Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
AbCellera’s nonhormal menopause drug shows early success
On Monday, AbCellera announced exciting results from their Phase 2 study testing ABCL635, an investigational antibody targeting the neurokinin 3 receptor (NK3R), in women with menopause. The drug achieved best-in-class results for hot flashes, reducing the mean number per day down from 10 to 3.5, compared to 8.8 in the placebo group. The drug also resulted in improved sleep with no serious or severe adverse events. With this data so far pointing to a better efficacy and safety profile compared to the currently available Veozah from Astellas Pharma and Lynkuet from Bayer, AbCellera’s drug could soon be a lucrative addition to the market, providing a better option for many women. “If validated in Phase 3, ABCL635 could offer a new treatment option with a more convenient dosing regimen and potentially less toxicity,” said JoAnn Pinkerton, Professor of Obstetrics and Gynecology at the University of Virginia School of Medicine in the press release. – Allison Whitten
Skylark Bio advances gene therapy for genetic hearing loss
Skylark Bio has dosed the first patient in a Phase 1/2 trial of SKY-GJB2, an investigational gene therapy designed to treat GJB2-related hearing loss, the most common genetic cause of nonsyndromic pediatric deafness. The SONIX trial will evaluate the safety, tolerability, and preliminary efficacy of a single injection of SKY-GJB2 directly into the cochlea in children aged nine months to seven years, with preliminary data expected by the end of 2026. There are currently no approved disease-modifying treatments for GJB2-related hearing loss, with existing interventions such as hearing aids and cochlear implants helping patients access sound but not addressing the underlying genetic cause. The program enters the clinic following the recent approval of Otarmeni, an AAV-based gene therapy for OTOF-related genetic hearing loss, providing clinical and regulatory validation for targeted inner-ear gene delivery. Skylark plans to expand its genetic medicine platform into other monogenic hearing disorders, including SLC26A4-related hearing loss. – Bree Foster
Sangamo's bankruptcy auction sends key assets to PTC and Lilly
Sangamo Therapeutics, operating under Chapter 11 protection since June 2026, concluded a court-supervised Section 363 auction that will send its most valuable remaining programs to two acquirers for a combined $163.55 million in cash at closing, plus up to $100 million in milestones. PTC Therapeutics outbid original stalking-horse bidder Astellas Pharma to acquire Sangamo's Fabry disease gene therapy isaralgagene civaparvovec for $111 million upfront and up to $100 million tied to FDA approval milestones. Eli Lilly will pay $50 million for Sangamo's capsid delivery, zinc finger, and modular integrase platform technologies along with its prion disease program. The sales, still subject to Delaware bankruptcy court approval at hearings expected in the third quarter, mark a steep comedown for a company once seen as a gene-editing pioneer. They also highlight how bankruptcy auctions are increasingly becoming a channel through which larger, well-capitalized players like PTC and Lilly can pick up de-risked, late-stage genomic medicine assets at a fraction of their original development cost. — Andrea Corona
Patient groups flag safety signal for Neurocrine's PWS drug
Three Prader-Willi syndrome patient organizations issued a joint clinician statement flagging seven deaths and more than 100 serious adverse events, largely tied to fluid overload and related respiratory and cardiac complications, reported to the FDA's Adverse Event Monitoring System as of July 31, 2026, in patients taking Vykat XR (diazoxide choline). The drug is now under Neurocrine Biosciences, which gained it through its $2.9 billion acquisition of Soleno Therapeutics earlier this year, and remains the only approved treatment for hyperphagia in Prader-Willi syndrome. The groups did not establish that Vykat XR caused the events, given the complex underlying conditions common among affected individuals, and stopped short of recommending against its use, instead pointing clinicians toward new guidance on patient selection and monitoring for those with severe obesity, cardiac disease, edema, or untreated sleep apnea. It's a reminder for drug developers of how rare but serious safety signals can surface only after a first-in-class therapy for a small, medically complex population reaches broader real-world use, and a test of Neurocrine's ability to manage a young acquisition's biggest commercial asset through its first major post-market safety scrutiny. — Andrea Corona
Epicrispr raises $90 million for epigenetic editing therapy
Epicrispr Biotechnologies has raised $90 million to accelerate development of EPI-321, an investigational epigenetic editing therapy for facioscapulohumeral muscular dystrophy (FSHD), that is currently in an early stage clinical trial. EPI-321 uses CRISPR-based epigenetic editing to silence DUX4, a gene whose abnormal activation drives muscle cell death and degeneration in FSHD, without altering the underlying DNA sequence. The approach is designed to suppress production of the DUX4 protein, with early clinical data showing that three patients had improved lean muscle volume at six months compared to baseline. With no approved disease-modifying treatment currently available, several companies are pursuing different ways to suppress DUX4, including Novartis, Arrowhead Pharmaceuticals, Dyne Therapeutics, and Sarepta Therapeutics. The Series C financing, which brings Epicrispr's total funding since its inception to $213 million, will be used to further advance EPI-321, accelerate its pipeline of programmable epigenetic medicines, and expand its proprietary Gene Expression Modulation System platform and manufacturing capabilities. – Bree Foster
Definium’s LSD pill shows promise for anxiety
Early results from Definium Therapeutics’ Phase 3 Voyage Trial demonstrated a statistically significant and clinically meaningful improvement in anxiety scores on the Hamilton Anxiety Rating Scale compared to placebo in 214 adults. The LSD-based pill, DT120, is a partial agonist of serotonin-2A (5-HT2A) receptors. The positive results in anxiety add to the pill’s success in treating mental health conditions, as the company recently announced reduced symptoms of major depression this June. The timing could be ripe to fast-track approval of the pill after President Trump’s executive order to support the development and approval of psychedelic drugs earlier this year. “Importantly, the consistent, large effect size we’ve now observed across three studies underscores the potential of DT120 to transform psychiatry and usher in a new era of mental health care,” said Rob Barrow, CEO of Definium, in the press release. – Allison Whitten
