Gene therapy

Launch of CGTxchange to reactivate cell and gene therapy programs

Fri, 15 May 2026 09:00:00 -0400

The American Society of Gene & Cell Therapy (ASGCT) and Orphan Therapeutics Accelerator (OTXL) have announced the public launch of CGTxchange, an AI-enhanced clearinghouse and marketplace built to help reactivate cell and gene therapy programs that have been shelved despite strong scientific and clinical evidence.

ASGCT 2026: Directed evolution in gene therapy

Fri, 15 May 2026 09:00:00 -0400

Directed evolution has become a central pillar in gene therapy. This engineering strategy enables the generation of more efficient variants of genetic editors and delivery vectors. Molecular diversification methods are increasingly sophisticated and are now accelerated by machine learning and AI tools, as showcased at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) held in Boston this week.

Will FDA show Affinity for fast yes to Regenxbio in DMD?

Thu, 14 May 2026 12:00:00 -0400

How the U.S. FDA might view the latest Duchenne muscular dystrophy (DMD) phase III data with gene therapy RGX-202 became the question for Regenxbio Inc., shares of which (NASDAQ:RGNX) closed May 14 at $6.25, down 38%, or $3.80, after the results were made public.

KHN-921 gains IND clearance for MYBPC3-associated HCM

Thu, 14 May 2026 09:00:00 -0400

Chengdu Origen Biotechnology Co. Ltd. and Vanotech Ltd. have announced IND clearance by the FDA for KHN-921 for the treatment of hypertrophic cardiomyopathy (HCM) associated with MYBPC3 mutations.

ASGCT 2026: Uncovering the mechanisms of AAV toxicity

Thu, 14 May 2026 09:00:00 -0400

Gene therapies rely on vectors to reach the target tissue where they act, such as adeno-associated viruses (AAVs) or lipid nanoparticles (LNPs), among other delivery strategies. Each combination is optimized for a specific cell type and indication, aiming to overcome challenges such as efficacy, specificity and toxicity. On May 13, 2026, two sessions included in the scientific symposia of the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), being held in Boston this week, addressed AAV-related toxicities, which have led to fatal cases in clinical trials and remain an area for improvement in approved therapies.

ASGCT 2026: Circular RNA, the new beast in gene and cell therapy

Wed, 13 May 2026 09:00:00 -0400

Circular RNA (circRNA) is not a new concept, but it is a novel strategy in the field of gene and cell therapy. While mRNA vaccines have revolutionized medicine, this RNA fragment without free ends surpasses their performance in both efficacy and durability, bringing it to the attention of several pioneering companies. The latest advances in circRNA presented at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) clearly surpass the performance achieved with linear mRNA.

BTX-001 enables single-dose treatment for geographic atrophy

Wed, 13 May 2026 09:00:00 -0400

Researchers from Beacon Therapeutics Holdings Ltd. reported the development of BTX-001, an intravitreal gene therapy designed to target the complement pathway through delivery of a C5 inhibitor.

MHRA sets out proposal to redefine gene therapies

Tue, 12 May 2026 12:00:00 -0400

The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has opened a consultation on changes it is proposing to the legal definition of gene therapies, to reflect the advances in technology and manufacturing over the two decades since the current legislation was drawn up. The aim is to correct the mismatch between regulatory terminology and modern science, which MHRA says “can lead to uncertainty” over how a product is classified and to “inconsistent oversight and potential barriers for developers."

Fractyl Health’s RJVA-001 cleared to enter clinic in Netherlands

Tue, 12 May 2026 09:00:00 -0400

Fractyl Health Inc. has received clinical trial application authorization in the Netherlands to initiate a first-in-human phase I/II study of RJVA-001, the first clinical candidate from the company’s Rejuva Smart GLP-1 gene therapy platform.

Alzheimer’s, beyond the brain

Mon, 11 May 2026 09:00:00 -0400

Researchers at Daping Hospital in China have reported that liver-targeted delivery of the APOE3-Christchurch (APOE3Ch) variant, a rare protective form of apolipoprotein E, can indirectly reduce brain pathology, highlighting the therapeutic potential of peripheral approaches to Alzheimer’s disease.

Entrada sinks on mixed DMD data with ENTR-601-44

Thu, 07 May 2026 12:00:00 -0400

A lower-than-expected increase in dystrophin over baseline in the first and lowest-dose cohort of a phase I/II study of ENTR-601-44 in Duchenne muscular dystrophy (DMD) caused shares of Entrada Therapeutics Inc. to plunge more than 57%, despite the cohort meeting the safety and tolerability primary objective.

Apertura Gene Therapy and TSC Alliance partner on gene therapies

Wed, 06 May 2026 09:00:00 -0400

Apertura Gene Therapy LLC and the TSC Alliance have established a collaboration to advance gene therapy programs designed to treat tuberous sclerosis complex (TSC).

Elaaj Bio advances gene therapy for CDKL5 deﬁciency disorder

Wed, 06 May 2026 09:00:00 -0400

Elaaj Bio, a wholly owned subsidiary of the nonproﬁt Loulou Foundation, has entered into a partnership with Viralgen Vector Core SL to advance a gene therapy program for CDKL5 deﬁciency disorder.

AAV9-delivered AntagoNATs have preclinical efficacy as one-time treatment for Dravet syndrome

Mon, 04 May 2026 09:00:00 -0400

Dravet syndrome is a rare, severe, lifelong developmental and epileptic encephalopathy that begins in infancy and is marked by prolonged, often fever-triggered seizures that are difficult to control. It is usually caused by mutations in the SCN1A gene and is associated with developmental delay, cognitive and behavioral impairment, and reduced life expectancy.

Latus Bio series A financing supports early-stage programs

Mon, 04 May 2026 09:00:00 -0400

Latus Bio Inc. has closed a $97 million series A financing to support its broad therapeutics pipeline based on novel AAV capsid variants. Proceeds from the financing are expected to fund operations through milestones that include initial clinical data from the company’s two most advanced programs: LTS-201 for Huntington’s disease and LTS-101 for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease.

Dermatology specialist Leo Pharma makes a $50M move into gene therapy

Thu, 30 Apr 2026 11:00:00 -0400

Dermatology specialist Leo Pharma A/S is moving into gene therapy with the $50 million acquisition of Replay Holdings LLC, a seed-stage company that is developing high capacity herpes simplex virus vectors to treat rare inherited diseases.

Single-gene therapy for LSDs with modified lysosomal enzyme shows preclinical efficacy

Mon, 27 Apr 2026 09:00:00 -0400

In previous work, researchers from Kawasaki Medical School and collaborating institutions engineered a modified HEXB construct, modHexB, to improve GM2 ganglioside (GM2) recognition and GM2-activating protein (GM2A) interaction. The team has now combined these previous advancements to develop a new gene therapy strategy for Sandhoff disease.

A free gene therapy? Regeneron’s Otarmeni approved for hearing loss

Thu, 23 Apr 2026 12:00:00 -0400

Children and adults with a type of congenital hearing loss now have a free treatment option, with the U.S. FDA’s accelerated approval of Regeneron Pharmaceuticals Inc.’s DB-OTO, an AAV-mediated gene therapy. Branded Otarmeni (lunsotogene parvec), it is cleared specifically for hearing loss caused by variants in the otoferlin gene.

AAV-based gene therapy encoding NeuroD1 inhibits glioma growth and extends survival

Wed, 22 Apr 2026 09:00:00 -0400

Previous work showed that neurogenic transcriptional factors, such as NeuroD1 and Neurogenin 2, and small-molecule cocktails can reprogram glioma cells into neuron-like cells while also suppressing their proliferative and invasive phenotypes.

Entos and L-CMD Research Foundation collaborate

Tue, 21 Apr 2026 09:00:00 -0400

Entos Pharmaceuticals Inc. has established a collaboration with the L-CMD Research Foundation with the aim of developing a curative therapy for LMNA-related congenital muscular dystrophy (L-CMD).

Pleased to own ya, Kelonia: Lilly’s $7B CAR T deal

Mon, 20 Apr 2026 08:00:00 -0400

The already-thriving CAR T space took another big stride forward as Eli Lilly and Co. disclosed its plan to acquire Boston-based Kelonia Therapeutics Inc. for as much as $7 billion in cash, including an up-front payment of $3.25 billion, with the rest coming if clinical, regulatory and commercial goals are reached.

Roche plots route to EMA approval for DMD gene therapy

Fri, 17 Apr 2026 12:00:00 -0400

Roche Holding AG is making good on its promise to try and convince the EMA of the benefits of Elevidys (delandistrogene moxeparvovec), announcing a further global phase III trial of the Duchenne muscular dystrophy gene therapy.

New guidance advises on assessing safety of gene editing

Wed, 15 Apr 2026 09:00:00 -0400

The U.S. FDA’s latest draft guidance on gene therapies focuses on nonclinical studies using next-generation sequencing-based methods and bioinformatics to evaluate safety risks associated with off-target editing and loss of genome integrity in human gene-edited products.

New guidance advises on assessing safety of gene editing

Tue, 14 Apr 2026 12:00:00 -0400

MRD word heard loud, clear in Allogene LBCL cema-cel phase II

Mon, 13 Apr 2026 12:00:00 -0400

“Home-run” efficacy and what Cowen analyst Tyler Van Buren called “pristine” safety in the phase II Alpha3 study with CAR T therapy cemacabtagene ansegedleucel (cema-cel) sent shares of Allogene Therapeutics Inc. (NASDAQ:ALLO) skyward to a $4.46 high, up nearly 64%, on April 13. Shares eventually closed at $3.06, up 34 cents.

LHON updates: Gene therapy progress; idebenone receives US CRL

Thu, 02 Apr 2026 12:00:00 -0400

Chiesi Group’s idebenone faced a regulatory setback last month after the U.S. FDA issued a complete response letter (CRL) to the company’s NDA for Leber hereditary optic neuropathy (LHON), a rare inherited disorder that causes sudden vision loss.But a chance missed for Chiesi may be an opportunity for gene therapies, including Gensight Biologics SA’s lenadogene nolparvovec (Lumevoq; GS-010).

FDA approves Rocket’s Kresladi as first gene therapy for LAD-I

Fri, 27 Mar 2026 12:00:00 -0400

In a win for the rare disease space, the U.S. FDA granted accelerated approval for Rocket Pharmaceuticals Inc.’s Kresladi (marnetegragene autotemcel) as the first gene therapy option for treating severe leukocyte adhesion deficiency-I (LAD-I), an ultrarare genetic immune disorder characterized by an immunodeficiency predisposing those affected to recurrent and fatal infections.

Efficient editing in skeletal muscle of dystrophic mice with SORT LNPs encapsulating Cas9 mRNA

Wed, 25 Mar 2026 09:00:00 -0400

Gene editing holds promise for treating neuromuscular disorders such as limb-girdle muscular dystrophy, but its clinical translation remains challenging due to a lack of complementary delivery tools for the extensive network of skeletal muscles in the human body. A team at University of Massachusetts Chan Medical School compared editing outcomes mediated by either Cas9 mRNA and RNP delivery to skeletal muscle via local injection in the context of the previously described selective organ targeting (SORT) lipid nanoparticles (LNPs) platform.

Grant supports Tessera’s genetic approaches to HIV cure

Mon, 23 Mar 2026 09:00:00 -0400

Tessera Therapeutics Inc. has received a grant from the Gates Foundation to support early-stage research exploring multiple genetic approaches aimed at developing a scalable cure for HIV. This research will evaluate several potential strategies leveraging Tessera’s Gene Writing platform to engineer immune cells in vivo.

MDA: Vinay away, AA in play for Regenxbio gene therapy?

Thu, 12 Mar 2026 12:00:00 -0400

With top-line pivotal data with gene therapy RGX-202 for Duchenne muscular dystrophy (DMD) due in the next quarter, Regenxbio Inc. rolled out positive interim data from the phase I/II Affinity trial at the Muscular Dystrophy Association Clinical and Scientific Conference (MDA) in Orlando, Fla., where Bridgebio Pharma Inc., Capricor Therapeutics Inc., and Solid Biosciences Inc. also had clinical findings to talk about.