Gene Editing Weekly — September 26–October 2, 2026: a base-editing trade-secrets suit, Germany's first Casgevy treatment, and a second route to fetal hemoglobin
Beam sues a Chinese base-editing rival and its U.S. partner over trade secrets, a Berlin hospital gives Germany its first CRISPR treatment, and a new fetal-hemoglobin pathway opens a second route against sickle cell disease.
The coverage window for this issue is September 26 – October 2, 2026, Eastern Time. The seven days hold one legal fight over base editing, the first CRISPR-based treatment given to a patient in Germany, three peer-reviewed papers, and no new patient data from a gene-editing therapy. The legal fight sits closest to a clinic, so this issue starts there.
| Track | Primary change | Evidence boundary | Next milestone |
|---|---|---|---|
| Legal | Beam Therapeutics sued YolTech Therapeutics and Serapha Bio on September 25, alleging a former Beam scientist carried base-editing trade secrets to the Chinese company. 1 | A complaint filed in federal court. The allegations are untested, and Serapha denies them. 1 | The defendants' responses, and whether the case reaches SERP-01's Phase 2/3 start. |
| Clinical rollout | Charité in Berlin gave Germany's first CRISPR treatment to a 19-year-old with beta-thalassemia. 2 | One patient treated outside a trial, reported by the hospital, with follow-up ongoing. 2 | Whether other German centres take the therapy up, and how payers price it. |
| Company | Cellectis put preclinical data and dates on the two in vivo lipid-nanoparticle programs it had announced in September. 3 | Preclinical data presented by management at an investor forum; neither program has been dosed in a person. 3 | A China investigator-initiated trial for HEAL-101, planned for the first quarter of 2027. |
| Research | A genome-wide study identified a second pathway, BACH2–NRF2, that switches on fetal hemoglobin independently of BCL11A. 4 | Human genetics plus cell and pharmacological work; the pathway has not been targeted in a person. 4 | Whether a second target joins BCL11A in clinical development. |
| Delivery | A Toronto team reported a lipid nanoparticle, LC-1, that keeps its potency as editor cargo grows and reaches mouse liver, brain and lung. 5 | Mouse data; no non-human primate or human results. 5 | Whether the lipid holds up in larger animals and on repeat dosing. |
| Research | Prime editing corrected a GATA2 deficiency variant in patient blood stem cells. 6 | Ex vivo preclinical work; no patient has been treated. 6 | A sponsor, a manufacturing route, and an IND. |
| Governance | Trends in Genetics published a commentary arguing for therapeutic germline editing and new regulatory pathways. 7 | An opinion piece by one author, not a decision by a regulator or a professional body. 7 | Whether a regulator or bioethics body takes the argument up. |
| FDA ledger | CBER's biologics ledger carried no genome-editing action this week. 8 | A weekly posting ledger, which shows what the agency chose to post. 8 | The October 30 deadline for the Expedited IND pilot. |
Corporate and legal
A base-editing lawsuit lands on the alpha-1 antitrypsin race
Beam Therapeutics filed a complaint on September 25 in the U.S. District Court for the District of Massachusetts against YolTech Therapeutics, a Shanghai base-editing company, and Serapha Bio, its U.S. partner. 19 Beam alleges that a former employee, Zi Jun "Emma" Wang, "secretly founded a Chinese biotechnology company called YolTech while still working at Beam," and that five months after leaving she filed patent applications on YolTech's behalf based on Beam's confidential and trade-secret information. 1
Beam says Wang worked as a scientist there from 2021 to 2022 and pulled records in a pattern that departed from her ordinary work: dozens of files she had never opened, across technical areas beyond her own, at night and on weekends. 1 The complaint also names YolTech's chief executive, Yuxuan "Jensen" Wu, as a defendant. 10 Beam is asking the court to stop the use of its trade secrets and to recover the value it says the two companies took from its investments. 1
Both defendants say they will fight. Serapha's chief corporate affairs officer, Shiva Fritsche, wrote that Serapha "categorically refutes Beam's claims and will vigorously defend the company," and YolTech plans to contest the allegations. 1112
The dispute centers on one licensed program. YolTech licensed an alpha-1 antitrypsin deficiency (AATD) candidate to Serapha for an undisclosed upfront payment, and Serapha calls it SERP-01. 1 Serapha went public through a reverse merger in June with $230 million from investors including RA Capital Management and RTW Investments, and in September it named a leadership team, led by chief executive Kenneth Mills and chief medical officer Weston Miller. 1 SERP-01 targets the same mutation as Beam's BEAM-302 and changes a nucleotide at the same position in the DNA. 1
AATD is an inherited disorder caused by variants in the SERPINA1 gene. 13 The Z allele makes very little of the protective protein, and the abnormal protein can also build up and damage the liver; people with two copies of it, called PiZZ, face high risk of both emphysema and AATD-associated liver disease. 13 Wave Life Sciences estimates about 200,000 people in the United States and Europe are PiZZ. 14
Several programs now aim at the same defect.
| Company / program | Editing modality | Clinical stage | Next milestone |
|---|---|---|---|
| Beam Therapeutics — BEAM-302 | Base editing | Phase 1/2; enrolling a new cohort | Beam has said it will pursue accelerated U.S. approval if the new cohort's data support it. 1 |
| Serapha Bio — SERP-01 (licensed from YolTech) | Base editing | IND cleared | A Phase 2/3 trial planned for late 2026 or early 2027. 1 |
| Prime Medicine — PM647 | Prime editing | IND cleared September 24, 2026 | First-in-human dosing, with initial data guided to 2027. 15 |
| Wave Life Sciences — WVE-006 | RNA editing; corrects the messenger RNA and leaves the DNA unchanged | Phase 1b/2a | A single 2-year registrational trial, after FDA feedback on the pathway. 14 |
Three of those programs correct the same base in the same gene, so the comparisons that will matter are dose, durability and immune profile. The fourth edits RNA and sits one step removed from the DNA editors this briefing otherwise tracks.
Clinical and regulatory
Germany treats its first patient with Casgevy
Charité – Universitätsmedizin Berlin said on October 1 that a 19-year-old patient with severe beta-thalassemia had become the first person in Germany treated with Casgevy. 2 The hospital collected the patient's blood stem cells, edited them, and reinfused roughly 900 million of them in May 2026, after chemotherapy to clear space in the bone marrow. 2 The patient no longer needs regular blood transfusions, his hemoglobin is back in the normal range, and his immune system has recovered, according to the hospital. 16 His health insurer agreed to cover the treatment after a case-by-case review. 2
The European Union has authorized Casgevy since 2024 for people aged 12 and older with transfusion-dependent beta-thalassemia or severe sickle cell disease. 16 A stem cell transplant can cure beta-thalassemia, but the hospital says the procedure is generally limited to patients around 14 years old or younger. 16 Treated patients are monitored for 15 years. 2
What the case shows is that the ex vivo route — apheresis, laboratory editing, chemotherapy conditioning and reinfusion — can be delivered inside a national health system and paid for. The efficacy evidence comes from the trials behind the approval.
The week's CBER ledger
The FDA's "What's New for Biologics" list carried five postings dated inside the window: approval letters for BIVIGAM and ASCENIV dated September 29, two standard operating procedures posted September 29, and an approval letter for ROTARIX dated September 25. 8 None concerns a genome-editing product, so the ledger records no editing approval, complete response letter, advisory committee action, IND clearance or BLA acceptance for the week. 8 The vaccines advisory committee met on October 1 to recommend strains for the 2027 Southern Hemisphere influenza season, which sits outside this field. 8
Research
A second way to switch on fetal hemoglobin
Nature published the work on September 30. 4 Vijay Sankaran of Dana-Farber/Boston Children's Cancer and Blood Disorders Center and colleagues ran a genome-wide association study across European, African and Asian ancestries and identified a pathway built on two proteins, BACH2 and NRF2. 17 BACH2 normally acts as a brake on fetal hemoglobin production, and reducing it lets NRF2 switch the fetal hemoglobin genes on. 17
The team also showed that a drug that inhibits BACH2 raises fetal hemoglobin in human red blood cell precursors. 17 Sankaran's lab identified BCL11A, the gene that Casgevy and the other sickle cell editing therapies switch off to raise fetal hemoglobin. 17 Because BACH2–NRF2 and BCL11A act through independent pathways, the authors want to test whether hitting both gives more benefit than either alone. 17
This is human genetics backed by cell and pharmacological experiments. The paper's own framing is that the pathway may support future gene-editing or drug treatments, and the team plans further work on whether it can be targeted safely. 17
A lipid nanoparticle built for bulky cargo
Nature Biotechnology published this work on September 28. 5 Screening libraries of ionizable lipids is normally done with short reporter transcripts, such as a 1.7-kilobase luciferase messenger RNA, that stand in poorly for the 5- to 6-kilobase messages encoding Cas9 or a base editor. 18 Bowen Li's group at the University of Toronto made 384 ionizable lipids and compared delivery of the short reporter with a 5.7-kilobase base-editor construct. 18
The screen settled on a lipid called LC-1, which held its potency as the cargo grew. 18 Two doses carried Cas9 messenger RNA and guide RNA to 79% of mouse hepatocytes after intravenous dosing, 48% of neurons after injection into the spinal fluid, and 27% of lung epithelial cells after delivery into the airway, up to fourfold above the comparator lipids ALC-0315 and LP-01. 18
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A single intravenous dose of an adenine base editor aimed at Pcsk9 produced 79.5% A-to-G editing in mouse liver and lowered plasma PCSK9 by 85.7% over 60 days. 18 LC-1 also edited human PCSK9 in liver-humanized mice, the R553X allele of CFTR in a cystic fibrosis mouse model, and Ube3a-ATS in mouse brain. 18 The authors attribute the gain to LC-1 binding RNA more tightly and releasing about twice as much messenger RNA into the cytosol. 18 Every number above comes from mice.
Prime editing in blood stem cells for GATA2 deficiency
Science Translational Medicine published the work on September 30. 6 GATA2 deficiency is a severe immunodeficiency caused by variants in one copy of the GATA2 gene, and transplanting edited blood stem cells is the current curative route. 6
A team at Aarhus University used prime editing to correct a GATA2 variant in CD34+ stem and progenitor cells taken from patients, reaching up to 70% editing efficiency and raising the share of functional GATA2 alleles from 50% to 77%. 6 The edited cells engrafted better than untreated cells, and the authors found limited on-target genotoxicity and no off-target editing at the 20 sites most likely to be hit. 6 A short prestimulation step kept the cells' stem-like properties, reduced p53 activation and improved multilineage engraftment. 6 The group also reported a method for inserting complementary DNA at the same site, which it calls PASSIGE. 6
The result is preclinical and ex vivo. No patient has received the treatment, and a programme would still need a sponsor, a manufacturing route and an IND.
Where the large editors stand
Nature's technology desk ran a survey of the field's editor toolkit on September 28. 19 The article lays out the constraints: CRISPR–Cas9 must break DNA before it edits, and its efficiency falls as the inserted segment grows, so it is usually limited to stretches under about 2,000 base pairs; base editing corrects single DNA letters but must be built for each variant, and the article's example is ABCA4 in Stargardt's disease, where hundreds of distinct editors would be needed to cover the known changes; prime editing avoids cutting both DNA strands but carries only a few hundred bases. 19
The candidate answers use enzymes that stitch in larger DNA. PASTE combines prime editing with an integrase called Bxb1 to install landing pads and insert the payload, and in cynomolgus monkeys about half the liver cells carried the corrected version of a haemophilia B gene after one treatment. 19 evoCAST, a CRISPR-associated transposase, can insert up to 15,000 bases but needs six or seven proteins. 19 The limit the article names is delivery: getting those components into cells at once is what keeps the larger editors experimental. 19
Company and pipeline
Cellectis puts numbers on its lipid-disorder pivot
Cellectis used the Stifel Virtual Cardiometabolic Forum on September 30 to put data and dates on the two in vivo programs behind its September 14 strategic turn, which this briefing covered on September 18. 3
HEAL-101 targets APOC3 for severe hypertriglyceridemia. It is a base editor delivered by lipid nanoparticle that converts a cytosine to a thymine in exon 4 of the gene, creating a stop codon that shuts off the protein. 3 In humanized mice the edit cut APOC3 expression by 70%, and in transgenic mice carrying ten copies of the human gene triglycerides fell 76%, with roughly 80% on-target editing and no off-target edits or chromosomal abnormalities detected. 3 Cellectis plans a China investigator-initiated trial for the first quarter of 2027 and data in the second half of the year, with an IND and a European filing running alongside for a later global study. 3
HEAL-201 targets PCSK9 for severe hypercholesterolemia. It uses two TALE-based epigenetic modulators to methylate and acetylate the chromatin around the gene and switch it off, leaving the DNA sequence untouched. 3 The company said a full transcriptome read showed PCSK9 was the only gene meaningfully downregulated. 3 Its China study is planned for the second half of 2027 with initial data in the first half of 2028, about four months behind HEAL-101, and the company projects cash into the second half of 2028. 3 Cellectis puts the addressable population for each program at one million to two million people in the United States and Europe, and cites current triglyceride therapy costing about $50,000 to $70,000 a year. 3
Both programs are preclinical, and the numbers come from the company's own presentation. The clinical case will rest on the China studies and whatever follows them.
Adjacent to editing, but in the same disease
Wave Life Sciences said on October 1 that FDA feedback supports a registrational pathway for WVE-006 in AATD. 14 Wave plans a single 2-year registrational trial designed to support full approval, with a 1-year interim analysis that could support accelerated approval on AATD biomarkers, and the agency agreed that the company can measure wild-type and mutant AAT protein with its existing mass-spectrometry assay. 13 WVE-006 is an RNA editor given by subcutaneous injection: it corrects the mutation in the messenger RNA and leaves the DNA unchanged. 13 In the ongoing Phase 1b/2a trial, the 200 mg biweekly cohort reached total AAT of 11.9 µM with mutant Z-AAT cut about 71%, and the 400 mg monthly cohort reached 13.6 µM with Z-AAT down about 67.7%. 13 Data from a 600 mg monthly cohort are due in the fourth quarter. 14
RNA editing is a different modality from the DNA editors this briefing follows, and Wave is clear about the distinction when it argues that WVE-006 avoids the irreversible bystander edits and insertions-and-deletions that DNA base editors can produce. 13 It matters here because it competes for the same patients as the base editors in the table above.
Separately, uniQure reported additional 36- and 48-month Phase I/II results for ifezuntirgene inilparvovec, its AAV-delivered gene-addition therapy for Huntington's disease. 20 The company keeps pursuing accelerated approval for the therapy. 21
Governance
A call to edit the germline
Trends in Genetics published "No bird soars in a calm: therapeutic germline gene editing" online on September 30, a Science & Society commentary by Cathy Tie of Origin Genomics. 7 Her argument, as stated in the abstract, is that health systems spend trillions of dollars treating inherited disease, that correcting the disease mutation at the embryo stage could move medicine from treatment to prevention, and that advances in precision editing and rising public support mean the field is ready for new clinical and regulatory pathways. 7
The piece carries one author's argument. A framework for heritable editing would still need a regulator or an international professional body to adopt it.
What to watch
- The lawsuit's first procedural steps. YolTech has said it will contest the claims and Serapha says it will defend the case, so the next filings will show whether either side seeks an early injunction and whether the dispute disturbs SERP-01's plan to begin Phase 2/3 late this year or in early 2027. 112
- The AATD readout queue. Beam's new cohort, Prime Medicine's first patient for PM647, and Wave's 600 mg monthly cohort are the near-term data points in a disease with three DNA editors and one RNA editor. 1415
- Cellectis's China study. HEAL-101 is scheduled to enter an investigator-initiated trial in the first quarter of 2027, which would give the first read on the company's in vivo base editor. 3
- A second fetal hemoglobin target. The BACH2–NRF2 finding raises the question of whether a pathway independent of BCL11A can carry a drug or an editor into development. 17
- Large-cargo delivery. The LC-1 lipid and Nature's survey of the bigger editors point the same way: the next advances in inserting whole genes depend on delivery. 19
References
- 1Beam sues 2 biotechs, alleging theft of base editing trade secrets
biopharmadive.com
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- 6
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- 8What's New for Biologics
fda.gov
- 9Beam sues rival startup, claims former scientist stole secrets
fiercebiotech.com
- 10
- 11
- 12
- 13Wave Life Sciences Aligns With FDA on Registrational Path for RNA Editing Therapy WVE-006 in AATD
biopharminternational.com
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- 18Cargo-Sized Lipid Screen Extends Gene Editing Beyond the Liver
crisprmedicinenews.com
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