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Beam Therapeutics | Company Profile | iPS Cells & Regenerative Medicine

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COMPANY PROFILE / iPS CELLS & REGENERATIVE MEDICINE

Beam Therapeutics
A base-editing company changing a single DNA letter without cutting the strand, pursuing both an ex vivo blood stem cell therapy and an in vivo liver therapy

Beam is a base-editing specialist that does not work with iPS cells. It earns a place in this field because its therapy that edits the patient's own blood stem cells and returns them (risto-cel), the same broad approach as CASGEVY, uses a method that does not cut the DNA. It is also developing a technology that replaces the conditioning drugs with an antibody (ESCAPE) and an approach that edits blood stem cells directly inside the body. The BLA for risto-cel is planned for the end of 2026 at the earliest. This profile concentrates on what the company invests in, who it works with and what it is aiming for.

Primary sources: Beam's Form 10-K for the year ended December 2025 (filed with the U.S. SEC on 24 February 2026) and its Form 10-Q for the quarter ended June 2026 (filed 4 August 2026). Last updated October 2026.

What this article covers
  1. Beam in 30 seconds, and where it stands in iPS cells and regenerative medicine
  2. The logo and how the name is written
  3. Where the company sits in iPS cells and regenerative medicine
  4. Company scale
  5. Resources committed to iPS cells and regenerative medicine
  6. Track record so far
  7. Plans ahead
  8. What the company is aiming for
  9. Partnership map for genome editing and blood stem cell therapy
  10. Technologies and products on offer
  11. The risks this company carries
  12. Glossary, references and claim-to-source audit

1. Beam in 30 seconds, and where it stands in iPS cells and regenerative medicine

LEGAL NAMEBeam Therapeutics Inc.A Delaware corporation. Began operations in January 2017
HEADQUARTERSCambridge,
Massachusetts, U.S.
238 Main Street (cover of the 10-K)
LISTINGNasdaq: BEAMA U.S.-listed company
CATEGORY IN THIS FIELDGenome editing & gene therapySpecialises in base editing
HOW THE THERAPIES WORKBase editing
(both outside and inside the body)
Changes a single DNA letter without cutting the DNA
LEAD PRODUCTSristo-cel (sickle cell disease)
BEAM-302 (AATD)
Both in Phase 1/2. BLA for risto-cel at the end of 2026 at the earliest
EMPLOYEES511At 31 December 2025, of whom 399 are in R&D
CASH AND SECURITIESAbout US$1.2 billionAt 30 June 2026
The one thing to grasp first

Beam is a base-editing company: it changes a single DNA letter without cutting the strand. It works along two main lines, neither involving iPS cells: "risto-cel", in which the patient's own blood stem cells are edited outside the body and returned, and "BEAM-302" and other programmes that edit the liver inside the body using lipid nanoparticles (LNPs) Sourced.

Risto-cel, like CASGEVY, edits autologous blood stem cells, but the editing method is different (base editing rather than a method that cuts the DNA). Beam is also developing a technology that replaces the conditioning drugs with an antibody (ESCAPE). We found no product in Beam's 10-K or 10-Q that uses iPS cells or cells from another person Not yet confirmed.

2. The logo and how the name is written

A note on names: the legal entity is Beam Therapeutics Inc. We could not confirm an official Japanese-language rendering in the material we used, so this article uses the English name throughout. "Beam" is also an ordinary English word, so in this article it always means the company when written with a capital B. Risto-cel (formerly BEAM-101) is the same product from the same company; only the name has changed. Its partners Pfizer, Eli Lilly and Bristol Myers Squibb are all separate companies.

Official site: https://beamtx.com/

3. Where the company sits in iPS cells and regenerative medicine

Three types of company involved in iPS cells and regenerative medicine All are "regenerative medicine companies", but they treat disease in completely different ways Large pharma & strategic partners Genome editing & gene therapy iPS cell & cell therapy Enters via deals; has sales network Rewrites genes directly to treat Makes and transplants cells Vertex / Novartis Bristol Myers Squibb and others CRISPR Therapeutics / Intellia Beam / Prime Medicine and others Fate / Sana Cuorips / Heartseed and others Late-stage and sales funding Editing tools, safety know-how Cell production and QC scale Thin in-house cell/gene base Swayed by partner strategy shifts Heavy cost and long-term safety Beam is the middle box: a base-editing specialist that changes one DNA letter without cutting It edits a patient's own blood stem cells outside the body, and the liver inside it using LNPs
Fig. 1 Three types of company involved in iPS cells and regenerative medicine, and where Beam sits. The classification is our own, and the example companies are drawn from those covered in this series Our calculation. Only the middle box is highlighted. Risto-cel edits autologous blood stem cells and does not use iPS cells.
Therapeutic approachWhere Beam fitsStatusCategory
Genome editing (base editing, outside the body)risto-cel (BEAM-101). The patient's own blood stem cells are edited by electroporation and returned after conditioningPhase 1/2 (BEACON). BLA at the end of 2026 at the earliestSourced
Genome editing (base editing, inside the body)BEAM-302 (AATD), BEAM-301 (GSDIa), BEAM-304 (PKU). Delivered to the liver in LNPsBEAM-302 and BEAM-301 are in Phase 1/2; the IND for BEAM-304 was accepted (June 2026)Sourced
Editing blood stem cells inside the bodyAn LNP that targets blood stem cells is in development. Prioritised as the "next wave" in sickle cell diseaseLead optimisationSourced
Replacing conditioning with an antibody (ESCAPE)BEAM-103 (anti-CD117 antibody). Usable with both ex vivo and in vivo approachesPhase 1 (healthy adults)Sourced
iPS-cell-derived cellsNo mention of "iPS" in the 10-K or 10-QCould not be confirmedNot yet confirmed
Mesenchymal stem cells and tissue engineeringNot describedCould not be confirmedNot yet confirmed

Our explainers on genome editing, off-target effects and safety assessment, genome-editing therapies for blood disorders, lipid nanoparticles and mRNA give the background to each row of the table.

4. Company scale

Figures are in U.S. dollars and the fiscal year ends in December. Beam has no product sales, and its revenue comes from collaboration agreements Sourced.

Measure (US$ millions)20242025First half of 2026
Collaboration revenue63.5139.732.2
Research and development expense367.6409.6199.6
General and administrative expense111.5113.866.4
Operating loss415.6383.7233.8
Gain on sale of Orbital stake-255.1-
Net loss376.780.0217.0

The 2025 net loss was small, at US$80.0 million, because Bristol Myers Squibb's acquisition of Orbital produced a US$255.1 million gain on the sale of Beam's shares. The operating loss of 383.7 was about the same as the year before Sourced. The 10-K attributes the rise in 2025 collaboration revenue mainly to US$109.1 million recognised on completion of the Pfizer collaboration.

Beam had 511 employees at 31 December 2025 (399 in R&D, 112 in administration, and 118 with an M.D. or Ph.D.) Sourced.

A word of caution

The figures above are for the whole company. They do not measure the size of any iPS cell or regenerative medicine business. The first-half 2026 operating loss of 233.8 is the difference between the 10-Q's collaboration revenue of 32.2 and operating expenses of 266.0, calculated by us Our calculation. Cash is about US$1.2 billion, but the company says it covers "at least the next 12 months" of expenses, which is a shorter period than CRISPR Therapeutics' (more than 24 months) Sourced.

5. Resources committed to iPS cells and regenerative medicine

There are no figures for iPS cells and regenerative medicine alone. We show the resources relevant to genome editing and blood stem cell therapy, along four lines.

ResourceDetailsCategory
People (company-wide)511 (31 December 2025): 399 in R&D, 112 in administration, 118 with an M.D. or Ph.D.Sourced
People (iPS cells and regenerative medicine)No dedicated team is describedCould not be confirmed
SitesA research building of about 130,258 sq ft in Cambridge (leased to 2034). A cGMP manufacturing building of about 100,000 sq ft in Research Triangle Park, North Carolina (leased to 2037), designed to make both ex vivo edited cell therapies and LNP products for use inside the bodySourced
Money (R&D expense)US$409.6 million company-wide (2025); US$199.6 million in the first half of 2026Sourced
Money (financing)Cash of about US$1.2 billion (30 June 2026). Net proceeds of US$470.5 million from the March 2025 equity raise. A loan agreement with Sixth Street in February 2026 (US$100.0 million initially, with up to US$300 million more depending on the filing, approval and sales of risto-cel)Sourced
Investments and acquisitionsHeld 75 million shares of Orbital Therapeutics and received US$255.1 million on its acquisition by BMS in December 2025 (with up to about US$26.3 million more from escrow)Sourced
In-licensingAn exclusive licence to base-editing patents from Harvard University (2017). The first success-fee measurement gave US$15.0 million (paid in shares). An option to obtain exclusive licences to patents from Editas (Cas9 and Cas12a). A cross-licence with Prime MedicineSourced
Out-licensing and collaborationPfizer (from December 2021; opted in to a liver candidate in December 2025), Apellis (from June 2021; opted in to the FcRN programme, fee of US$3.8 million), Verve / Lilly (in October 2023 Lilly acquired the rights under the Verve agreement, with US$200.0 million upfront to Beam and up to US$350.0 million), and OrbitalSourced
Universities, research institutes and public fundingHarvard University (patents) and the Broad Institute (the party to whom success fees are owed). No public funding or grants could be confirmed in the material we usedSourced / could not be confirmed

6. Track record so far

WhenWhat happenedWhat it means
January and June 2017Begins operations. In June, exclusive licence agreement with Harvard University for base-editing patentsSecured the rights to the editing tool
April 2019Exclusive licence agreement with Verve for base editing against three cardiovascular targetsOut-licensed an area it does not pursue itself
June and December 2021Research collaboration with Apellis on complement-related targets in June. Four-year research collaboration with Pfizer in December (in vivo base editing, three targets)Partnerships with large companies began
August 2022Phase 1/2 trial of risto-cel (BEACON) begins (ClinicalTrials.gov start date: 30 August 2022)An ex vivo edited cell therapy entered the clinic
October 2023Lilly acquires the rights under the Verve agreement. US$200.0 million to Beam, up to US$350.0 millionTurned contractual rights into cash
June 2024Phase 1/2 trial of BEAM-302 begins (ClinicalTrials.gov start date: 19 June 2024)In vivo editing entered the clinic
March 2025Equity raise with net proceeds of US$470.5 millionThickened its funding
December 2025Presents data on 31 people treated with risto-cel at ASH. Pfizer opts in to a liver candidate. BMS acquires Orbital and Beam receives US$255.1 million. A long-term follow-up study of BEACON also beginsSeveral milestones came together
February 2026Loan agreement with Sixth Street (US$100.0 million initially)An additional borrowing facility tied to the filing and approval of risto-cel
March 2026Presents updated data for BEAM-302. Based on FDA advice, aims for an accelerated approval path with a 12-month AAT measure at 60 mg as the primary endpointThe design leading to an application for approval took shape
May-June 2026Biogen acquires Apellis in May. The IND for BEAM-304 (PKU) is accepted by the FDA in JuneA partner was reorganised and a new product started
July 2026First patient dosed in the main cohort of BEAM-302 (an expansion of about 50 people)The trial supporting an application for approval began

7. Plans ahead

RISTO-CEL BLAEnd of 2026
at the earliest
Updated BEACON data also by the end of 2026
BEAM-302Main cohort of
about 50 people
First patient dosed in July 2026
BEAM-301Initial data to be
reported in 2026
Phase 1/2 in GSDIa
BEAM-304Preparing to start
Phase 1/2
IND accepted in June 2026
BEAM-103 (ESCAPE)Phase 1
(healthy adults)
Dosing due to finish in the first half of 2026
EDITING BLOOD STEM CELLS INSIDE THE BODYLead optimisationThe next wave in sickle cell disease
ADDITIONAL BORROWINGUp to US$300 millionTied to acceptance of the risto-cel BLA, approval and sales
iPS CELL-RELATEDNo plan describedNothing found in the 10-K or 10-Q

Of the items in the table, the company gives timing for the risto-cel BLA and the main cohort of BEAM-302 Sourced. All are plans, not guarantees of approval or launch Not yet confirmed.

What could not be confirmed

None of the following appears in the material we used Not yet confirmed.

The timing of approval and the price of risto-cel / the timing of an application for approval of BEAM-302 / the Phase 1 results of BEAM-103 / any plan to enter iPS cells and regenerative medicine. Timings and prices in press reports cannot be verified against primary sources, so this article does not use them.

8. What the company is aiming for

This section includes things the company does not say directly. We set them out as inferences within what the primary sources support, with the evidence for each.

What the evidence suggestsPrimary information it rests onCategory
In sickle cell disease, it intends to move from editing outside the body to editing inside itThe 10-K states that, as the next wave in sickle cell disease, it is prioritising in vivo delivery to blood stem cellsSourced
The reason is to avoid the burdens of conditioning and cell collectionThe 10-K says ex vivo editing requires conditioning such as busulfan, and ESCAPE aims at "non-genotoxic" conditioning. The company does not state its reason for the shift, howeverInference
The first commercial product will be risto-celThe BLA is planned for the end of 2026 at the earliest. Sixth Street's additional borrowing facility is tied to acceptance of the risto-cel BLA, approval and salesInference
It values owning its own manufacturingIt owns a cGMP manufacturing building of about 100,000 sq ft and says early clinical trials will be supplied mainly in houseSourced
Areas it does not pursue itself it out-licenses to large companies and turns into cashAgreements with Pfizer, Lilly and Orbital (BMS) brought in revenue and US$255.1 million in 2025Inference
One piece of evidence in more detail

For readers of this series, we read the most important item as ESCAPE, which replaces the conditioning drugs with an antibody Inference. Ex vivo editing therapies (CASGEVY included) need conditioning that destroys bone marrow cells with drugs to make room for the edited stem cells. Beam's 10-K describes ESCAPE as a technology that combines "antibody-based conditioning with stem cells edited at several genes", and says it could enable a "non-genotoxic" treatment strategy Sourced. The stage reached is the BEAM-103 Phase 1 in healthy adults, and no effect in patients has yet been shown Not yet confirmed. The 10-K also notes that the contractual definition of Beam's field includes "conditioning for cell transplantation", which shows that replacing conditioning is a challenge for cell transplantation as a whole.

9. Partnership map for genome editing and blood stem cell therapy

Genome-editing and blood stem cell therapy partnerships centred on Beam Only relationships in the FY2025 10-K and June 2026 10-Q; press-only links are excluded Beam Genome editing Pfizer Liver candidate opt-in (Dec 2025) Eli Lilly (acquired Verve) Took Verve deal rights (2023) Orbital (acquired by BMS) Stake sold for US$255.1 million Apellis (acquired by Biogen) Opted in to FcRN programme Prime Medicine Technology cross-licence Harvard University Exclusive base editing patents Co-development and licensing Equity stake and sale
Fig. 2 Beam's partnerships in genome editing and blood stem cell therapy. Sources: Form 10-K for FY2025 and Form 10-Q for June 2026. Only relationships described in both filings are shown. Sixth Street Lending Partners, with which Beam has a loan agreement, is not a therapeutic partner and is left out of the figure.

The thing to notice in this map is that almost all of Beam's partners have been acquired, or are now acquirers. Verve was acquired by Lilly, Orbital by BMS and Apellis by Biogen Sourced. The counterparties in Beam's collaborations change each time the large drug companies reorganise.

10. Technologies and products on offer

ProductHow it worksTargetStatus
risto-cel
(BEAM-101)
The patient's own blood stem cells are base-edited by electroporation (an edit that raises fetal hemoglobin). Conditioning with busulfanSevere sickle cell diseasePhase 1/2 (BEACON). FDA orphan and RMAT designations. BLA at the end of 2026 at the earliest
BEAM-302Base editor delivered in a liver-targeted LNP to correct the E342K mutation in the SERPINA1 gene. Single doseAlpha-1 antitrypsin deficiency (AATD)Phase 1/2. Agreed an accelerated approval path with the FDA
BEAM-301Base editor delivered in a liver-targeted LNP to correct the R83C mutation in the G6PC geneGlycogen storage disease type Ia (GSDIa)Phase 1/2
BEAM-304Base editor delivered in a liver-targeted LNPPhenylketonuria (PKU)IND accepted (June 2026)
BEAM-103 (ESCAPE)An anti-CD117 antibody that replaces the conditioning drugsHematopoietic stem cell transplantation in generalPhase 1 (healthy adults)

Trial results (figures from company statements as recorded in the 10-K and 10-Q; not peer-reviewed papers)

TrialPopulation and designResultTime point and caveats
BEACON (risto-cel)31 people with severe sickle cell disease (interim data at ASH 2025). Single-arm, open-labelMean HbF above 60% and HbS below 40%. No severe vaso-occlusive crises reported by the treating clinicians after dosing. Median neutrophil engraftment at 17.5 daysAs of 6 August 2025, follow-up 0.3 to 20.4 months. No comparison group. One person died of respiratory failure 4 months after dosing (the investigator judged it likely related to the conditioning drug busulfan)
BEAM-302 Phase 1/2 (March 2026 update)AATD. 29 people dosed (24 in Part A, 5 in Part B), followed up to 18 months as of 10 February 2026. Safety assessed in 26 people given a single doseIn the 60 mg group of Part A, mean total AAT at steady state was 16.1 µM, mutant Z-AAT fell by a mean of 84%, and the share of circulating AAT that was newly made normal M-AAT was 94%No comparison group. In the repeat-dose group (3 people), one person had a Grade 4 ALT elevation after the second dose (no symptoms, no treatment needed)
A materials engineer's view: two lines that rely on different "materials", electroporation and LNPs

Beam has two routes for delivery, which differ: electroporation (electrical pulses that get a base editor into cells) outside the body and LNPs inside it Sourced. For electroporation, the dedicated equipment, buffers and single-use containers that get the editor in efficiently without damaging the cells bear on quality, while for LNPs the lipid composition and the uniformity of particle size are decisive Inference.

The 10-K says it has put mRNA manufacturing and cell processing in place as in-house capabilities, and that its cGMP building of about 100,000 sq ft is designed for both ex vivo edited cell therapies and non-viral products for use inside the body Sourced. ESCAPE's antibody (anti-CD117) is also an attempt to replace the small-molecule conditioning drug with an antibody medicine, which changes the kind of materials used at a fundamental level.

11. The risks this company carries

RiskDetailsCategory
No product yetNo product sales and no approved product. The risto-cel BLA is the first milestoneSourced
Safety linked to conditioningIn BEACON, one death was judged likely to be related to the conditioning drug busulfan. Ex vivo editing therapies presuppose conditioningSourced
Safety of in vivo editingIn the repeat-dose group of BEAM-302, one person had a Grade 4 ALT elevation (no symptoms). No serious adverse events were reported in the 26 people given a single doseSourced
CompetitionIn sickle cell disease, the 10-K names many competitors, including CRISPR Therapeutics, Vertex, Genetix (formerly bluebird bio) and Novartis. Some already have approved therapiesSourced
PatentsThe base-editing patents are exclusively licensed from Harvard University, which can license third parties in certain circumstances. Cas9 and Cas12a depend on an option from EditasSourced
Funding and borrowingCash is about US$1.2 billion, but the period the company says it covers is 12 months. The Sixth Street loan carries a liquidity covenant (a minimum of US$40 million, rising with additional borrowing)Sourced
Partners' reorganisationVerve (Lilly), Orbital (BMS) and Apellis (Biogen) have been acquired. Whether the contracts continue depends on the acquirers' policiesSourced / Inference

12. Glossary

Base editing
An editing method that changes a single base (A, C, G or T) into another without cutting both strands of the DNA.
Electroporation
A method that opens temporary pores in the cell membrane with electrical pulses so that an editing agent can get in.
HbF (fetal hemoglobin)
An oxygen-carrying protein made before birth. Higher levels are thought to prevent red blood cells from sickling.
Conditioning
Using drugs before treatment to reduce the cells in the bone marrow and make room for the edited cells. It places a heavy burden on the body.
ESCAPE
A technology Beam is developing that uses an antibody for conditioning. It aims at conditioning that does not damage DNA.
AATD
Alpha-1 antitrypsin deficiency. An inherited disease in which a protein made in the liver is abnormal, causing damage to the lungs and liver.
LNP (lipid nanoparticle)
A tiny particle made of lipids that wraps nucleic acids and delivers them into cells.
BLA / RMAT
A BLA is the U.S. application for approval of a biologic. RMAT is an FDA designation that speeds up the development of regenerative medicine.
Accelerated approval
A U.S. pathway that approves a drug first on the strength of a surrogate marker (biomarker). A confirmatory trial is required afterwards.
Opt-in
When a partner exercises its right to take part in development.
Fig. 3 Concept diagram of Beam's base editing (AI-generated concept; it does not depict actual equipment or products).

13. References

  1. Beam Therapeutics Form 10-K for the fiscal year ended December 31, 2025 (filed with the U.S. SEC on 24 February 2026). Base-editing business, clinical work on risto-cel (BEAM-101) and BEAM-302 and others, collaboration agreements, patent licences, sites, headcount and financials. https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm
  2. Beam Therapeutics Form 10-Q for the quarterly period ended June 30, 2026 (filed with the U.S. SEC on 4 August 2026). BEAM-302 data, acceptance of the BEAM-304 IND, the current status of collaborations, the Sixth Street loan, and first-half 2026 financials. https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm
  3. ClinicalTrials.gov NCT05456880, BEACON: a study evaluating BEAM-101 in severe sickle cell disease (sponsor: Beam Therapeutics). Start date 30 August 2022, Phase 1/2. https://clinicaltrials.gov/study/NCT05456880
  4. ClinicalTrials.gov NCT07373639, a long-term follow-up study of participants who received BEAM-101. Start date 15 December 2025, estimated enrolment 50. https://clinicaltrials.gov/study/NCT07373639
  5. ClinicalTrials.gov NCT06389877, a study evaluating BEAM-302 in alpha-1 antitrypsin deficiency. Start date 19 June 2024, Phase 1/2, estimated enrolment 106. https://clinicaltrials.gov/study/NCT06389877
  6. Beam Therapeutics Official website (used to check the company overview and the logo). https://beamtx.com/

14. Claim-to-source audit

Claim in the articleCategorySource
Legal name, Cambridge headquarters, Nasdaq: BEAM, start of operations in January 2017SourcedReferences 1 and 6 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm https://beamtx.com/
Collaboration revenue, R&D expense, G&A expense, operating loss and net loss (2024 and 2025), and US$109.1 million of revenue on completion of the Pfizer collaborationSourcedReference 1 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm
First-half 2026 financials, cash of about US$1.2 billion, and the statement that it covers 12 months of expensesSourcedReference 2 https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm
US$255.1 million gain on the sale of the Orbital stake (BMS acquisition, December 2025)SourcedReference 1 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm
511 employees (31 December 2025), 399 in R&D, 118 with an M.D. or Ph.D.SourcedReference 1 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm
Cambridge building of about 130,258 sq ft and Research Triangle Park cGMP building of about 100,000 sq ftSourcedReference 1 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm
The March 2025 equity raise (net proceeds of US$470.5 million)SourcedReference 1 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm
The Sixth Street loan agreement (US$100.0 million initially, up to US$300 million more, interest rate, covenants)SourcedReferences 1 and 2 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm
How risto-cel works, the design of the BEACON trial, a BLA at the end of 2026 at the earliest, RMAT and orphan designationsSourcedReferences 1, 2 and 3 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm https://clinicaltrials.gov/study/NCT05456880
BEACON interim data (31 people, HbF above 60%, HbS below 40%, engraftment at 17.5 days, one death)SourcedReferences 1 and 2 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm
How BEAM-302 works, the start of the trial, the March 2026 data (84% reduction in Z-AAT and others), the accelerated approval path, and the start of the main cohort in July 2026SourcedReferences 2 and 5 https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm https://clinicaltrials.gov/study/NCT06389877
The BEACON long-term follow-up study (NCT07373639, started 15 December 2025)SourcedReference 4 https://clinicaltrials.gov/study/NCT07373639
BEAM-301, BEAM-304 (IND accepted June 2026) and BEAM-103 (ESCAPE, Phase 1)SourcedReferences 1 and 2 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm
Prioritising in vivo delivery to blood stem cells as the next wave in sickle cell diseaseSourcedReference 1 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm
Exclusive licence from Harvard University (2017), US$15.0 million success fee, the Editas option, and the cross-licence with Prime MedicineSourcedReference 1 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm
Pfizer's December 2025 opt-in and Apellis's opt-in to FcRN, including the US$3.8 million payment and Biogen acquisitionSourcedReferences 1 and 2 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm
Verve / Lilly (October 2023, US$200.0 million and up to US$350.0 million, a cumulative US$50.0 million by June 2026)SourcedReferences 1 and 2 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm
Orbital / BMS (a non-exclusive cross-licence, acquisition completed in December 2025)SourcedReference 1 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm
First-half 2026 operating loss of 233.8 (the difference between 32.2 and 266.0)Our calculationReference 2 https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm Subtraction performed in this article
The three types of company (large pharma and strategic partners / genome editing and gene therapy / iPS cell and cell therapy)Our calculationThis article's own classification. Example companies are drawn from those covered in this series
Use of iPS cells, mesenchymal stem cells, and any allogeneic cell therapy of its ownNot yet confirmedReferences 1 and 2 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm Not stated in the 10-K or 10-Q. This article does not conclude that they do not exist either
The timing of approval and price of risto-cel, the timing of a BEAM-302 application, and the results of BEAM-103Not yet confirmedReference 2 https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm Not stated in the 10-Q. Figures from press reports are not used
Public funding and grantsNot yet confirmedReference 1 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm Not stated in the 10-K
The reading that the reason for moving from ex vivo to in vivo is to avoid the burdens of conditioning and collectionInferenceReference 1 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm The company does not state its reason. This article's interpretation
The reading that the first commercial product will be risto-celInferenceReferences 1 and 2 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm This article's interpretation
The reading that it out-licenses areas it does not pursue itself and turns them into cashInferenceReference 1 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm This article's interpretation
The reading that ESCAPE could change the problem of conditioningInferenceReference 1 https://www.sec.gov/Archives/edgar/data/1745999/000119312526065194/beam-20251231.htm This article's interpretation. No effect in patients has been confirmed
The reading that partners' reorganisation affects whether contracts continueInferenceReference 2 https://www.sec.gov/Archives/edgar/data/1745999/000119312526331541/beam-20260630.htm This article's interpretation
The reading that the materials for electroporation and LNPs determine qualityInferenceThis article's interpretation (from a materials and process viewpoint)

Last updated 1 October 2026 / Troy Technical
Every figure in this article comes from the statutory filings Beam has made with the U.S. Securities and Exchange Commission (SEC) and from ClinicalTrials.gov. The clinical trial data are company statements as confirmed in the 10-K and 10-Q, not peer-reviewed papers. No research-firm estimates or press-based figures have been used. Passages marked "Inference" are this article's interpretation of the primary sources, not statements made by the company. This article summarises a company's activities; it does not recommend any treatment and is not medical advice.

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