Disease

Digital Health

Nyxoah raises $110m to accelerate Genio sleep apnoea launch in US

The Belgian medtech closed a $95m equity round and expects a $15m EIB loan tranche as it scales its FDA-approved Genio neurostimulator commercially.
The Belgian medtech closed a $95m equity round and expects a $15m EIB loan tranche as it scales its FDA-approved Genio neurostimulator commercially.
Cell & Gene Therapy

Cartesian licenses mRNA payload to WestGene for in vivo CAR-T

Cartesian Therapeutics and WestGene Biopharma will combine mRNA CAR-T payloads with targeted LNPs to run a Phase 1 MG trial from late 2026.
Cartesian Therapeutics and WestGene Biopharma will combine mRNA CAR-T payloads with targeted LNPs to run a Phase 1 MG trial from late 2026.
Genomics

CONNECTA Therapeutics starts Phase IIa trial of CTH120 in fragile X

The Barcelona biotech has dosed its first participants in a 30-patient study of TrkB modulator CTH120, the only compound in late-stage development for
The Barcelona biotech has dosed its first participants in a 30-patient study of TrkB modulator CTH120, the only compound in late-stage development for
Cell & Gene Therapy

Cullinan Therapeutics reports remissions in SLE and RA with CD19 TCE

Phase 1 data for CLN-978 and velinotamig show clinical remissions and complete renal responses in heavily pre-treated autoimmune patients.
Phase 1 data for CLN-978 and velinotamig show clinical remissions and complete renal responses in heavily pre-treated autoimmune patients.
Regulatory Science
Biotech

Humacyte ATEV beats AV fistula on catheter-free days in Phase 3

Humacyte's bioengineered vessel delivered 91 more catheter-free days than standard-of-care AV fistula in female dialysis patients, triggering a supplemental BLA filing.
Humacyte's bioengineered vessel delivered 91 more catheter-free days than standard-of-care AV fistula in female dialysis patients, triggering a supplemental BLA filing.
Biotech

Linnaeus Therapeutics' LNS8801 selected for NIA lifespan testing

The GPER agonist joins the NIA's Interventions Testing Programme, complementing a separate $22m ARPA-H contract to evaluate healthspan preservation.
The GPER agonist joins the NIA's Interventions Testing Programme, complementing a separate $22m ARPA-H contract to evaluate healthspan preservation.
Regulatory Science

Rafael Holdings completes Phase 3 enrolment for NPC drug Trappsol Cyclo

Rafael Holdings has reached last-patient-last-visit in its 94-patient TransportNPC trial, with topline data and an NDA submission both expected in H2
Rafael Holdings has reached last-patient-last-visit in its 94-patient TransportNPC trial, with topline data and an NDA submission both expected in H2
Biotech

Tectonic Therapeutic completes APEX Phase 2 enrolment for TX45

The Watertown biotech enrolled 191 patients across 14 countries; topline results for the PH-HFpEF candidate are expected in early Q1 2027.
The Watertown biotech enrolled 191 patients across 14 countries; topline results for the PH-HFpEF candidate are expected in early Q1 2027.
Regulatory Science

Teva closes $700m Emalex buy to add ecopipam for Tourette syndrome

Teva paid $700 million upfront for Emalex Biosciences, acquiring a Phase 3-complete D1 antagonist with an NDA filing expected in the second half of
Teva paid $700 million upfront for Emalex Biosciences, acquiring a Phase 3-complete D1 antagonist with an NDA filing expected in the second half of
Regulatory Science

Tris Pharma wins FDA Orphan Drug Designation for TRN-257 in IH

The New Jersey biotech's once-nightly, low-sodium oxybate candidate secured ODD based on its potential to eliminate the disruptive mid-night second dose.
The New Jersey biotech's once-nightly, low-sodium oxybate candidate secured ODD based on its potential to eliminate the disruptive mid-night second dose.
Cell & Gene Therapy

Actuate Therapeutics' elraglusib enters BEACON2 neuroblastoma trial

Elraglusib will be assessed alongside dinutuximab beta and chemotherapy in relapsed and refractory paediatric neuroblastoma across up to 60 international sites.
Elraglusib will be assessed alongside dinutuximab beta and chemotherapy in relapsed and refractory paediatric neuroblastoma across up to 60 international sites.
Regulatory Science

Alterity Therapeutics wins FDA alignment on ATH434 Phase 3 MSA trial

Alterity's End-of-Phase 2 meeting secured FDA agreement on trial design, dosing, and endpoints for its pivotal ATH434 programme in multiple system atrophy.
Alterity's End-of-Phase 2 meeting secured FDA agreement on trial design, dosing, and endpoints for its pivotal ATH434 programme in multiple system atrophy.
Biotech

Atavistik Bio wins FDA IND clearance and Fast Track tag for ATV-1601

The Cambridge biotech can now advance directly into a randomised Phase 1/2 study of its oral AKT1 inhibitor in hereditary haemorrhagic telangiectasia.
The Cambridge biotech can now advance directly into a randomised Phase 1/2 study of its oral AKT1 inhibitor in hereditary haemorrhagic telangiectasia.
Pharmaceuticals

Chiesi wins Health Canada priority review for FILSUVEZ in EB

Health Canada has accepted Chiesi's birch triterpenes gel for priority review, potentially making it the first therapy approved for EB wounds in Canada.
Health Canada has accepted Chiesi's birch triterpenes gel for priority review, potentially making it the first therapy approved for EB wounds in Canada.
Regulatory Science

Disc Medicine aligns with FDA on bitopertin CRL response path

Disc Medicine said a Type A FDA meeting confirmed the Phase 3 APOLLO study could support a traditional approval for bitopertin in EPP, with data
Disc Medicine said a Type A FDA meeting confirmed the Phase 3 APOLLO study could support a traditional approval for bitopertin in EPP, with data
Pharmaceuticals

Imviva Biotech wins FDA IND clearance for dual-target allogeneic CAR-T

CTA313 targets CD19 and BCMA in a Phase 1b basket trial spanning lupus, progressive MS, and autoimmune encephalitis.
CTA313 targets CD19 and BCMA in a Phase 1b basket trial spanning lupus, progressive MS, and autoimmune encephalitis.
Pharmaceuticals

Sanofi halts MOBILIZE Phase 3 trial of riliprubart in refractory CIDP

An independent data monitoring committee found the study unlikely to demonstrate sufficient efficacy; no safety concerns were identified with riliprubart.
An independent data monitoring committee found the study unlikely to demonstrate sufficient efficacy; no safety concerns were identified with riliprubart.
Cell & Gene Therapy

TG Therapeutics launches Phase 2 MG trial after positive BRIUMVI data

Subcutaneous BRIUMVI hit MCID in 82% of myasthenia gravis patients at Week 24, prompting a potentially registration-directed Phase 2 sequential-therapy trial.
Subcutaneous BRIUMVI hit MCID in 82% of myasthenia gravis patients at Week 24, prompting a potentially registration-directed Phase 2 sequential-therapy trial.
Cell & Gene Therapy

Windward Bio doses first patients in Phase 2 COPD study of WIN378

The Basel biotech has initiated SIRIUS, a Phase 2 COPD trial of its twice-yearly anti-TSLP antibody WIN378, alongside an ongoing asthma programme.
The Basel biotech has initiated SIRIUS, a Phase 2 COPD trial of its twice-yearly anti-TSLP antibody WIN378, alongside an ongoing asthma programme.
Genomics

Alkeus Pharmaceuticals doses first patient in Phase 3 Stargardt trial

The Cambridge biotech has initiated its pivotal NORTHSTAR study of oral gildeuretinol in Stargardt disease, with no approved treatment currently available.
The Cambridge biotech has initiated its pivotal NORTHSTAR study of oral gildeuretinol in Stargardt disease, with no approved treatment currently available.
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