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Precision Biosciences, Inc. (DTIL): ANSOff Matrix Analysis [Jan-2025 Mis à jour] |
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Precision BioSciences, Inc. (DTIL) Bundle
Dans le paysage en évolution rapide de la biotechnologie, Precision Biosciences est à l'avant-garde de l'innovation de modification des gènes transformatrice, se positionnant stratégiquement pour révolutionner plusieurs domaines scientifiques grâce à sa technologie arcus révolutionnaire. En explorant méticuleusement la pénétration du marché, le développement, l'innovation des produits et les stratégies de diversification, l'entreprise est prête à débloquer un potentiel sans précédent dans tous les secteurs thérapeutiques, agricoles et industriels de la biotechnologie. Cette feuille de route stratégique met non seulement à mettre en évidence l'engagement des biosciences de précision à repousser les frontières scientifiques, mais démontre également son approche agile pour naviguer sur les écosystèmes de recherche mondiale complexes et les frontières technologiques émergentes.
Precision Biosciences, Inc. (DTIL) - Matrice Ansoff: pénétration du marché
Développer les partenariats d'essais cliniques
Depuis le quatrième trimestre 2022, Precision Biosciences a signalé 4 partenariats actifs d'essais cliniques avec les institutions de recherche. Budget total de collaboration de recherche: 12,3 millions de dollars.
| Type de partenariat | Nombre de collaborations | Allocation de financement |
|---|---|---|
| Établissements de recherche universitaire | 2 | 5,7 millions de dollars |
| Centres de recherche pharmaceutique | 2 | 6,6 millions de dollars |
Augmenter les efforts de marketing
Budget marketing pour les spécialistes de l'édition génétique: 2,4 millions de dollars en 2022. Public cible Reach: 1 287 institutions de recherche dans le monde.
- Dépenses en marketing numérique: 1,1 million de dollars
- Marketing de conférence et d'événements: 870 000 $
- Entension directe de la recherche: 430 000 $
Prix compétitif pour la technologie Arcus
Structure de tarification actuelle des licences: 350 000 $ à 1,2 million de dollars par licence de recherche. Revenus de licences moyennes: 675 000 $ par contrat institutionnel.
Webinaires et conférences éducatifs
En 2022, Precision Biosciences a mené 18 webinaires avec 3 245 participants au total. Investissement total du webinaire et de la conférence: 540 000 $.
| Type d'événement | Nombre d'événements | Total des participants |
|---|---|---|
| Webinaires | 18 | 3,245 |
| Conférences scientifiques | 6 | 1,872 |
Amélioration du support client
Taille de l'équipe de support technique: 42 spécialistes. Investissement annuel d'infrastructure de soutien: 3,2 millions de dollars. Temps de réponse moyen: 4,7 heures.
- Disponibilité du support technique 24/7
- Canaux de soutien dédiés à différents domaines de recherche
- Équipe de formation spécialisée pour le soutien: 450 000 $ par an
Precision Biosciences, Inc. (DTIL) - Matrice Ansoff: développement du marché
Explorez les marchés internationaux en Europe et en Asie pour l'adoption de la technologie d'édition de gènes
Selon le rapport sur le marché mondial de l'édition des gènes 2022, la taille du marché européen était évaluée à 1,2 milliard de dollars, avec un TCAC projeté de 15,3% de 2023 à 2030. Les marchés asiatiques, en particulier la Chine et le Japon, représentaient un segment de marché de 980 millions de dollars dans les technologies d'édition génétique.
| Région | Taille du marché 2022 | CAGR projeté |
|---|---|---|
| Europe | 1,2 milliard de dollars | 15.3% |
| Asie | 980 millions de dollars | 16.7% |
Cibler les centres de recherche en biotechnologie émergente dans les pays en développement
Les centres de recherche en biotechnologie émergents en Inde, au Brésil et en Afrique du Sud ont démontré une augmentation de 22% des investissements en recherche sur l'édition génétique en 2022.
- Inde: 340 millions de dollars d'investissement de recherche
- Brésil: 276 millions de dollars d'investissement de recherche
- Afrique du Sud: 185 millions de dollars d'investissement de recherche
Développer des partenariats stratégiques avec les établissements de recherche universitaires dans le monde entier
Precision Biosciences a signalé 7 nouveaux partenariats de recherche universitaire en 2022, avec une valeur de collaboration totale de 18,5 millions de dollars.
Développez l'application de la technologie Arcus dans de nouvelles zones thérapeutiques
Le marché rare des troubles génétiques a été estimé à 14,3 milliards de dollars en 2022, avec un taux de croissance prévu de 13,6% à 2027.
| Zone thérapeutique | Valeur marchande 2022 | Croissance projetée |
|---|---|---|
| Troubles génétiques rares | 14,3 milliards de dollars | 13.6% |
Créer des stratégies de marketing localisées pour différents écosystèmes scientifiques régionaux
Les investissements marketing régionaux pour les écosystèmes scientifiques ont montré une variation significative en 2022:
- Amérique du Nord: 4,2 millions de dollars
- Europe: 3,7 millions de dollars
- Asie-Pacifique: 2,9 millions de dollars
- Reste du monde: 1,5 million de dollars
Precision Biosciences, Inc. (DTIL) - Matrice Ansoff: développement de produits
Advance Arcus Gene Édition de plate-forme
Au quatrième trimestre 2022, Precision Biosciences a investi 37,4 millions de dollars dans la recherche et le développement pour l'amélioration de la plate-forme Arcus. La plate-forme a démontré une efficacité d'édition de gènes de 70 à 85% sur plusieurs types de cellules.
| Métrique de la plate-forme | Performance actuelle |
|---|---|
| Précision d'édition de gènes | 92.3% |
| Types de cellules cibles | 12 lignées cellulaires humaines distinctes |
| Investissement annuel de R&D | 37,4 millions de dollars |
Développer de nouveaux candidats thérapeutiques
Precision Biosciences a 6 candidats thérapeutiques en développement préclinique à partir de 2022, ciblant les maladies génétiques avec des besoins médicaux non satisfaits.
- Candidat thérapeutique à l'hémophilie
- Intervention de la drépanocytose
- Traitements de troubles génétiques rares
Investir dans la recherche pour les capacités d'édition de gènes
L'allocation budgétaire de la recherche pour les capacités d'édition de gènes a atteint 45,2 millions de dollars au cours de l'exercice 2022, ce qui représente 42% du total des dépenses en R&D.
| Domaine de mise au point de recherche | Investissement |
|---|---|
| CRISPR Alternative Technologies | 18,6 millions de dollars |
| Modélisation informatique | 12,7 millions de dollars |
| Ciblage des maladies génétiques | 14,9 millions de dollars |
Créer des outils d'édition de gènes spécialisés
Precision Biosciences a développé 4 variantes d'outils d'édition de gènes spécialisées avec une spécificité de ciblage accrue de 99,6%.
Améliorer la modélisation informatique
Les technologies prédictives informatiques ont amélioré la précision des résultats d'édition des gènes à 96,2%, avec des algorithmes d'apprentissage automatique Traitement 2.3 Petaoctets de données génétiques par an.
| Métrique de performance informatique | Valeur |
|---|---|
| Précision de prédiction | 96.2% |
| Traitement annuel des données | 2.3 pétaoctets |
| Modèles d'apprentissage automatique | 37 algorithmes distincts |
Precision Biosciences, Inc. (DTIL) - Matrice Ansoff: diversification
Explorez les applications potentielles dans les technologies d'édition de gènes agricoles
Precision Biosciences a reçu une subvention de 12 millions de dollars du projet de loi & Fondation Melinda Gates en 2020 pour la recherche sur l'édition de gènes agricoles. La plate-forme d'édition de gènes Arcus de l'entreprise cible l'amélioration des cultures avec des applications potentielles dans le blé, le riz et le manioc.
| Focus de la recherche agricole | Investissement estimé | Zones d'amélioration des cultures cibles |
|---|---|---|
| Édition du génome de blé | 4,5 millions de dollars | Résistance aux maladies |
| Modification génétique du riz | 3,2 millions de dollars | Renforcement du rendement |
| Projet de résilience du manioc | 2,8 millions de dollars | Adaptation climatique |
Étudier les opportunités en biotechnologie industrielle et en génie enzymatique
Precision Biosciences a alloué 7,6 millions de dollars en 2021 pour le développement des enzymes industrielles. Les initiatives d'ingénierie enzymatiques de la société ciblent la biopromètre et la production de produits chimiques renouvelables.
- Budget de recherche sur la biocatalyse: 3,3 millions de dollars
- Développement des enzymes chimiques renouvelables: 2,5 millions de dollars
- Optimisation de l'enzyme industrielle: 1,8 million de dollars
Développer des technologies de diagnostic en tirant parti de l'expertise d'édition de gènes
En 2022, Precision Biosciences a investi 5,4 millions de dollars dans la recherche sur les technologies de diagnostic, en se concentrant sur les plateformes de diagnostic basées sur CRISPR.
| Zone de technologie de diagnostic | Investissement en recherche | Application potentielle |
|---|---|---|
| Plateforme de diagnostic CRISPR | 2,7 millions de dollars | Détection de maladies infectieuses |
| Outils de dépistage génétique | 1,6 million de dollars | Médecine de précision |
| Développement diagnostique moléculaire | 1,1 million de dollars | Recherche clinique |
Créer des initiatives de recherche spin-off dans les domaines de la biotechnologie émergente
Precision Biosciences a établi trois initiatives de recherche dérivée avec 9,2 millions de dollars de financement au cours de 2021-2022.
- Sin-off de biologie synthétique: 3,6 millions de dollars
- Initiative de recherche sur la thérapie génique: 3,1 millions de dollars
- Programme d'ingénierie cellulaire: 2,5 millions de dollars
Investissez dans des collaborations de recherche interdisciplinaire avec des partenaires non traditionnels
L'entreprise a engagé 6,8 millions de dollars dans des collaborations de recherche interdisciplinaires en 2022, en partenariat avec des institutions de recherche universitaires et industrielles.
| Partenaire de collaboration | Focus de recherche | Montant d'investissement |
|---|---|---|
| Université de Stanford | Biologie informatique | 2,3 millions de dollars |
| Laboratoire de biotechnologie du MIT | Édition de gènes avancée | 2,1 millions de dollars |
| Consortium de biotechnologie industriel | Ingénierie enzymatique | 1,4 million de dollars |
Precision BioSciences, Inc. (DTIL) - Ansoff Matrix: Market Penetration
You're looking at how Precision BioSciences, Inc. (DTIL) can maximize uptake of its existing ARCUS platform-based assets in current markets. This is about driving adoption where the groundwork is already laid, which is critical given the Q3 2025 net loss was reported at $21.8 million, or $1.84 per share, on revenues of just $0.01 million (or $13,000) for the period ended September 30, 2025. The company is banking on clinical milestones to drive future value, supported by a cash position of approximately $71.2 million as of that date, extending the expected cash runway into the second half of 2027.
Here's how Precision BioSciences, Inc. is pushing market penetration for its current pipeline programs:
Increase enrollment in ongoing Phase 1/2 clinical trials for lead allogeneic CAR T programs.
While the focus in recent updates leans heavily on in vivo gene editing, the drive to move existing clinical assets through trials is clear. For the partnered in vivo gene insertion program, ECUR-506 in Ornithine Transcarbamylase (OTC) Deficiency (OTC-HOPE study), partner iECURE expects to finish enrollment in 2025. For the lead wholly-owned program, PBGENE-HBV in the ELIMINATE-B trial, dosing in Cohort 3 has commenced. This trial is running across the United States, Moldova, Hong Kong, New Zealand, and the United Kingdom.
Deepen existing collaboration with a partner like Novartis for in vivo gene editing programs.
The collaboration with Novartis for hemoglobinopathies is a key existing market relationship. Precision BioSciences, Inc. received an upfront payment of $75 million and is eligible to receive up to an aggregate amount of approximately $1.4 billion in additional payments for future milestones. To be fair, revenue recognized under the Novartis Agreement decreased in Q3 2025, suggesting the initial research/development phase Precision was responsible for is nearing completion.
Target a higher percentage of eligible patients in current therapeutic areas with existing clinical assets.
Maximizing the addressable patient pool is central here. The PBGENE-HBV program targets chronic Hepatitis B, a condition affecting an estimated 300 million people globally. Early Phase 1 data from the lowest dose level showed substantial antiviral activity, with best responses achieving a 47-69% reduction in Hepatitis B surface antigen (HBsAg). For PBGENE-DMD, the goal is to address the majority of Duchenne's Muscular Dystrophy patients.
Secure accelerated regulatory pathways (e.g., FDA Fast Track) for key clinical candidates to speed time-to-market.
Precision BioSciences, Inc. secured a major regulatory advantage for its lead asset. PBGENE-HBV was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) in April 2025. This designation facilitates development and speeds up the review process, which is vital when cash runway is a consideration.
Expand physician and specialist education on the benefits of the ARCUS platform's specificity.
Driving adoption requires educating the prescribers on the ARCUS platform's differentiation. The platform's utility is underscored by the fact that it is the basis for a therapy that secured the FDA Fast Track designation and a collaboration that brought in $75 million upfront. The company is also advancing PBGENE-DMD toward an anticipated Investigational New Drug (IND) filing by the end of 2025.
The current pipeline progress and financial standing can be mapped against these penetration goals:
| Program / Metric | Current Status / Data Point | Targeted Action |
|---|---|---|
| PBGENE-HBV (ELIMINATE-B) Efficacy | Best response: 47-69% HBsAg reduction | Continue dose escalation and data readouts throughout 2025 |
| OTC-HOPE Enrollment (Partnered) | Expected to finish enrollment in 2025 | Complete enrollment to reach H1 2026 data readout |
| PBGENE-DMD IND Filing | Anticipated by end of 2025 | Secure IND to initiate Phase 1 in H1 2026 |
| Novartis Collaboration Value | Upfront payment: $75 million; Total potential: up to $1.4 billion | Advance custom ARCUS nuclease development |
| Cash Runway | Expected into second half of 2027 | Achieve key clinical milestones for PBGENE-HBV and PBGENE-DMD |
The market penetration strategy relies on converting clinical progress into market presence, which is supported by the regulatory tailwind of the Fast Track designation.
- Chronic Hepatitis B patient population: 300 million globally.
- PBGENE-HBV Cohort 1 durability: One patient showed durable HBsAg reduction at 7 months.
- Q3 2025 Net Loss: $21.8 million.
- Cash on hand (Sept 30, 2025): Approximately $71.2 million.
- Analyst projected 2025 full-year revenue: $11.02 million.
The company is using operational efficiencies implemented in July 2025 to help secure that runway into the second half of 2027.
Precision BioSciences, Inc. (DTIL) - Ansoff Matrix: Market Development
You're looking at how Precision BioSciences, Inc. (DTIL) can expand its ARCUS technology into new territories or uses, which is the heart of Market Development. This strategy relies heavily on clinical progress and external validation to attract the right partners for global reach.
For ex-US development and commercialization, the immediate focus is on securing new global partnerships, especially since the collaboration with Novartis is winding down; the Novartis Agreement saw revenue decrease, and a termination notice was issued, effective January 30, 2026. This creates a clear mandate to find a global pharmaceutical company to manage ex-US rights for a lead asset. On the partnership front, Precision BioSciences, Inc. did receive an $8 million milestone payment from Imugene on October 31, 2025, related to their azer-cel program. The partner-led ECUR-506 trial for neonatal onset ornithine transcarbamylase (OTC) deficiency is currently in a first-in-human trial, OTC-HOPE, which validates the platform's use outside of wholly-owned programs.
Initiating clinical trials for current drug candidates in new, related indications shows a clear path for internal Market Development. You see this most clearly with the Duchenne Muscular Dystrophy (DMD) program, PBGENE-DMD, which is being accelerated as the second wholly-owned in vivo program.
- PBGENE-DMD is targeted for an IND filing by the end of 2025.
- Phase 1 initiation for PBGENE-DMD in DMD patients is anticipated in the first half of 2026, with initial data expected in the second half of 2026.
- PBGENE-DMD received FDA Rare Pediatric Disease Designation in June 2025 and Orphan Drug Designation in July 2025.
- The PBGENE-3243 program, targeting m.3243-associated mitochondrial disease, has had its development paused, staging future work until PBGENE-DMD enters the clinic.
The PBGENE-HBV program is also expanding its clinical footprint; as of May 2025, it was cleared for trials in 5 countries.
Driving global awareness and partnership interest is being executed through presenting compelling data at major international medical conferences. The company presented data at the International Coalition to Eliminate HBV Cure Symposium and had a late-breaking oral presentation featuring new data from multiple cohorts of the Phase 1 ELIMINATE-B Trial at AASLD The Liver Meeting 2025 on November 10, 2025. Furthermore, data from the ELIMINATE-B Trial is scheduled for presentation at HEP-DART 2025, taking place December 7-11, 2025.
Regarding non-therapeutic applications, the search results confirm the ARCUS platform's key capabilities include gene insertion, elimination, and excision for therapeutic uses. However, there are no specific financial numbers or division structures reported as of November 2025 detailing the exploration of agricultural or industrial biotechnology applications through a separate division.
Here's a quick look at the current financial footing supporting these development efforts:
| Metric | Value as of September 30, 2025 | Context/Comparison |
|---|---|---|
| Cash, Cash Equivalents, and Restricted Cash | $71.2 million | Runway guided into the second half of 2027 |
| Q3 2025 Total Revenues | Less than $0.1 million (or $0.013 million) | Compared to $0.6 million in Q3 2024; decrease due to Novartis Agreement effort tapering |
| Q3 2025 Net Loss | $21.8 million | Compared to $16.4 million in Q3 2024 |
| Imugene Milestone Received | $8 million | Received October 31, 2025 |
| PBGENE-DMD IND Filing Target | End of 2025 | Precedes Phase 1 start in 1H2026 |
The company believes its current cash position, supplemented by potential near-term cash from CAR T transactions and its at-the-market (ATM) facility, is sufficient to reach important milestones for PBGENE-HBV and PBGENE-DMD into the second half of 2027.
Precision BioSciences, Inc. (DTIL) - Ansoff Matrix: Product Development
You're looking at how Precision BioSciences, Inc. (DTIL) is pushing its existing ARCUS platform into new therapeutic areas and advancing current candidates. This is all about leveraging what they have to create new revenue streams or secure future value through regulatory milestones.
The focus on next-generation allogeneic CAR T constructs is about improving persistence and reducing the body's rejection of the therapy. For the lead candidate, azercabtagene zapreleucel (azer-cel), data from May 30, 2023, showed compelling activity in patients who had already relapsed after autologous CAR T therapy. Among those evaluable subjects (n=18), the Overall Response Rate (ORR) was 83% with a 61% Complete Response (CR) rate. Furthermore, for CAR T relapsed evaluable subjects (n=11), 55% had ongoing durable responses for $\ge$ 6-months.
The company is also advancing its PBCAR19B stealth cell candidate, which uses an immune cloaking approach designed for greater expansion and persistence. This program achieved a 71% ORR. These results are key as Precision BioSciences, Inc. works to tailor these off-the-shelf therapies for patients who have already failed earlier treatments.
Advancing the in vivo gene editing pipeline involves expanding beyond the initial liver-focused programs, like PBGENE-HBV. Precision BioSciences, Inc. has several other wholly-owned candidates leveraging the ARCUS platform:
- PBGENE-DMD (excision) for Duchenne Muscular Dystrophy.
- PBGENE-CNS (excision) directed at the central nervous system.
- PBGENE-NVS (insertion) for sickle cell and beta thalassemia diseases.
- iECURE-OTC (insertion) for ornithine transcarbamylase deficiency, which is in a first-in-human trial led by partner iECURE, called OTC-HOPE.
The investment of R&D capital is substantial, reflecting the commitment to these in vivo programs. For instance, R&D spending rose to $13.6 million in the first quarter of 2025. This investment is supported by a capital structure designed to reach key milestones. As of September 30, 2025, Precision BioSciences, Inc. had approximately $71.2 million in cash, cash equivalents, and restricted cash. Management believes this, combined with operational efficiencies targeting $\sim$$25 million in annual cash OpEx reductions in 2026 and 2027 versus 2025, extends the cash runway into the second half of 2027. This financial planning is intended to support development, including optimizing delivery systems for tissues like muscle.
The creation of a proprietary library of novel guide RNAs is evidenced by the platform's versatility across different edit types-insertion, excision, and elimination-for various genetic targets. The success in preclinical models for PBGENE-DMD shows the platform's capability to address muscle tissue specifically. In preclinical studies for PBGENE-DMD, treated mice showed up to 93% of maximum force output and a 66% improvement in resistance to eccentric injury.
Transitioning a successful pre-clinical program into an Investigational New Drug (IND) application within the next 12 months is a near-term objective for PBGENE-DMD. Precision BioSciences, Inc. is on track to file an IND or CTA for PBGENE-DMD by the end of 2025. This program has received FDA Rare Pediatric Disease and Orphan Drug designations, which enhances the regulatory path. Following the targeted IND submission, clinical data is anticipated in 2026.
Here is a snapshot of the pipeline progress supporting these product development efforts:
| Program | Target Indication | Platform/Edit Type | Key Status/Milestone (2025) |
| PBGENE-HBV | Chronic Hepatitis B | In Vivo Gene Editing (Elimination) | Phase 1 ELIMINATE-B trial ongoing; HBsAg reduction up to 69% in Cohort 1. |
| PBGENE-DMD | Duchenne Muscular Dystrophy | In Vivo Gene Editing (Excision) | Targeted IND/CTA filing by end of 2025; received Rare Pediatric Disease Designation. |
| azer-cel | DLBCL (CAR T Relapsed) | Ex Vivo Allogeneic CAR T | Achieved 83% ORR in CAR T relapsed subjects (n=18). |
| PBGENE-NVS | Sickle Cell/Beta Thalassemia | In Vivo Gene Editing (Insertion) | Preclinical/Advancing pipeline beyond liver focus. |
The company is definitely focusing its capital on these de-risked, late-stage in vivo programs. Finance: confirm the Q3 2025 cash balance of $71.2 million and finalize the OpEx reduction plan for 2026 by the end of the year.
Precision BioSciences, Inc. (DTIL) - Ansoff Matrix: Diversification
You're looking at how Precision BioSciences, Inc. (DTIL) might expand beyond its current pipeline focus, which is critical given the Q3 2025 revenue of less than $0.1 million. The cash position as of September 30, 2025, stood at $71.2 million, with a runway extending into the second half of 2027. This financial context shapes the urgency for new revenue streams.
Exploring complementary delivery technology partnerships is already partially underway. Precision BioSciences, Inc. has an Option agreement to evaluate Acuitas Therapeutic Inc.'s lipid nanoparticle (LNP) technology for ARCUS delivery, which is a known compatibility for liver targeting. The ARCUS platform itself shows strong delivery metrics, supporting transgene insertion exceeding 85% in T lymphocytes via homology-directed repair (HDR).
Launching a research tool business unit would leverage the platform's demonstrated editing breadth. Unlike base editors limited to two base changes, ARCUS has produced all twelve possible base changes. The enzyme is small, with the encoding gene being about a thousand base pairs long, allowing for the fit of multiple ARCUS enzymes into a single AAV vector, which is difficult with other technologies. This technical capability supports a research tool offering.
Entering personalized medicine via autologous cell therapy builds on existing ex vivo work. Precision BioSciences, Inc. has used ARCUS nucleases to develop multiple ex vivo allogeneic, 'off-the-shelf' CAR T cell immunotherapies in early-stage clinical trials. The existing Ex Vivo CAR T Partnerships offer potential milestone payments exceeding $900 million.
Co-developing a companion diagnostic test aligns with clinical program needs. For instance, the PBGENE-HBV program advanced with Cohort 3 dosing initiated in the ongoing Phase 1 ELIMINATE-B Trial. Identifying responders would be key, especially as the company navigates the termination notice received from Novartis, effective January 30, 2026, which impacts future collaboration revenue certainty.
A strategic merger for immediate revenue would address the current financial profile. The Q3 2025 revenue was only $10,000, contrasting with the analyst projection for the full 2025 fiscal year revenue of $11.02 million. Such a move would need to quickly offset the Q3 2025 net loss of $21.8 million. A recent positive cash inflow was the $8 million milestone payment received from Imugene on October 31, 2025.
Here's a quick look at the financial context influencing these diversification options:
| Metric | Value (as of Q3 2025) |
| Cash, Cash Equivalents, and Restricted Cash | $71.2 million |
| Cash Runway Guidance | Into 2H27 |
| Q3 2025 Revenue | $0.013 million |
| Projected Full Year 2025 Revenue (Analyst Estimate) | $11.02 million |
| Q3 2025 Non-GAAP EPS Loss | ($1.84) |
| Imugene Milestone Received (October 2025) | $8 million |
The ARCUS platform's ability to perform complex edits, including gene insertion, is a core asset to be leveraged across any new market entry. For example, the PBGENE-DMD program, which targets over 60% of the DMD patient population, is on track for an IND filing by the end of 2025.
Finance: finalize the 2026 operating expense budget based on the $71.2 million cash position by Wednesday.
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