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A one-patient ALS drug cuts a nerve-damage marker by up to 50%
An antisense drug designed for a single patient with CHCHD10-linked ALS cut a nerve-damage marker by up to 50% after six doses, Mayo Clinic reported in Med.
VectorY triples its PIONEER-ALS gene therapy trial to 36 patients
VectorY expanded the Phase 1/2 trial of VTx-002, a gene-delivered antibody against TDP-43 in ALS, from 12 to 36 participants before any efficacy data came in.
Human pituitary organoids restore a stress hormone in a monkey
Nagoya University transplanted stem-cell pituitary organoids under the skin of a macaque without a pituitary gland; they made ACTH for six weeks.
DeepMind releases SynthID Bio to watermark AI-designed proteins
The open-sourced method hides a detectable signature in protein designs and AlphaFold 3 structures, with no loss of binding in wet-lab tests.
iRegene photoreceptor cell therapy cleared for trials in US and China
NouvSight001, an off-the-shelf photoreceptor replacement for retinitis pigmentosa, has trial clearances from China NMPA and the US FDA.
Cartesian's CAR-T works again when myasthenia gravis returns
Five patients retreated with Descartes-08 a median 16.6 months after their first course all improved, with no serious adverse events through month 12.
uniQure's Huntington's gene therapy effect narrows at four years
At 48 months, high-dose AMT-130 slowed decline on the cUHDRS by 44% against an external control, down from 80% at 36 months, and missed significance on that measure.
UCSF becomes first US center to give nonprofit gene therapy Waskyra
Fondazione Telethon named UCSF Benioff Children's Hospitals on 28 September 2026 as the first US treatment center for Waskyra, a gene therapy for Wiskott-Aldrich syndrome.
FDA approves AbbVie's tavapadon, a once-daily Parkinson's pill
The FDA approved tavapadon, sold as Juvmo, for adults with Parkinson's disease on 25 September 2026. It is a once-daily dopamine D1/D5 partial agonist.
Carvykti leaves half a myeloma cohort treatment-free at five years
Five years after a single CAR-T infusion, half of CARTITUDE-2 Cohort A remained alive and progression-free with no maintenance therapy, J&J reported.
Kyverna CAR-T gains in stiff person syndrome hold at one year
In the 26-patient KYSA-8 trial, walking speed improved 49% at 12 months after a single miv-cel dose. Kyverna plans to complete its rolling BLA in Q4 2026.
Merck's remigromig matches ranibizumab in a 984-patient DME trial
The Wnt-pathway antibody met its visual-acuity endpoint in the pivotal BRUNELLO study of diabetic macular edema, with more retinal adverse events than the comparator.
Wiskott-Aldrich gene therapy benefits last up to 13 years
Of 27 children treated with etuvetidigene autotemcel in Milan, 96% were alive at five years and all had stopped immunoglobulin replacement.
Claude agents find a CRISPR-like enzyme system in phage DNA
Anthropic's new biology lab says 950 Claude agents flagged ART, an uncharacterised phage enzyme system with CRISPR-like repeats. Its function is still unknown.
Beacon's XLRP gene therapy meets its pivotal endpoint
In the 85-patient VISTA trial, 31% of high-dose laru-zova recipients gained 15 or more letters of low-light acuity at a year, against none of the untreated controls.
GC Cell's CD5 CAR-NK therapy draws responses in five of eight patients
Interim Phase 1a data on GCC2005, presented at ICBMT 2026 in Busan, show a 62.5% response rate in relapsed T-cell lymphoma with no ICANS or GvHD.
FDA approves the first drug for ataxia in ataxia-telangiectasia
The FDA approved IntraBio's Aqneursa (levacetylleucine) for ataxia in A-T patients of at least 15 kg, after a 73-patient crossover trial.
Korea clears a CAR-NK therapy for one refractory lymphoma patient
The MFDS approved compassionate use of GC Cell's GCC2005, an off-the-shelf CD5-targeting CAR-NK therapy, for a peripheral T-cell lymphoma patient out of standard options.
FDA approves the first gene therapy for Sanfilippo syndrome type A
Fayuvi, a single-dose AAV9 gene therapy from Ultragenyx, was approved on 17 September 2026 for children with MPS IIIA, a disease whose median life expectancy is 15 years.
A stem-cell therapy misses its endpoint in infant heart surgery
Longeveron reported on 16 September 2026 that laromestrocel did not improve right ventricular ejection fraction at 12 months in 40 infants with hypoplastic left heart syndrome.
Prime assembly inserts gene-sized DNA without double-strand breaks
A method published in Nature on 16 September 2026 stitches gene-sized DNA into a chosen genomic site using paired prime-editing flaps, and works in cells that are not dividing.
IASO Bio wins FDA clearance to test an in vivo CD20 CAR-T
IASO208 generates CD20-targeted CAR T cells inside the body from a single infusion, with no leukapheresis and no lymphodepleting chemotherapy.
Vyriad opens a Phase 1 trial of an in vivo CAR-T at Mayo Clinic
VV169 builds BCMA-targeted CAR T cells inside the patient, removing the weeks of ex vivo manufacturing that conventional CAR-T requires.
RegCell raises $66M to reprogram T cells into Tregs
A $44M Series A led by Playground Global and a second AMED grant fund a platform that converts pathogenic T cells into antigen-specific regulatory T cells.
FDA and Rocket agree on a path to finish the Danon trial
The pivotal Phase 2 of RP-A501 continues at a recalibrated dose with a 12-patient population and 12-month co-primary endpoints aimed at accelerated approval.
A sulfur switch decides when ageing brain proteins clump
Oxidation pushes synapsin 1 and G3BP2 into condensates as mice age, while hydrogen-sulfide persulfidation keeps them fluid — and ergothioneine reversed the effect in cells.
FDA approves the first muscle-targeted therapy for SMA
The FDA approved Scholar Rock's ISEMBYLD on 11 September 2026 for patients with spinal muscular atrophy aged two and older already taking an SMN-targeted drug.
FDA takes up the first in vivo CRISPR therapy for review
The FDA accepted Intellia's application for lonvo-z on 8 September 2026 with priority review and a target decision date of 10 March 2027.
FDA accepts Nanoscope's BLA for optogenetic therapy MOGENRY
The agency filed Nanoscope's application for sonpiretigene isteparvovec on 9 September 2026, a one-time injection for retinitis pigmentosa with severe vision loss.
Opus Genetics reports first vision gains from BEST1 gene therapy
Three of five patients dosed in the BIRD-1 trial gained visual acuity, and the FDA agreed on a retinal-sensitivity endpoint for a pivotal study.
Serina's long-acting apomorphine advances to a second dose cohort
An independent safety committee cleared dose escalation in the Phase 1b registrational study of SER-252, a subcutaneous polymer formulation for advanced Parkinson's.
Alzheon publishes ALZ-801 biomarker results in APOE4 carriers
Plasma p-tau217 fell on oral valiltramiprosate through week 78, and the size of the drop tracked with slower brain shrinkage and slower cognitive decline.
BioAge doses first patient in oral NLRP3 trial for macular edema
QUELL-DME will test whether a once-daily pill can hold vision in diabetic macular edema, including in patients who get no eye injections at all.
Base editing hit every PCSK9 allele in human embryos
A Nature paper published on 9 September 2026 reports editing at all PCSK9 alleles with no indels detected, alongside chromosome breakage the authors say precludes reproductive use.
Moonwalk raised $70M to shrink fat with RNAi, not appetite
A Series B co-led by Alpha Wave and YK Bioventures, with Eli Lilly participating, funds an siRNA aimed at adipose tissue toward first-in-human trials in late 2027.
Beam cut mutant Z-AAT by 84% in alpha-1 patients
Updated Phase 1/2 data presented at the ERS Congress on 8 September 2026 showed total AAT rising from 5.0 to 14.4 micromolar at the 60 mg dose.
BrainChild opens a pivotal CAR-T trial for DIPG
The ILLUMINATE Phase 2 study delivers B7-H3 CAR T cells straight into the cerebrospinal fluid of children with a brainstem tumour that has no cure.
Genflow's SIRT6 gene therapy met its endpoint in old beagles
The SLAB trial in 24 dogs over ten years old hit its primary endpoint of reduced biological age on the GRIM methylation clock, Genflow said on 8 September 2026.
Sernova and Seraxis merge to put islet cells in an implant
BetaNova Biotherapeutics pairs Sernova's implantable Cell Pouch with Seraxis' stem-cell-derived islets, with first patient dosing targeted for early 2027.
A base editor cleared sickle cell in a Nigerian patient
CorrectSequence extended its transformer base-editing therapy to four patients from Nigeria, Laos, Malaysia and Pakistan, reported in Cell Stem Cell on 7 September 2026.
Novartis's del-desiran misses its endpoint in myotonic dystrophy
The Phase III HARBOR study of del-desiran showed no statistically significant improvement in hand myotonia over placebo in about 150 people with DM1.
Six aging clocks read lower biological age in an AI drug trial
Insilico Medicine reported in Nature Biotechnology on 7 September 2026 that all six proteomic clocks applied to 42 patients on rentosertib pointed the same way.
A daily pill slowed retinal atrophy in Stargardt disease
Alkeus published TEASE-1 results in JAMA Ophthalmology on 3 September 2026: oral gildeuretinol cut lesion growth 21.6% over 24 months in 50 patients.
Intellia borrows up to $400m against a gene editing launch
A senior secured term loan from OrbiMed funds the US launch of lonvo-z, an in vivo CRISPR therapy for hereditary angioedema, without issuing new shares.
NeuShen raises over $80m for three brain drugs already in trials
The Shanghai and Boston company closed an oversubscribed Series B on 4 September 2026 to take schizophrenia, seizure and depression candidates into global studies.
Two patients moved from a pig kidney to a human one
eGenesis reported on 3 September 2026 that two of five recipients of its gene-edited pig kidney went on to human transplants, and three passed eight months without dialysis.
China clears a weekly at-home injection for early Alzheimer disease
The NMPA approved a subcutaneous lecanemab autoinjector on 3 September 2026, moving anti-amyloid treatment out of the infusion suite and into the home.
A natural enzyme reads an eight-letter genetic alphabet
UC San Diego showed with cryo-EM that ordinary E. coli RNA polymerase transcribes the synthetic Hachimoji alphabet accurately, published 2 September 2026.
New Zealand clears two psychiatrists to prescribe MDMA
Medsafe authorised two named clinicians to prescribe pharmaceutical-grade MDMA with psychotherapy for PTSD, while the drug itself stays unapproved.
CAR T cells built inside the body ease multiple sclerosis
A Wuhan team used an injected lentivirus to generate CD19-targeting CAR T cells in 16 patients, skipping the laboratory manufacturing step that conventional CAR-T requires.
FDA approves the first drug for Alexander disease
Zanvastro, an Ionis antisense drug that lowers GFAP production, was cleared on 3 September 2026 for children and adults with the rare leukodystrophy.
A pig kidney bridged a patient to a human transplant
A Lancet report from Mass General Brigham describes 271 dialysis-free days on a gene-edited pig kidney before the patient received a human organ.
An FDA clearance opens the first trial for Mucolipidosis type IV
The FDA let an IND proceed for an AAV9 gene therapy delivered into the brain for MLIV, a disease the ML4 Foundation says affects fewer than 100 known children worldwide.
A jumping-gene startup raises $56M to insert genes without viruses
Typewriter Therapeutics left stealth on 2 September 2026 with a $56M Series A for an R2 retrotransposon system that writes a therapeutic gene into a fixed target site from RNA.
uniQure files for approval of a Huntington's gene therapy
uniQure submitted a BLA to the FDA and an MAA to the UK MHRA on 2 September 2026 for AMT-130, a one-time AAV gene therapy delivered into the brain by neurosurgery.
The MHRA pulls a vasculitis drug over unreliable trial data
From 1 September 2026 Avacopan Vifor may not be supplied to new UK patients, after the MHRA found the pivotal study unreliable; the licence is revoked in March 2027.
ARPA-H funds five teams to make RNA medicines on demand
The GIVE program commits up to $125 million to build distributed, automated manufacturing for individualized RNA-based genetic medicines.
An oral BTK inhibitor cuts MS relapse rates in two Phase III trials
Novartis reported that remibrutinib beat teriflunomide on annualized relapse rate across REMODEL-1 and REMODEL-2, without releasing the numbers.
Metal from joint replacements is reaching the brain, autopsies show
A Rush University study of 701 donated brains found implant-derived cobalt and titanium in brain tissue, with cobalt tracking higher Alzheimer's pathology but no cognitive decline.
Lab-grown nerve hits human sensory conduction speed on a chip
A Rice and ETH Zurich hydrogel platform kept human neurons alive past 100 days and measured myelinated signals at human sensory nerve velocities.
Novartis halts eight CAR-T trials after three immune deaths
Novartis paused its rap-cel autoimmune programme on 24 August after three cases of a severe immune reaction; Bristol Myers Squibb paused enrolment in its own trials.
A single CRISPR infusion held lipids down for a year
CTX310 cut ANGPTL3 by a mean 79 percent at one year in the highest-dose cohort, with results published in the New England Journal of Medicine.
CAR-T therapy frees three rheumatoid arthritis patients from drugs
Six patients whose disease had resisted up to eight biologics received anti-CD19 CAR T cells at Charite; three reached drug-free remission within a year.
A nanogel turns support cells into neurons in Alzheimer's mice
Nano-ERASER crosses the blood-brain barrier and degrades PTBP1, reprogramming astrocytes into neurons; treated mice rebuilt nests and solved water mazes.
An injected muscle graft that exercises itself, tested in mice
Chinese Academy of Sciences researchers report myografts that self-assemble under the skin, contract on their own, and improve bone, liver and cognitive measures.
Mitochondria from a woman's leg were injected into her eyes
A Mount Sinai team reports the first intravitreal mitochondrial transplant, with pupil responses to light returning for days to weeks, in a preprint case report.
Precision BioSciences doses the first patient in its Duchenne trial
PBGENE-DMD entered a Phase 1/2 study at Arkansas Children's Hospital, aiming to restore near full-length dystrophin instead of a truncated substitute.
FDA halts Regenxbio's Hunter syndrome gene therapy a second time
Spinal masses found on MRI in five participants treated three to six years ago led the agency to pause RGX-121 again, and the company shelved its resubmission.
US lawmakers ask the FDA to stop taking Chinese trial data on trust
Two representatives want China-generated data rejected unless the trial site has had an FDA audit within twelve months, citing three deaths in gene-editing and cell-therapy trials.
Regeneron wins approval for a drug that slows bone invading muscle
Pasatru blocks Activin A and cut new heterotopic ossification lesions by 90% or more at 56 weeks in adults with fibrodysplasia ossificans progressiva.
First gene therapy approved for glycogen storage disease type Ia
Genglycos won accelerated approval on 19 August 2026, cutting daily cornstarch intake by 31% against placebo in a 48-week randomised trial.
An HIV drug reaches a Rett syndrome trial by way of orbit
Brain organoids flown to the ISS aged faster than Earth controls; the mechanism they exposed led to a clinical trial of lamivudine, now under way.
FDA fast-tracks an off-the-shelf Parkinson cell therapy
XS411, an allogeneic iPSC-derived neural progenitor injection, received Fast Track designation on 4 August 2026 with its China Phase II fully enrolled.
Alabama CHIP pays the way to a gene therapy for deafness
A child covered by Alabama ALL Kids received Otarmeni, the first FDA-approved gene therapy for OTOF-related hearing loss, at Boston Children's Hospital.
Ohio State wraps gene therapy vectors in red blood cell membrane
Vesicles built from red blood cell lipids carried AAV past neutralising antibodies and still delivered their genetic cargo in mice.
A 3D-printed membrane raises artificial-lung oxygen transfer by 88%
Hannover Medical School and RWTH Aachen printed a silicone gas-exchange membrane on a minimal-surface geometry instead of hollow fibres, reported in Advanced Materials on 18 August 2026.
Mesoblast finishes dosing its Phase 3 disc-injection cell therapy
All 350 patients in MSB-DR004 have received a single intra-discal injection of rexlemestrocel-L or a sham, with the 12-month pain endpoint reading out in mid-2027.
A two-cell corneal graft improves sight in aniridia keratopathy
Nine adults received a collagen scaffold carrying both limbal epithelial stem cells and stromal keratocytes; treated eyes improved on the ocular surface score while untreated fellow eyes did not.
IASO Bio buys Singapore cell-therapy company MediSix
The Nanjing CAR-T maker completed its acquisition of MediSix Therapeutics on 13 August 2026, taking over its PEBL platform and the IASO107 programme.
A CRISPR-armed phage clears a drug-resistant infection in one patient
A kidney transplant recipient's malakoplakia mass fell from 745 cm3 to 82 cm3 over a year after SNIPR001 was added to antibiotics under an emergency IND.
Base editing cuts toxic huntingtin in mice and holds for a year
AAV9-delivered adenine base editors skipped HTT exon 13 in YAC128 mice, cutting toxic huntingtin fragments 60% and improving grip and rotarod at 12 months.
An off-the-shelf CAR T therapy is dosed as a same-day outpatient treatment
Fate Therapeutics dosed the first patient in RECLAIM-LN, a Phase 2 trial of the iPSC-derived CAR T cell FT819 in lupus nephritis, and discharged them the same day.
A cognition drug platform changes hands for $12 million down
AlzeCure Pharma received the full USD 12 million upfront and closed the initial transaction licensing its NeuroRestore platform and ACD856 to QuantumCell of Denmark.
A youth-associated blood protein restored debris clearance in old brains
Mount Sinai researchers report in Nature Communications that TIMP2 governs microglial state, and that giving it to aged mice restored their capacity to clear debris.
ReCode finishes enrolling its inhaled mRNA trial in cystic fibrosis
The Phase 2a study of RCT2100 is fully enrolled and running, with data expected in the fourth quarter of 2026.
A halo of IL-10 kept implanted insulin cells working past 100 days
Rice bioengineers co-encapsulated immune-calming cells with beta cells, holding glucose control in diabetic mice about five times longer than unprotected implants.
Aspen's Parkinson's cell therapy takes an RMAT designation
The FDA granted regenerative medicine advanced therapy status to sasineprocel, an autologous neuron therapy already in a Phase 1/2a trial.
A three-drug mix extended old mice lifespan by 42 percent
Berkeley researchers combined dichloroacetate, metformin and a low dose of navitoclax, and aged mice lived a median 1,002 days against 815 for controls.
Epicrispr raises $90 million to switch off the gene behind FSHD
The Series C funds pivotal studies of EPI-321, which silences DUX4 epigenetically rather than cutting DNA, after a Phase 1/2 trial reported muscle-volume gains.
A gene therapy for inherited deafness reaches its first child
Skylark Bio dosed the first patient in SONIX, a Phase 1/2 trial of SKY-GJB2, delivered as a single injection into the cochlea in children from nine months old.
Remedium Bio closes the first $10 million of its Series A
Lifespan Vision Ventures led the initial close, with Eli Lilly participating, for a platform that turns fat cells into adjustable factories for therapeutic proteins.
Beam completes dosing in its sickle cell BEACON trial
Every adult and adolescent enrolled in the Phase 1/2 trial of risto-cel has now been dosed, with a BLA possible as early as the end of 2026.
Precision BioSciences reports first biopsy evidence of cccDNA editing
A liver biopsy in the ELIMINATE-B trial showed a 1-log fall in cccDNA-derived transcripts after two doses of PBGENE-HBV, measured in tissue rather than in blood.
A child died in a Shanghai base-editing trial, undisclosed for a year
A 6-year-old girl died a week after an intrathecal base-editing infusion at Xinhua Hospital in March 2025; the death surfaced only in a July 2026 investigation.
A genome language model designed working viruses from scratch
Stanford used Evo 2 to generate bacteriophage genomes; of nearly 300 synthesised, 16 killed E. coli, and some outperformed the natural virus they were modelled on.
Beam doses the first patient in a pivotal base-editing trial
BEAM-302 rewrites the SERPINA1 mutation in the liver; the pivotal cohort of its Phase 1/2 trial began dosing in July and was disclosed on 4 August.
CRISPR Therapeutics reports FDA approval of Casgevy from age two
The company says the FDA cleared its gene-edited sickle cell and beta thalassemia therapy for children aged 2 and older, making about 5,500 more patients eligible.
CRISPR Therapeutics starts Phase 1 trials for hypertension and AATD
Two in vivo gene-editing candidates entered first-in-human testing: CTX340 against angiotensinogen, and CTX460, the first candidate from the SyNTase platform.
Intellia ties its worst liver signals to a single HLA allele
A genomic analysis of more than 600 samples found the highest liver enzyme elevations in Intellia in vivo CRISPR trials clustered in carriers of one HLA allele.
HuidaGene discloses a CRISPR trial death twelve months late
HuidaGene Therapeutics waited a year to disclose that a boy in the high-dose cohort of its Duchenne gene-editing trial died in August 2025.