene that causes mucopolysaccharidosis type II, also known as Hunter syndrome

Original title: “FDA rejects Regenxbio’s MPS II rare-disease gene therapy | STAT”