Taysha Gene Therapies and Catalent have signed a commercial supply agreement for TSHA-102, Taysha’s investigational gene therapy for Rett syndrome, under which Catalent will serve as the primary commercial manufacturer following potential FDA approval. The agreement builds on a partnership that has supported TSHA-102 development since 2020 and secures long-term manufacturing capacity at Catalent’s FDA-licensed gene therapy facility in Harmans, Maryland. Catalent will provide GMP manufacturing and commercial supply, leveraging its experience across more than 90 gene therapy programs. Taysha’s Chairman and CEO Sean P. Nolan stated that establishing long-term commercial manufacturing capacity is a critical component of the company’s launch readiness strategy, with BLA-enabling Process Performance Qualification activities already underway. Rett syndrome, caused by mutations in the MECP2 gene, affects an estimated 15,000 to 20,000 patients in the U.S., EU, and U.K., and currently has no approved disease-modifying therapies.
Expands partnership to provide commercial manufacturing for Taysha's gene therapy, adding revenue potential.
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Akamai Surges 21.3% on $11.6 Billion Anthropic Cloud Deal
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 jump 8% as FDA clears PM647 trial
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 Reports Three-Year Durability Data for Surabgene Lomparvovec in Diabetic Retinopathy
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.
Biotech & Genomic Medicine › Gene & Cell Editing ▲Technology
Biotech & Genomic Medicine › Rare Disease Technology
RGNX · Technology · Positive REGENXBIO reported positive three-year Phase II ALTITUDE durability data for surabgene lomparvovec in diabetic retinopathy.
ABBV · Technology · Positive Positive three-year durability data for sura-vec (ABBV-RGX-314), which AbbVie is co-developing with REGENXBIO.
Novartis Pelacarsen Phase 3 Failure Reshapes Lp(a) Race for CRISPR and Ionis
Novartis AG's Phase 3 Lp(a)HORIZON trial of pelacarsen failed to produce a statistically significant reduction in major cardiovascular events despite significantly lowering lipoprotein(a), a setback Citi says carries implications for other Lp(a)-lowering developers. Citi believes the result increases the likelihood that CRISPR Therapeutics prioritizes its next-generation CTX321 program over the earlier candidate CTX320, which has generated Lp(a) reductions of as much as 73% during dose escalation; CTX321 uses an updated guide RNA that showed approximately twice the potency of CTX320 in preclinical testing, and CRISPR expects to provide a program update in 2026. Citi retained a Buy rating and an $88 price target on CRISPR Therapeutics. On Ionis Pharmaceuticals, which discovered pelacarsen and licensed it to Novartis in 2019 for worldwide development and commercialization, Citi analyst Eric Joseph expects less than 5% of immediate downside because investor expectations were already modest, and the firm does not expect the result to affect Ionis' fiscal 2026 guidance; Citi maintained a Buy rating and a $100 price target on Ionis. The broader concern is that pelacarsen's failure to translate Lp(a) reduction into fewer cardiovascular events raises questions about how much Lp(a) must be lowered, how long patients must be treated, and whether different therapeutic approaches can deliver better clinical outcomes, leaving CTX321's greater preclinical potency unproven in humans.
Biotech & Genomic Medicine › RNAi / Antisense Oligonucleotides ▼Demand
Biotech & Genomic Medicine › Gene & Cell Editing Technology
Biotech & Genomic Medicine › Cardiovascular & Heart-Failure Therapeutics Technology
NOVN.SW · Technology · Negative Novartis' Phase 3 Lp(a)HORIZON trial of pelacarsen failed to significantly reduce major cardiovascular events despite lowering Lp(a).
CRSP · Technology · Positive Citi says pelacarsen's failure increases likelihood CRISPR prioritizes its more potent next-gen CTX321 Lp(a) program, and retains Buy/$88 PT.
IONS · Technology · Negative Ionis discovered and licensed pelacarsen to Novartis, and its Phase 3 failure raises doubts about the Lp(a) approach, though Citi sees under 5% immediate downside.
Basecamp Research Raises $140M Series C to Advance AI-Designed Therapeutics
Basecamp Research has raised a $140M Series C financing to train a new generation of EDEN models and advance a pipeline of AI-designed therapeutics toward clinical development. The oversubscribed round was led by S32, with participation from Anthropic's Anthology Fund, Catalio Capital Management, European Tech Collective, Firebrand River Capital, Inception Fund, King Philanthropies, NATO Innovation Fund, NVIDIA, PostScriptum, Redalpine, The Rockefeller Foundation, Singular, Sovereign AI and True Ventures, plus additional investment from senior leaders across pharma, biotech and global industry including André Hoffmann, Vice-Chairman of Roche. The London- and Boston-based company is applying EDEN, its biological foundation model, to in vivo cell therapy, pairing the model's ability to design long and complex DNA sequences with large serine recombinases that can precisely integrate them into the genome, an approach it says could transform treatment of cancer and autoimmune disease. Basecamp has demonstrated strong preclinical results across multiple modalities and disease areas, and has appointed Richard Pearce, formerly of Biogen, as Chief Business Officer to expand its pharmaceutical partnerships. Andy Conrad, General Partner at S32 and former CEO of Google's Verily, joins the Board of Directors. EDEN models are trained on the Trillion Gene Atlas, which Basecamp calls the world's largest proprietary biological AI training dataset, built with partners including NVIDIA, Anthropic, PacBio and Ultima Genomics and drawing on data collected through access and benefit-sharing partnerships in more than 30 countries across all seven continents.
Artificial Intelligence › Foundation Models & Research Labs Capital
Artificial Intelligence › AI Tooling, Data & MLOps Technology
Basecamp Research · Capital · Positive Basecamp Research raised a $140M oversubscribed Series C to train EDEN models and advance its AI-designed therapeutics pipeline.
NVDA · Capital · Positive NVIDIA participates in Basecamp Research's $140M Series C and co-built the Trillion Gene Atlas training dataset.
Anthropic · Capital · Positive Anthropic's Anthology Fund participated in Basecamp Research's $140M Series C round.
ProQR Appoints Chris Hart as Chief Data and AI Officer, Thomas Wolf as Board AI Advisor
ProQR Therapeutics N.V. announced the appointment of Chris Hart as Chief Data and AI Officer and Thomas Wolf to its Board of Directors as strategic AI advisor, moves the company said will support its strategy to scale AI across drug discovery and development using its Axiomer RNA editing platform. Hart most recently served as Vice President of Data Science and AI/ML at Eli Lilly and Company and brings more than two decades of experience in computational biology, genomics, AI and oligonucleotide drug development, including more than 15 years focused on AI/ML-driven optimization of oligonucleotide therapeutics. Wolf is co-founder and Chief Science Officer of Hugging Face, the open-source AI platform that Nvidia recently agreed to acquire for $12.9B. ProQR said its AI-enabled discovery process identified AX-0811, its first program in clinical testing, which achieved approximately 60% editing efficiency in humanized models, around six-fold higher than the prior generation AX-0810, with reduced discovery timelines from three years to approximately three months. The company expects up to 5 clinical data readouts over the next 12 months across 4 development programs, with target engagement data from the first two cohorts of AX-0811 expected in early January 2027, and additional programs including AX-0422 for MPS I Hurler syndrome and AX-2911 for PNPLA3-associated MASH progressing toward the clinic.