Vertex Pharmaceuticals logo

Vertex Pharmaceuticals

PublicFounded 1989🇺🇸Boston, Massachusetts
CompareVisit website
CEO

Reshma Kewalramani

Data verified as of Sep 2, 2026

Summary

Vertex Pharmaceuticals is the world leader in cystic fibrosis (CF) and one of biotech's few large-cap, highly profitable, self-funded innovators. Its CFTR-modulator franchise - anchored by Trikafta/Kaftrio and the next-generation once-daily Alyftrek (vanzacaftor/tezacaftor/deutivacaftor, FDA-approved December 2024) - treats the large majority of the addressable CF population and generated the bulk of $12.0B in FY2025 revenue, with $4.0B of GAAP net income. Vertex is now deploying that cash flow to diversify well beyond CF. It commercialized Casgevy (exagamglogene autotemcel), the first CRISPR/Cas9 gene-editing therapy ever approved, for sickle cell disease and beta thalassemia, partnered with CRISPR Therapeutics. In January 2025 it launched Journavx (suzetrigine), the first genuinely new class of pain medicine in over two decades - an oral, non-opioid NaV1.8 inhibitor. Its pipeline includes zimislecel (VX-880), a stem-cell-derived islet therapy that has rendered type 1 diabetes patients insulin-independent in trials; inaxaplin for APOL1-mediated kidney disease; and povetacicept (from the $4.9B Alpine acquisition) for IgA nephropathy. On September 1, 2026 it completed the ~$10B acquisition of endocrine-disease specialist Crinetics Pharmaceuticals - its largest-ever deal - adding the marketed acromegaly drug Palsonify and pipeline candidate atumelnant, and expanded its executive leadership team to run the new endocrine business. Vertex pushes the envelope by turning single-gene biology into functional cures.

Company OverviewRead the long-form profile of Vertex Pharmaceuticals

/ Recent News

View all →

  • View all news

    Browse the daily archive

Main Products

Trikafta / Kaftrio

Trikafta / Kaftrio

Active

Vertex's flagship cystic fibrosis triple-combination CFTR modulator (elexacaftor/tezacaftor/ivacaftor), effective for roughly 90% of CF patients and the company's dominant revenue engine - now complemented by the once-daily next-generation Alyftrek.

The CF standard of care worldwide. Vertex is converting patients onto the once-daily Alyftrek (approved December 2024) and expanding ex-US launches and younger-age labels to sustain franchise growth.

US CF Revenue (2025)$7.55B (+13% YoY)
Patient Coverage~90% of CF patients (Trikafta)

Journavx (suzetrigine)

Active

A first-in-class oral, non-opioid pain medicine (VX-548) that selectively inhibits the NaV1.8 sodium channel to block pain signals in the peripheral nervous system before they reach the brain - the first new class of pain drug in over 20 years.

FDA-approved January 30, 2025 for moderate-to-severe acute pain and launching commercially, with label-expansion studies into chronic pain (including diabetic peripheral neuropathy) under way.

FDA ApprovalJan 2025 (acute pain)
MechanismSelective NaV1.8 inhibitor (non-opioid)

Casgevy (exa-cel)

Active

The first approved CRISPR/Cas9 gene-editing medicine (exagamglogene autotemcel), a one-time therapy for sickle cell disease and transfusion-dependent beta thalassemia, developed and commercialized with CRISPR Therapeutics.

Approved across multiple jurisdictions and priced at ~$2.2M per one-time treatment. Vertex books the revenue (~$116M in FY2025) and is scaling a global network of authorized treatment centers; on July 1, 2026 the US label expanded to patients ages 2+ (from 12+) for both sickle cell disease and transfusion-dependent beta thalassemia - the first gene therapy cleared for children as young as two - and Vertex guides Casgevy revenue to roughly triple in 2026.

First ApprovalNov 2023 (UK); Dec 2023 (US)
Patients Started>500 cumulative (Q1 2026)

Zimislecel (VX-880)

In Development

An allogeneic, stem-cell-derived, fully differentiated islet cell therapy for type 1 diabetes designed to restore the body's own insulin production - a potential functional cure delivered as a one-time infusion.

In a pivotal Phase 1/2/3 program with FDA RMAT and Fast Track, EMA PRIME, and a UK Innovation Passport. All 10 participants in the reported cohort became insulin-independent at one year with >90% time-in-range; global regulatory submissions are expected in 2026.

Phase 1/2 Result10/10 insulin-independent at 1 year
ModalityStem-cell-derived islet cell therapy

Palsonify (paltusotine)

Active

An oral, once-daily somatostatin receptor agonist for acromegaly, acquired through the Crinetics Pharmaceuticals deal - Vertex's first marketed product outside CF, pain, and gene therapy, and the anchor of its new endocrine-disease business.

Approved in the US in September 2025 and already marketed; joined Vertex's portfolio when the Crinetics acquisition closed on September 1, 2026.

IndicationAcromegaly

Atumelnant

In Development

An oral pipeline candidate for endocrine disease acquired with Crinetics Pharmaceuticals, part of the endocrinology pipeline Vertex and Crinetics said could reach $5B+ in combined peak annual sales alongside Palsonify.

Joined Vertex's pipeline when the Crinetics acquisition closed on September 1, 2026; clinical stage and timelines to be detailed as Vertex integrates the endocrinology business.

What's Next

Zimislecel type 1 diabetes regulatory submission

File global regulatory submissions for the stem-cell islet therapy that has produced insulin-independence - potential functional cures - in trial participants, a landmark for cell therapy. Vertex temporarily postponed completion of dosing in early 2026 pending an internal manufacturing analysis, has since completed that analysis and resumed dosing, and continues to enroll and dose patients in the Phase 1/2/3 study. In parallel, the FDA cleared an IND for VX-017, a 'type O' universal-donor islet cell therapy, and Vertex is now weighing bringing VX-017 to market at or near the same time as zimislecel rather than sequencing the two. The company says it will provide updated regulatory and commercial timelines for the whole type 1 diabetes portfolio later in 2026; no BLA/MAA has been filed for either program as of late August 2026.

2026–2027 (at risk)

Povetacicept FDA decision in IgA nephropathy

Await the FDA decision on povetacicept (from the Alpine acquisition) for IgA nephropathy after Phase 3 RAINIER showed a 52% reduction in proteinuria; the BLA was accepted under accelerated approval (using a priority review voucher to cut review time from ten months to six) with a PDUFA target action date of November 30, 2026. If approved, povetacicept becomes Vertex's first commercialized nephrology product.

Nov 30, 2026

Inaxaplin APOL1 kidney disease Phase 3 interim analysis

Advance the AMPLITUDE Phase 2/3 trial of inaxaplin (VX-147), a once-daily oral therapy that could be the first treatment targeting the underlying cause of APOL1-mediated kidney disease (now expanded to include adolescents ages 10-17). Vertex has completed enrollment of the interim-analysis cohort and expects the pre-planned Week 48 interim analysis - which could support a US accelerated approval filing if positive - in late 2026 or early 2027; full study enrollment (~466 patients) remains on track to complete in the second half of 2026.

2H 2026 (full enrollment); interim analysis early 2027

Journavx Phase 3 studies in diabetic peripheral neuropathy

Broaden Journavx beyond acute pain into chronic pain. After the FDA indicated it did not see a path to a broad peripheral-neuropathic-pain label, Vertex narrowed its near-term plan to two identical, roughly 1,100-patient Phase 3 studies specifically in diabetic peripheral neuropathy (DPN) at a once-daily 70mg dose, with enrollment in both expected to complete by the end of 2026.

End of 2026 (DPN Phase 3 enrollment)

Alyftrek CF conversion and ex-US rollout

Convert Trikafta patients onto the once-daily Alyftrek and expand international launches to extend the cystic fibrosis franchise. Alyftrek is now approved in the US, UK, EU (covering an addressable population of about 31,000 CF patients), Canada, Switzerland, Australia and New Zealand. Vertex signed a Letter of Intent with the pan-Canadian Pharmaceutical Alliance on July 6, 2026 following positive reimbursement recommendations from CDA-AMC and INESSS, and has secured reimbursement agreements in Scotland, Spain, Sweden, Switzerland, New Zealand, Israel and Finland, with more countries in progress. Vertex also plans global regulatory submissions to extend the label to children ages 2-5.

2026 ongoing

Integrate Crinetics and launch the endocrine-disease business

Having closed the ~$10B Crinetics acquisition on September 1, 2026, Vertex must integrate the Palsonify (paltusotine) commercial business and the atumelnant pipeline into its operations under an expanded executive leadership team - Jonathan Poole as EVP and Chief Financial Officer and Jasper van Grunsven joining as EVP, Chief Pain and New Product Planning Officer - establishing rare endocrine disease as a fifth growth pillar alongside cystic fibrosis, hematology, acute pain, and renal disease.

Late 2026

Track Record

1 of 1 announced date hit

Close the ~$10B Crinetics acquisition

Target Early September 2026 · Resolved Sep 1, 2026

Vertex completed the acquisition of Crinetics Pharmaceuticals on September 1, 2026 for about $10.0 billion in total equity value (~$8.8 billion net of acquired cash), adding Palsonify (paltusotine) and atumelnant, and announced an expanded executive leadership team - Jonathan Poole as EVP and Chief Financial Officer and Jasper van Grunsven joining as EVP, Chief Pain and New Product Planning Officer - to run the new endocrine-disease business.

Hit

Operations & Revenue

StatusProfitable, commercial-scale

Vertex runs a dominant, high-margin cystic fibrosis franchise (US CF revenue rose 13% to $7.55B in 2025) that funds an aggressive diversification into non-opioid pain, CRISPR gene therapy, type 1 diabetes cell therapy, and kidney disease. Q2 2026 revenue rose 12% to $3.33B with net income of $1.10B and EPS of $4.34, prompting a raised full-year 2026 guidance of $13.1-13.2B; CASGEVY and JOURNAVX are now expected to contribute over $500 million combined for the year, growing 151% and roughly 4x YoY respectively in the quarter. FY2025 GAAP net income of $4.0B and a $2.0B annual buyback underline a rare profitable, self-funded innovator at biotech's frontier. The nearest credible CF challenger, Sionna Therapeutics, halted its lead program after a Phase 2 failure in August 2026, leaving Vertex's CF franchise uncontested; the pending ~$10B Crinetics acquisition (Q3 2026) will draw down cash.

Revenue Streams

Cystic fibrosis franchise

The overwhelming majority of revenue: Trikafta/Kaftrio plus the next-generation once-daily Alyftrek. US CF revenue grew 13% to $7.55B in 2025, with further growth from ex-US Alyftrek launches and label expansions to younger patients.

Journavx (acute pain)

Vertex's newly launched non-opioid pain medicine, generating early commercial revenue as it builds formulary access; more than 1 million prescriptions had been written by early 2026.

Casgevy (gene therapy)

The CRISPR gene-editing therapy for sickle cell disease and beta thalassemia, co-commercialized with CRISPR Therapeutics; Vertex books the product revenue (~$116M in FY2025) as authorized treatment centers scale globally.

Emerging (diabetes & kidney disease)

Future streams from zimislecel (type 1 diabetes), inaxaplin (APOL1-mediated kidney disease), and povetacicept (IgA nephropathy) as these programs approach the market.

Key Metrics

Employees

~6,400

Est. Annual Revenue

$3.33B in Q2 2026 (+12% YoY); $12.0B FY2025; 2026 guidance raised to $13.1-13.2B

Q2 2026 Revenue

$3.33B (+12% YoY); 2026 guidance raised to $13.1-13.2B

FY2025 GAAP Net Income

$4.0B (rebound from 2024 IPR&D-driven loss)

Cash & Investments

$13.6B (Jun 30, 2026); expected to fall materially after the ~$8.8B net Crinetics purchase closes

Market Cap

~$128.7B (Aug 14, 2026)

FY2025 R&D Spend

$3.9B (GAAP); Q2 2026 R&D $889M (+1% YoY)

Journavx Uptake

$50M Q2 2026 product revenue, up 71% sequentially and more than 4x YoY; ~535,000 prescriptions filled in Q2 2026 alone, nearing 1 million cumulative prescriptions

Casgevy Revenue

$76M in Q2 2026, up 151% YoY and 78% sequentially; third consecutive quarter with 100+ patient initiations, with more infusions in H1 2026 than all of 2025 combined

Approved Products

5 CF modulators + Journavx (pain) + Casgevy (gene therapy, now ages 2+)

Timeline

2026Record year: $12.0B revenue, $4.0B net income

Reports FY2025 revenue of $12.0B (+9%) and GAAP net income of $4.0B, guiding to $12.95–13.1B for 2026 including $500M+ from non-CF products, and continuing a $2.0B annual share-buyback program.

2026FDA expands Casgevy to children ages 2+

On July 1, 2026 the FDA approves expanded use of Casgevy (exa-cel) for both sickle cell disease and transfusion-dependent beta thalassemia in patients ages 2 and older - down from 12+ - making it the first gene therapy cleared for children as young as two and adding roughly 5,500 newly eligible U.S. children. Vertex and partner CRISPR Therapeutics guide Casgevy revenue to roughly triple in 2026 as authorized treatment centers scale.

2026Agrees to acquire Crinetics for ~$10B - largest deal ever

On July 6, 2026 Vertex agrees to acquire endocrine-disease specialist Crinetics Pharmaceuticals for $85 per share in cash (~$10B equity value, ~$8.8B net of cash; about a 102% premium) - its largest acquisition to date. The deal adds Palsonify (paltusotine), an oral once-daily acromegaly drug approved in September 2025, plus an endocrinology pipeline (including atumelnant) the companies say could deliver $5B+ in combined peak annual revenue. Structured as a one-step merger requiring a Crinetics shareholder vote - Crinetics filed a preliminary proxy in July 2026 - plus antitrust clearances, and funded from cash on hand plus a committed $4.5B bridge facility. Closing is expected in Q3 2026 and had not yet completed as of late July.

2026Partners with AbCellera on autoimmune T-cell engagers

On July 29, 2026 Vertex enters a collaboration with AbCellera to discover multispecific T-cell engagers for autoimmune diseases and other conditions. AbCellera leads early discovery on its proprietary TCE platform while Vertex funds all R&D and holds development and commercialization rights, paying AbCellera $28M upfront plus preclinical, development, regulatory and commercial milestones and tiered sales royalties.

2026Q2 2026 results beat, guidance raised

Vertex reported Q2 2026 revenue of $3.33 billion on August 3, up 12% year over year, with net income of $1.10 billion and EPS of $4.34. The company raised full-year 2026 revenue guidance to $13.1-13.2 billion, driven partly by CASGEVY revenue growing 151% year over year to $76 million and JOURNAVX revenue roughly quadrupling to $50 million. ALYFTREK, the once-daily next-generation CF modulator, surpassed $1 billion in cumulative revenue in the first half of 2026 on rapid uptake in the US, Germany, and the UK.

2026Closest cystic fibrosis rival fails Phase 2, shares hit all-time high

Sionna Therapeutics said on August 10, 2026 that its cystic fibrosis add-on candidate SION-719 - designed to be paired with Vertex's Trikafta and the closest credible challenge to Vertex's CF franchise - missed the primary endpoint in its Phase 2a trial, and the company halted the program; Sionna's stock lost roughly 90% of its value in a day. Vertex shares rose to an all-time high on the news, underscoring how uncontested its cystic fibrosis franchise remains.

2026Loses patent case, clearing a path for generic Kalydeco

The US District Court for the District of Delaware ruled on August 24, 2026 that Lupin's proposed generic version of Vertex's cystic fibrosis drug Kalydeco (ivacaftor) does not infringe Vertex's patents. Judge Stephanos Bibas found that Lupin's formulation, containing about 74% ivacaftor, falls outside the 'about 80%' concentration range claimed in Vertex's patents, rejecting Vertex's broader interpretation of 'about.' The ruling clears a legal hurdle for Lupin, a first-to-file applicant that already holds tentative FDA approval and could gain 180 days of marketing exclusivity for the generic pending final FDA approval - the first real threat to a Vertex CF modulator's exclusivity, though Kalydeco is now an older, smaller part of the CF franchise next to Trikafta and Alyftrek.

2026Completes $10B Crinetics acquisition, enters endocrine disease

Vertex completes its acquisition of Crinetics Pharmaceuticals on September 1, 2026 for about $10.0 billion in total equity value (~$8.8 billion net of acquired cash), adding the marketed acromegaly drug Palsonify (paltusotine) and pipeline candidate atumelnant. Vertex expands its executive leadership team to run the new business, naming Jonathan Poole EVP and Chief Financial Officer and adding Jasper van Grunsven as EVP, Chief Pain and New Product Planning Officer.

2025Journavx (suzetrigine) - first new pain-drug class in 20+ years

On January 30, 2025 the FDA approves Journavx (suzetrigine), a first-in-class oral, non-opioid NaV1.8 inhibitor for moderate-to-severe acute pain - the first genuinely new class of pain medicine in more than two decades.

2025Zimislecel delivers functional cures in type 1 diabetes

At the June 2025 ADA meeting, Vertex reports that all 10 type 1 diabetes participants dosed with zimislecel became insulin-independent at one year with >90% time-in-range; the data are published in the New England Journal of Medicine.

2024Alpine Immune Sciences acquisition ($4.9B)

Acquires Alpine Immune Sciences for ~$4.9B, adding povetacicept (a BAFF/APRIL inhibitor) and a broader immunology and nephrology pipeline. The one-time acquired-IPR&D charge drove a temporary GAAP net loss that year.

2024Alyftrek - next-generation once-daily CF combo approved

The FDA approves Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) in December 2024, a once-daily next-in-class CFTR modulator that Vertex begins converting patients onto to extend its CF franchise.

2023Casgevy becomes the world's first approved CRISPR medicine

Casgevy (exa-cel), developed with CRISPR Therapeutics, is authorized by the UK MHRA in November 2023 and approved by the FDA for sickle cell disease in December 2023 - the first CRISPR/Cas9 gene-editing therapy ever approved.

2019Trikafta approved - the transformational triple combo

The FDA approves Trikafta (elexacaftor/tezacaftor/ivacaftor), a triple-combination modulator that is effective for roughly 90% of CF patients and becomes Vertex's revenue engine.

2019Acquires Semma Therapeutics for diabetes cell therapy

Buys Semma Therapeutics for ~$950M, entering stem-cell-derived islet therapy for type 1 diabetes - the program that became zimislecel (VX-880).

2015Orkambi and the CRISPR Therapeutics partnership

Launches Orkambi, its first CFTR combination therapy, and forms a gene-editing partnership with CRISPR Therapeutics that would later produce Casgevy.

2012Kalydeco - first drug to treat CF's underlying cause

The FDA approves Kalydeco (ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis rather than just its symptoms, and the first CFTR potentiator.

1991IPO on NASDAQ (VRTX)

Goes public on NASDAQ under ticker VRTX, funding an early pipeline that spanned HIV and hepatitis C before its pivot to cystic fibrosis.

1989Founded to industrialize structure-based drug design

Joshua Boger and Kevin Kinsella found Vertex in Cambridge, Massachusetts, to transform the treatment of serious diseases through rational, structure-based drug design.

Funding

Cumulative disclosed raise · dated rounds

$10B$20B2020202220242026$16B raised
RoundDateAmountInvestorsSource
IPO (NASDAQ: VRTX)1991Public since 1991Public markets; now a Nasdaq-100 and S&P 500 component
Semma Therapeutics acquisition2019~$950MCapital deployment (type 1 diabetes cell therapy)
ViaCyte acquisition2022~$320MCapital deployment (diabetes cell-therapy assets)
Alpine Immune Sciences acquisition2024~$4.9BCapital deployment (povetacicept; immunology/nephrology)
Crinetics Pharmaceuticals acquisition (completed)2026~$10.0B total equity value (~$8.8B net of acquired cash); $85/share cashCapital deployment (Palsonify; endocrinology). Closed September 1, 2026
Share repurchases2025$2.0B (annual buyback)Self-funded from operating cash flow

/ Related Companies

All Biotech
Anthropic logo

Anthropic

Biotech

Anthropic is a San Francisco AI-safety and research company, founded in 2021 by former OpenAI researchers led by siblings Dario and Daniela Amodei, that develops the Claude family of large language models. As of mid-August 2026 its lineup spans Claude Haiku 4.5, Claude Sonnet 5, the everyday-default Claude Opus 5 (which replaced Opus 4.8 on July 24), and its most capable widely released model, Claude Fable 5 (plus the invitation-only Claude Mythos 5). Claude reaches customers through the claude.ai apps, the Claude API, enterprise and government plans, and Claude Code - its agentic coding tool, which runs at a $2.5B+ run-rate with 2M+ weekly active users - alongside the Claude Cowork 'AI coworker' and the June 2026 Claude Science research workbench, built on the ~$400M acqui-hire of ex-Genentech drug-discovery startup Coefficient Bio, that positions Anthropic in AI-driven pharma R&D. Anthropic differentiates on safety and interpretability research and runs one of the industry's largest multi-vendor compute footprints spanning Amazon (Project Rainier; Amazon's committed capital reached $33B in April 2026), Google (TPUs, up to $40B plus a 3.5 GW Broadcom-built expansion), AMD, CoreWeave, and newer data-center leases with TeraWulf, Riot Platforms and Volta Infra - and was reported on August 13, 2026 to be in talks to acquire AI-infrastructure startup Decart for roughly $6B. Annualized run-rate revenue topped $65B as of late July 2026 (up from ~$47B in May and ~$9B at the end of 2025; some IPO investors are modeling $100-120B by year-end), more than 1,000 customers now spend over $1M/year, and preliminary Q2 2026 revenue exceeded $11.5B with its first profitable quarter since founding. In May 2026 it raised a $65B Series H at a $965B valuation (secondary markets have since priced it above $1T-$1.2T) and confidentially filed for an IPO in June 2026; CFO Krishna Rao is now holding early investor meetings for a possible fall listing that some backers are modeling near a $2T valuation, though Anthropic itself has not set a target. On August 27, 2026 a San Francisco federal judge (Rita Lin) ruled the Pentagon's supply-chain-risk designation of Anthropic unlawful as First Amendment retaliation and arbitrary and capricious, writing that the measures were illegal and baseless; a separate D.C. Circuit case over a second designation remains pending, and the government is expected to appeal. The same day Anthropic opened a research preview of the Model Hardware Standard (MHS), a model-agnostic specification started with HHMI Janelia that lets AI agents operate lab and manufacturing hardware such as microscopes, liquid handlers and robotic arms, with a plan to open-source the standard after the preview.

BioNTech logo

BioNTech

Biotech

BioNTech is a Mainz-based immunotherapy company that, with Pfizer, co-developed Comirnaty (BNT162b2) - the first authorized mRNA COVID-19 vaccine and one of the best-selling drugs in history. Founded in 2008 by Uğur Şahin and Özlem Türeci (with Christoph Huber) as an individualized cancer-immunotherapy company, BioNTech is now aggressively redeploying its pandemic-era war chest (~€16.6B in cash) back toward oncology, including a manufacturing-network pullback (up to 1,860 roles, sites in Germany and Singapore) as it winds down excess COVID capacity. Its centerpiece is BNT327 (pumitamig), a PD-L1xVEGF-A bispecific antibody obtained through the 2024-25 Biotheus acquisition and co-developed with Bristol Myers Squibb in a partnership worth up to ~$11.1B, now advancing across seven pivotal ROSETTA-program trials (part of 14 pivotal trials companywide) in lung, breast, gastric and other solid tumors. The pipeline also spans a second late-stage ADC platform with DualityBio (trastuzumab pamirtecan, whose breast-cancer indication reached a China BLA filing in April 2026, and the newly Phase 3 elfetabart drozuntecan in prostate cancer), a fourth late-stage asset in gotistobart (BNT316/ONC-392, with OncoC4), a next-generation anti-CTLA-4 antibody that cut the risk of death 54% versus chemotherapy in Phase 3 stage-1 lung-cancer data, individualized neoantigen mRNA cancer vaccines (autogene cevumeran/BNT122 with Genentech), CAR-T and infectious disease. COVID revenue keeps declining sharply (H1 2026 revenue just €223.7M, FY2025 was €2.87B) and BioNTech cut its FY2026 revenue guidance to €1.6-1.9B on August 4, 2026 while posting a widening H1 2026 net loss of €1.35B - yet the stock jumped ~23% on August 19, 2026 as investors refocused on the oncology pipeline, lifting market cap to ~$27.6B. Incoming CEO Guido Oelkers (succeeding founder Uğur Şahin by February 1, 2027) inherits a validated-platform leader financing a multi-year bet to become a multi-product oncology company by 2030. On August 28, 2026 BioNTech terminated the Phase 2 BNT122-01 trial of autogene cevumeran as an adjuvant monotherapy in resected colorectal cancer after an independent Data Safety Monitoring Board found a numerical survival imbalance versus watchful waiting and judged continuation unlikely to change the outcome, though it reported no new safety signal; the separate Phase 2 IMcode003 trial testing autogene cevumeran in combination with chemotherapy and checkpoint inhibition for pancreatic cancer is unaffected and continues.

CRISPR Therapeutics logo

CRISPR Therapeutics

Biotech

CRISPR Therapeutics is a gene-editing pioneer co-founded in 2013 by Nobel laureate Emmanuelle Charpentier - co-inventor of CRISPR/Cas9 - alongside Rodger Novak and Shaun Foy. Its flagship, Casgevy (exagamglogene autotemcel), is the world's first approved CRISPR/Cas9 gene-editing medicine: a one-time therapy for sickle cell disease and transfusion-dependent beta thalassemia, developed and commercialized with Vertex Pharmaceuticals (which books the sales; CRISPR keeps ~40% of the economics). Casgevy is now approved across 9+ jurisdictions, priced at ~$2.2M per treatment, and in July 2026 the FDA expanded its US label to children as young as two. Commercial uptake has been slower than hoped - constrained by cell-collection bottlenecks - but cumulative patient starts have surpassed 500. Beyond Casgevy, CRISPR is pivoting toward in vivo liver gene editing (cardiovascular programs CTX310 and CTX320), allogeneic CAR-T for autoimmune disease and cancer (zugo-cel/CTX112, now also being tested with Lilly's pirtobrutinib in aggressive B-cell lymphomas), and type 1 diabetes, where a next-generation deviceless beta-cell replacement candidate, CTX213, is progressing toward the clinic on the strength of durable C-peptide data from its predecessor CTX211. It has also expanded beyond gene editing into RNA interference through a 2025 collaboration with Sirius Therapeutics on CTX611 (SRSD107), a long-acting Factor XI siRNA for thromboembolic disorders now in Phase 2, with topline data due in H2 2026. In its Q2 2026 update (August 3, 2026) it reported $2,364.4M in cash and investments, a narrowed net loss of $91.2M, and the start of Phase 1 trials for two next-wave in vivo candidates, CTX340 (refractory hypertension) and CTX460 (alpha-1 antitrypsin deficiency, the first program from its new SyNTase editing platform), pairing a historic scientific position with a still-nascent commercial ramp.

Google DeepMind logo

Google DeepMind

Biotech

Google DeepMind is Alphabet's consolidated artificial-intelligence research lab, formed in April 2023 by merging the original DeepMind (founded in London in 2010, acquired by Google in 2014) with Google Brain. Co-founder and Nobel laureate Demis Hassabis stepped down as CEO on August 5, 2026, moving to Chair of Google DeepMind and Chief Scientist of Alphabet to focus on long-term AGI research and Isomorphic Labs; Koray Kavukcuoglu, an SVP reporting directly to Sundar Pichai, now runs day-to-day operations and the Gemini roadmap. The lab builds the Gemini family of frontier multimodal models - which powers the Gemini app (900M+ monthly active users as of mid-2026), Google Search's AI Mode and AI Overviews, Google Cloud's Vertex AI, and, via a 2026 partnership, Apple's next-generation Siri. Beyond consumer AI, DeepMind pioneered game-playing systems (AlphaGo, AlphaZero) and scientific breakthroughs including AlphaFold - whose protein-structure database has been used by 3M+ researchers and earned Hassabis and John Jumper the 2024 Nobel Prize in Chemistry - plus AlphaGenome, AlphaEvolve, Veo and Genie world models, and Gemini Robotics. Its drug-discovery spinout Isomorphic Labs is preparing first-in-human oncology trials. The August leadership change follows a stretch of talent departures (Jumper among them) and delays shipping Gemini 3.5 Pro, and coincided with Google chief scientist Jeff Dean also leaving the company after 27 years.

Moderna logo

Moderna

Biotech

Moderna is the pioneer and, with BioNTech, one of the two global leaders in messenger-RNA (mRNA) medicine. Founded in 2010 and incubated by Flagship Pioneering, it rose to prominence with Spikevax, its COVID-19 vaccine that reached full FDA approval in 2022 and drove roughly $18B of revenue at its 2022 peak. Moderna is now navigating a steep post-COVID transition: FY2025 revenue fell ~40% to $1.9B and it posted a $2.8B net loss, prompting ~$2.2B of annual cost cuts and a 10% workforce reduction. The company is rebuilding around a four-product respiratory franchise - next-gen COVID vaccine mNEXSPIKE, RSV vaccine mRESVIA, Europe's first flu+COVID combination mCOMBRIAX, and, as of its August 5, 2026 FDA approval, mFlusiva, the first mRNA-based flu vaccine - while its highest-value bet is intismeran autogene (mRNA-4157/V940), an individualized neoantigen cancer therapy partnered with Merck that has shown durable five-year benefit in melanoma and, as of August 19, 2026, met both endpoints of its pivotal Phase 3 melanoma trial, with a regulatory filing planned within months. Its first rare-disease candidate, propionic acidemia therapy mRNA-3927 (partnered with Recordati since early 2026), is in a fully enrolled registrational study with data expected in 2026. With ~$6.9B in cash, 36 development programs, and a targeted return to cash-flow breakeven in 2028, Moderna is a validated-platform leader working through significant near-term financial and execution headwinds.

Oklo logo

Oklo

Biotech

Oklo is developing fast-fission power plants under its Aurora product line while building a vertically integrated nuclear business around power sales, fuel recycling, and radioisotopes. Aurora units are designed to produce 15-75 MWe and run on fresh, recycled, or down-blended fuel, targeting customers such as data centers, industrial sites, military facilities, utilities, and remote communities. Its first plant, Aurora-INL at Idaho National Laboratory, is advancing under DOE's Reactor Pilot Program - the DOE approved its Preliminary Documented Safety Analysis on June 11, 2026, leaving the final Documented Safety Analysis as the last safety-basis step before authorization. Oklo is deepening vertical integration through the June 2026 acquisitions of Oak Ridge precision manufacturer ARMEC and sodium-systems engineering firm Creative Engineers, plus its May 2026 selection for DOE's Surplus Plutonium Utilization Program, while Atomic Alchemy builds U.S. isotope supply around the Groves test reactor in Lockhart, Texas, which reached first criticality on August 5, 2026.