China’s NMPA Proposes 30-Day Review Pathway for Cell and Gene Therapies

Editas Medicine traded higher on Thursday after Australian authorities cleared a Phase 1/2 trial for the company's gene-editing therapy EDIT-401, and Wells Fargo upgraded the biotech to Overweight from Equal Weight in reaction. The Cambridge, Massachusetts-based company said the Human Research Ethics Committee of Australia approved the initiation of its Phase 1/2 Strive trial for EDIT-401 in patients with hyperlipidemia. Strive is designed to test a single dose of the in vivo gene editing medicine in heterozygous familial hypercholesterolemia, a genetic disorder characterized by high levels of low-density lipoprotein cholesterol, also known as bad cholesterol. Wells Fargo analyst Yanan Zhu called the initiation of what the company described as the first-in-human clinical trial of EDIT-401 a key step towards unlocking a highly differentiated LDL-C approach, and raised his price target on the stock to $6 from $4, reflecting revised probability of success assumptions for EDIT-401. Initial safety and tolerability data from Strive are expected in Q1 2027, ahead of topline safety and efficacy data from Part 1 of the study anticipated later that year.

Akamai Technologies surged 21.3% in premarket trading after announcing a seven-year, $11.6 billion cloud services commitment from AI company Anthropic, designed to support Anthropic's growing CPU workload demands across Akamai Cloud's distributed infrastructure. The deal includes provisions allowing the relationship to expand by up to an additional $9 billion, potentially taking the total commitment to about $20 billion, and Akamai also disclosed a hardware supply agreement with Lenovo and authorized contract manufacturer Jabil to procure about $1.7 billion in memory components. People Incorporated rose 6.5% in premarket trading after The Wall Street Journal reported that MGM Resorts International is weighing a potential takeover bid for Barry Diller's media conglomerate, with an offer potentially coming within days, just one day after People Incorporated withdrew its own $48.30-per-share cash proposal to acquire MGM's remaining public shares. Select Water Solutions rose 5.5% after announcing a definitive agreement to acquire Pilot Water Solutions for $700 million, plus up to $15 million in potential contingent consideration, in a transaction including $600 million in cash and $100 million in Class A common stock. Prime Medicine climbed 11.7% in after-hours trading to $3.49 after the U.S. Food and Drug Administration cleared its Investigational New Drug application for PM647, an investigational in vivo Prime Editor designed to correct the E342K mutation in the SERPINA1 gene, the root cause of Alpha-1 Antitrypsin Deficiency, paving the way for a global first-in-human Phase 1/2 clinical trial. On the downside, Comcast slipped 1.6% in premarket trading to $21.77 after KeyBanc downgraded the stock to Underweight from Sector Weight with an $18 price target, Twilio fell 3.7% after HSBC downgraded it to Reduce from Hold with a $211 price target, Zscaler fell 3.6% after naming Ross Tackett Chief Revenue Officer effective Oct. 1, succeeding Mike Rich, and Scholastic tumbled 11% after reporting fiscal first-quarter 2027 results that missed expectations, with revenue down 3.9% year over year to $216.8 million and an adjusted loss per share of $3.63.

Prime Medicine shares jumped 8% Friday after the FDA cleared its IND application for PM647, an experimental gene-editing therapy for Alpha-1 Antitrypsin Deficiency. The clearance allows Prime Medicine to begin a Phase 1/2 trial evaluating PM647 in adults with the inherited disorder, with initial clinical data expected in 2027. PM647 is designed to fix the genetic mutation that causes Alpha-1 Antitrypsin Deficiency, and Prime Medicine said the treatment could restore the normal protein and potentially help treat both the lung and liver problems caused by the disease.

REGENXBIO Inc. announced positive three-year long-term follow-up data from the Phase II ALTITUDE study of investigational surabgene lomparvovec, also known as sura-vec or ABBV-RGX-314, in non-proliferative diabetic retinopathy using suprachoroidal delivery, presented at the Retina Society 59th Annual Scientific Meeting in Los Angeles. In data as of August 17, 2026, 60% of all Dose Level 3 participants with three-year visits, or 6 of 10, achieved a greater than 2-step improvement on the Diabetic Retinopathy Severity Scale without additional treatment for diabetic retinopathy, and these participants experienced no vision-threatening events. Additionally, the majority of participants, 3 of 4, who achieved a 1-step DRSS improvement at one year without supplemental anti-VEGF injections went on to achieve a greater than 2-step DRSS improvement by three years without additional treatment. No new sura-vec-related safety signals and no intraocular inflammation were observed through three years in 17 participants receiving short-course prophylactic topical steroids. Dose Level 3 is being evaluated in the Phase IIb/III NAAVIGATE trial of sura-vec in NPDR, and REGENXBIO is developing sura-vec in collaboration with AbbVie.