According to GlobalData, parent company of Clinical Trials Arena, the cell and gene therapy (CGT) sector is shifting amid a more selective and strategy-driven funding landscape. "Genespire to take rare disease gene therapy to clinic in 2026 " was originally created and published by Clinical ... According to GlobalData, parent company of Clinical Trials Arena, the cell and gene therapy (CGT) sector is shifting amid a more selective and strategy-driven funding landscape. "Genespire to take rare disease gene therapy to clinic in 2026 " was originally created and published by Clinical Trials Arena, a GlobalData owned brand. Now, companies across the pharmaceutical industry are developing therapies to treat methylmalonic acidaemia. Moderna’s lipid nanoparticle-powered mRNA therapy, mRNA-3705, is currently the only drug in the clinic – where it is being evaluated as a bi- or tri-weekly treatment for the rare disease in a Phase I/II study (NCT04899310). GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia. Rare disease biotech Genespire is looking to take its single-dose gene therapy – GENE202 – to the clinic in 2026, CEO and partner at Sofinnova Partners, Lucia Faccio, tells Clinical Trials Arena.

Original title: “Genespire to take rare disease gene therapy to clinic in 2026”

Genespire is looking to take its ... as a treatment for rare metabolic disease, methylmalonic acidaemia. Image credit: Visual Generation via ShutterStock.com. Rare disease biotech Genespire is looking to take its single-dose gene therapy – GENE202 – to the clinic in 2026, CEO and partner at Sofinnova Partners, Lucia Faccio, tells Clinical Trials ... Genespire is looking to take its single-dose gene therapy, GENE202, to the clinic by the end of 2026, where it will be evaluated as a treatment for rare metabolic disease, methylmalonic acidaemia. Image credit: Visual Generation via ShutterStock.com. Rare disease biotech Genespire is looking to take its single-dose gene therapy – GENE202 – to the clinic in 2026, CEO and partner at Sofinnova Partners, Lucia Faccio, tells Clinical Trials Arena. Now, companies across the pharmaceutical industry are developing therapies to treat methylmalonic acidaemia. Moderna’s lipid nanoparticle-powered mRNA therapy, mRNA-3705, is currently the only drug in the clinic – where it is being evaluated as a bi- or tri-weekly treatment for the rare disease in a Phase I/II study (NCT04899310). GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia. If all goes to plan, the Italian biotech will initiate a clinical trial by the end of 2026, while also performing observational studies to better characterise this rare disease and the unmet patient needs. This news comes as GENE202 was granted orphan designation from both the US Food and Drug Administration (FDA) and the European Commission (EC), which was announced by the company on 20 January.

Original title: “Genespire to take rare disease gene therapy to clinic in 2026 - Clinical Trials Arena”

The Food and Drug Administration (FDA) is announcing the issuance of a priority review voucher to the sponsor of a rare pediatric disease product application. The Federal Food, Drug, and Cosmetic Act (FD&C Act) authorizes FDA to award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA is required to publish notice of the award of the priority review voucher. FDA has determined that KYGEVVI (doxecitine and doxribtimine), ap

Original title: “Issuance of Priority Review Voucher; Rare Pediatric Disease Product; KYGEVVI (Doxecitine and Doxribtimine)”

The Food and Drug Administration (FDA) is announcing the issuance of approval of a product redeeming a priority review voucher. The Federal Food, Drug, and Cosmetic Act (FD&C Act) authorizes FDA to award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA is required to publish notice of the issuance of priority review vouchers as well as the approval of products redeeming a priority review voucher. FDA has determined that

Original title: “Notice of Approval of Product Under Voucher: Rare Pediatric Disease Priority Review Voucher; RHAPSIDO (Remibrutinib)”

NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients. This decision follows a full submission assessed through the SMC orphan medicine process and applies only in the context of an approved NHS Scotland Patient Access Scheme, which allows confidential pricing arrangements for high-cost therapies. SCD is a chronic, inherited blood disorder caused by mutations in the beta-globin gene, leading to the production of sickle haemoglobin. Clinical experts highlighted its potential to deliver durable disease modification after a single intervention, reducing dependence on hydroxycarbamide, blood transfusions, and recurrent hospital care. Exagamglogene autotemcel is a personalised, autologous, cell-based gene therapy that uses CRISPR/Cas9 gene-editing technology to modify a patient’s own stem cells. All patients experienced treatment-related adverse events, which were primarily attributable to busulfan conditioning treatment rather than to the gene therapy itself. Common events included febrile neutropenia, infections, and cytopenias, typically occurring within the first 6 months after infusion. No cases of graft failure or graft-vs-host disease were reported. Cite this: Edited by Zeel Mehta. Scotland Approves Gene Therapy for Sickle Cell Disease - Medscape - January 20, 2026.

Original title: “Scotland Approves Gene Therapy for Sickle Cell Disease”

CVMP opinions on veterinary medicinal productsThe Committee adopted a positive opinion on a marketing authorisation from Elanco for Lotilaner / Milbemycin Elanco (…

Original title: “Meeting highlights from the Committee for Veterinary Medicinal Products (CVMP) 13-14 January 2026”

In 2025, EMA recommended 104 medicines for marketing authorisation. Of these, 38 had a new active substance which had never been authorised in the European Union (EU) before.…

Original title: “Human medicines in 2025”

You know there have been trials ... temporary, and then do the gene therapy I mean. The other question is: Cumulative treatment, which you know in rare diseases doesn't seem to be anything that you know would happen in clinical trials, very interestingly compared to other ... You know there have been trials going into enzyme replacement and other things, but really what would be ideal would be having a bridge to gene therapy so that when someone has got symptoms of MLD, which are early juvenile or pre- symptomatic children, that you don't have to worry as much if you can give them a bridge, something temporary, and then do the gene therapy I mean. The other question is: Cumulative treatment, which you know in rare diseases doesn't seem to be anything that you know would happen in clinical trials, very interestingly compared to other conditions. So if you think about childhood cancer, you know treatment is always cumulative. You know you start regimen, this regimen and then when they don't respond, you add something else. And you add something else and you add something else, and that's well known and you know when you have clinical trials showing that something can be added on as added therapy that is beneficial in rare diseases it seems to be harder to do. Is that you know, because it's rare disease, funding and research and things and patient eligibility, sometimes a patient having had some other treatment precludes them from a particular clinical trial because that may modify the result, whereas if you look at childhood cancer that doesn't happen. So it is interesting how we may need to think, change our thinking with rare diseases. Recorded Webinar MLD-S4-M3 (Dr Dipak Ram and Dr Mireia Del Toro)

Original title: “Rare Diseases – Current and Upcoming Treatments | EIP Metachromatic Leukodystrophy (MLD)”

Kone AS, Ait Ssi S, Sahraoui S, ... and Treatment. Int J Mol Sci. 2022;23(21):13424. Published 2022 Nov 3. doi:10.3390/ijms232113424 ... New data reinforces Ipsen’s commitment to bringing solutions and addressing care gaps in neurological diseases at TOXINS ... Ipsen expands early development pipeline with Simcere Zaiming’s innovative antibody drug conjugate ... Ipsen update on Phase II FALKON trial in patients with ultra-rare bone disease, ... Kone AS, Ait Ssi S, Sahraoui S, Badou A. BTN3A: A Promising Immune Checkpoint for Cancer Prognosis and Treatment. Int J Mol Sci. 2022;23(21):13424. Published 2022 Nov 3. doi:10.3390/ijms232113424 ... New data reinforces Ipsen’s commitment to bringing solutions and addressing care gaps in neurological diseases at TOXINS ... Ipsen expands early development pipeline with Simcere Zaiming’s innovative antibody drug conjugate ... Ipsen update on Phase II FALKON trial in patients with ultra-rare bone disease, fibrodysplasia ossificans progressiva (FOP) This Breakthrough Therapy Designation is based on data from the Phase I/II EVICTION trial. Updated clinical data orally presented at the American Society of Hematology (n=57)1 from the EVICTION trial showed treatment with IPN60340 in combination with Ven-Aza achieved very encouraging high responses. IPN60340 is an investigational therapy under evaluation for people 75 years or older living with acute myeloid leukemia who due to comorbidities are prevented from receiving treatment with intensive chemotherapy. About Ipsen We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. EVICTION is a first-in-human, dose-escalation (Part 1) and cohort-expansion (Part 2) clinical trial of IPN60340 (ICT01) in patients with various advanced relapsed or refractory solid or hematologic cancers that have exhausted standard-of-care treatment options, as well as newly-diagnosed AML.

Original title: “U.S. FDA grants Ipsen’s IPN60340 (ICT01) Breakthrough Therapy Designation in first line unfit Acute Myeloid Leukemia”

The U.S. Food and Drug Administration today approved the Zycubo (copper histidinate) injection as the first treatment for Menkes disease in pediatric patients.

Original title: “FDA Approves First Treatment for Children With Menkes Disease”

We are concerned that this decision ... of rare disease treatments, effectively creating barriers for generating clinical evidence within a unique patient population with ultra-rare conditions thereby significantly delaying—or preventing altogether— patient access to urgently needed therapies…... We are concerned that this decision may have far-reaching consequences for the development of rare disease treatments, effectively creating barriers for generating clinical evidence within a unique patient population with ultra-rare conditions thereby significantly delaying—or preventing altogether— patient access to urgently needed therapies…. The FDA has issued a new complete response letter to Atara Biotherapeutics' regarding its biologics license application (BLA) for allogeneic Epstein-Barr virus (EBV)–specific T-cell immunotherapy tabelecleucel (tab-cel; marketed as Ebvallo) for the treatment of patients 2 years or older with EBV-positive posttransplant lymphoproliferative disease (EBV+ PTLD) who have received at least 1 prior therapy.1 · The CRL indicates that the FDA did not find the data from the pivotal phase 3 ALLELE clinical trial (NCT03394365) supporting the BLA to provide sufficient evidence of tab-cel’s efficacy. The CRL indicates that the ALLELE clinical trial does not sufficiently show efficacy of the product. We intend to engage with the FDA to urgently pursue a path forward, in collaboration with Atara Biotherapeutics and our clinical and patient partners, to enable timely accelerated approval of tabelecleucel. We continue to be committed to making tabelecleucel available to patients through our Expanded Access Program.” · Tab-cel has previously been approved in the European Union, the United Kingdom, and Switzerland for the treatment of relapsed/refractory EBV+ PTLD in patients aged 2 years and older who have received at least 1 prior therapy.3 Notably, Atara stated in January 2025 that a manufacturing facility operated by FUJIFILM Diosynth Biotechnologies and located in Thousand Oaks, California, had received approval to manufacture tab-cel for the European Medicines Agency.4

Original title: “FDA Hits Atara Biotherapeutics’ T-Cell Immunotherapy Tabelecleucel for EBV+ PTLD With Second CRL | CGTlive®”

The Chief Medical Officer of ModeX Therapeutics discusses MDX2003, a groundbreaking tetraspecific therapy that promises enhanced efficacy and safety in non-Hodgkin lymphoma treatment. This exchange appears in the January issue of Evidence-Based Oncology, the annual recap of the meeting of the ... The Chief Medical Officer of ModeX Therapeutics discusses MDX2003, a groundbreaking tetraspecific therapy that promises enhanced efficacy and safety in non-Hodgkin lymphoma treatment. This exchange appears in the January issue of Evidence-Based Oncology, the annual recap of the meeting of the American Society of Hematology. Abbadessa: MDX2003 is currently administered via intravenous (IV) infusion, which would allow its use in the community clinic setting. Our plan is to develop this molecule for favorable dosing logistics, which will depend on data from the phase 1 trial. Our MSTAR molecules can be formulated for non-IV administration, and this can be addressed as the program progresses. AJMC: To confirm, this treatment would be for NHL conditions. Is there a particular disease state where it will be tested first? A phase 1 trial for MDX2003 will begin in early 2026, focusing on B-cell non-Hodgkin lymphomas. MDX2003 is administered via IV infusion, with potential for community clinic use and non-IV formulations in the future.SHOW MORE · The Chief Medical Officer of ModeX Therapeutics discusses MDX2003, a groundbreaking tetraspecific therapy that promises enhanced efficacy and safety in non-Hodgkin lymphoma treatment. The phase 1 trial will be carried out in a range of B-cell non-Hodgkin lymphomas, which usually express CD19 and CD20. These lymphomas include diffuse large B-cell lymphoma, follicular lymphoma, mantle cell lymphoma, marginal zone lymphomas, indolent lymphomas, and Waldenström macroglobulinemia. More detailed information, with the inclusion and exclusion criteria, will be available on ClinicalTrial.gov once the study starts.

Original title: “What Would Make Therapy Last? How About 2 Targets Each on B Cells and T Cells? | AJMC”

After a lengthy downturn, the biotech industry finally gathered momentum in 2025. Key readouts in obesity, infectious disease and many rare conditions could help it continue. But, while Intellia’s clinical progress has at times displayed CRISPR’s massive therapeutic potential, the company has burned through more than $2 billion since inception and twice laid off staff amid shifting research plans. Its share price sits near record lows, too, following the death of a study participant and suspension of one of its top programs. Intellia still has a shot at success, though. In the first half of 2026, it’s expecting results from a Phase 3 trial testing a possible treatment for a rare condition called hereditary angioedema. Clinical trial results have fueled many of these outcomes, spurring interest from acquirers or investors. This year, other important readouts lay ahead in obesity, infectious diseases, rare disorders and more. Miller, of Evercore, projects sales would top out at $500 million annually in peak years, given the way gene therapy launches have gone so far. — Ben Fidler ... Vertex Pharmaceuticals is best known for a cystic fibrosis drug business that now regularly brings in more than $10 billion each year. But pressure to recreate that success elsewhere is intensifying, as a pain medicine it recently brought to market may not be the blockbuster it once hoped. One drug Vertex is developing for a rare kidney disease could be part of the solution. The second is in people with obesity and diabetes, while the third is in those with obesity and established cardiovascular disease, as defined by a previous heart attack or stroke. Combined, the trials have enrolled more than 5,000 people. Success in these studies would keep Lilly ahead of a raft of emerging competitors — among them chief rival Novo Nordisk, which is currently trying to win approval of a dual-acting therapy.

Original title: “10 clinical trials to watch in the first half of 2026 | BioPharma Dive”

The Food and Drug Administration (FDA or the Agency) has determined the regulatory review period for ALHEMO and is publishing this notice of that determination as required by law. FDA has made the determination because of the submission of an application to the Director of the U.S. Patent and Trademark Office (USPTO), Department of Commerce, for the extension of a patent which claims that human biological product.

Original title: “Determination of Regulatory Review Period for Purposes of Patent Extension; ALHEMO”

The Food and Drug Administration (FDA or the Agency) is announcing the issuance of two Emergency Use Authorizations (EUA) (Authorization) under the Federal Food, Drug, and Cosmetic Act (FD&C Act) for new animal drug products. FDA has issued one EUA for a new animal drug product as requested by Elanco US Inc. for the treatment of infestations caused by New World screwworm (Cochliomyia hominivorax) (NWS) larvae (myiasis) in dogs and puppies and one EUA for a new animal drug product as requested by

Original title: “Authorization of Emergency Use for Two Animal Drugs for the Treatment of New World Screwworm; Availability”

The Food and Drug Administration (FDA) is announcing the issuance of a priority review voucher to the sponsor of a rare pediatric disease product application. The Federal Food, Drug, and Cosmetic Act (FD&C Act) authorizes FDA to award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA is required to publish notice of the award of the priority review voucher. FDA has determined that FORZINITY (elamipretide), approved Septem

Original title: “Issuance of Priority Review Voucher; Rare Pediatric Disease Product; FORZINITY (Elamipretide)”

The Food and Drug Administration (FDA) is announcing the issuance of approval of a product redeeming a priority review voucher. The Federal Food, Drug, and Cosmetic Act (FD&C Act) authorizes FDA to award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA is required to publish notice of the issuance of priority review vouchers as well as the approval of products redeeming a priority review voucher. FDA has determined that

Original title: “Notice of Approval of Product Under Voucher: Rare Pediatric Disease Priority Review Voucher; KEYTRUDA QLEX (pembrolizumab and berahyaluronidase alfa-pmph)”

The Food and Drug Administration (FDA, the Agency, or we) is classifying the electrical tongue nerve stimulator to treat motor deficits into class II (special controls). The special controls that apply to the device type are identified in this order and will be part of the codified language for the classification of the electrical tongue nerve stimulator to treat motor deficits. We are taking this action because we have determined that classifying the device into class II will provide a reasonab

Original title: “Medical Devices; Neurological Devices; Classification of the Electrical Tongue Nerve Stimulator To Treat Motor Deficits”

The U.S. Food and Drug Administration today awarded a national priority voucher to teclistamab in combination with daratumumab for relapsed/refractory multiple myeloma.

Original title: “FDA Proactively Awards National Priority Voucher Based on Strong Phase 3 Study Results”

The U.S. Food and Drug Administration recently approved two new oral medicines to treat a common sexually transmitted infection called gonorrhea.

Original title: “FDA Approves Two Oral Therapies to Treat Gonorrhea”