Precision BioSciences Receives FDA Orphan Drug Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
FDA Orphan Drug Designation: Precision BioSciences has received Orphan Drug Designation from the FDA for its gene therapy PBGENE-DMD, aimed at treating Duchenne muscular dystrophy (DMD), highlighting the urgent need for effective treatments in this area.
Clinical Development Plans: The company is advancing towards clinical trials with initial data expected in 2026, following successful preclinical studies that demonstrated significant functional improvements in a mouse model of DMD.
Trade with 70% Backtested Accuracy
Analyst Views on DTIL
About DTIL
About the author
FDA Approval Enables Initiation of DMD Clinical Trial
- Clinical Trial Launch: The FDA's 'Study May Proceed' notification allows Precision BioSciences to initiate the FUNCTION-DMD Phase 1/2 clinical trial, aimed at evaluating the safety and efficacy of PBGENE-DMD in patients with Duchenne muscular dystrophy (DMD), marking a significant advancement in the gene editing field.
- Gene Editing Innovation: PBGENE-DMD employs a gene excision approach designed to permanently correct the dystrophin gene in approximately 60% of DMD patients, restoring near full-length functional dystrophin protein, which is expected to significantly improve functional outcomes for patients and offer potential long-term therapeutic effects.
- Investor Event Arrangement: The company plans to host a virtual investor event in March following the Muscular Dystrophy Association meeting to discuss the PBGENE-DMD program and the FUNCTION-DMD clinical study, featuring participation from multiple key opinion leaders, thereby enhancing collaboration with patients and the medical community.
- Future Outlook: The first clinical trial site is expected to be activated in the first half of 2026, with initial data anticipated by the end of 2026; if supportive data is obtained, the company will engage with the FDA to align on the regulatory path forward, advancing treatment options for DMD.
Precision BioSciences Receives FDA Approval for Clinical Trial
- FDA Clinical Trial Approval: Precision BioSciences received a 'Study May Proceed' notification from the FDA, allowing the initiation of the FUNCTION-DMD Phase 1/2 clinical trial to evaluate the safety and efficacy of PBGENE-DMD in patients with Duchenne muscular dystrophy, marking a significant advancement in the gene editing field.
- Gene Editing Innovation: PBGENE-DMD utilizes a gene excision approach targeting mutations in exons 45-55 affecting 60% of DMD patients, aiming to restore near full-length functional dystrophin protein, which is expected to significantly improve muscle function and address existing treatment gaps.
- Clinical Trial Progress: The company plans to activate its first clinical trial site in the U.S. in the first half of 2026, with initial data expected by year-end 2026; if results are supportive, the company will engage with the FDA to discuss the regulatory pathway forward, demonstrating its commitment to accelerating treatment progress.
- Investor Event Arrangement: Precision plans to host a virtual investor event in March following the Muscular Dystrophy Association meeting to discuss the PBGENE-DMD program and the FUNCTION-DMD clinical study, featuring participation from key opinion leaders in the medical and patient advocacy communities, enhancing communication with stakeholders.
Precision BioSciences Advances PBGENE-HBV and PBGENE-DMD Trials, Data Updates Expected in 2026
- Clinical Trial Progress: Precision BioSciences' PBGENE-HBV is currently undergoing the Phase 1/2a ELIMINATE-B trial, which has shown dose-dependent antiviral activity, with data updates expected at medical conferences in 2026, potentially offering a cure for chronic hepatitis B patients.
- Strong Financial Position: As of December 31, 2025, Precision anticipates having approximately $137 million in cash and cash equivalents, which is expected to fund key data milestones for PBGENE-HBV and PBGENE-DMD, ensuring operational stability through 2028.
- New DMD Treatment Approach: The PBGENE-DMD Phase 1/2 FUNCTION-DMD trial is expected to receive IND clearance in Q1 2026, aiming to provide durable functional muscle improvement for 60% of DMD patients, with initial data anticipated by the end of 2026.
- Collaborative Project Advancements: The ECUR-506 project with iECURE has reached key alignment with the FDA, supporting its potential as a curative treatment for neonatal OTC deficiency, with clinical trial data expected to be released in the first half of 2026, further enhancing Precision's market position.
iECURE Secures FDA RMAT Designation for ECUR-506, Advancing OTC Deficiency Treatment
- Regulatory Milestone: iECURE's ECUR-506 has received FDA's Regenerative Medicine Advanced Therapy (RMAT) designation, which will provide accelerated review and intensive guidance for its development in treating neonatal onset ornithine transcarbamylase deficiency, thereby expediting the clinical development process.
- Clinical Study Alignment: The company has aligned with the FDA on primary and secondary efficacy endpoints, comparators, and sample size for the ongoing OTC-HOPE study, laying the groundwork for a future Biologics License Application (BLA) and showcasing its potential in the rare disease space.
- Innovation Passport: ECUR-506 has also been awarded an Innovation Passport under the UK's Innovative Licensing and Access Pathway (ILAP), making it one of only three selected investigational medicinal products, underscoring its potential to address significant unmet medical needs.
- Clinical Data Support: Encouraging clinical data from the OTC-HOPE study, including a complete clinical response observed in the first treated infant, further validates the efficacy of ECUR-506 and may inform the development of other indications in the pipeline.
Biotech Stocks Surge in After-Hours Trading with Gains from BioAtla, Entero, and Sonnet
BioAtla Inc. Gains: BioAtla's shares surged 13.94% in after-hours trading, attributed to investor reactions to recent financing agreements, despite no new company-specific news.
Metagenomi Inc. and PAVmed Inc. Movements: Metagenomi's stock rose 5.71% with no new updates, while PAVmed advanced 5.72% after reporting a significant net loss and a sharp decline in revenue.
Precision BioSciences' Positive Outlook: Precision BioSciences gained 2.94% after announcing it will present Phase 1 trial data at an upcoming conference, boosting investor optimism.
Entero Therapeutics Rebranding: Entero Therapeutics' shares increased 3.68% following its announcement of a rebranding to GridAI Technologies Corp., effective December 1, 2025, along with a new ticker symbol.
Precision BioSciences Initiates $75 Million Stock and Warrant Sale
Stock Offering Announcement: Precision BioSciences announced an underwritten offering of approximately 10.8 million shares of common stock, along with warrants for an additional 5.4 million shares, priced at $6.14 per share.
Pre-Funded Warrants Details: Certain investors will receive pre-funded warrants for up to 1.4 million shares and half-warrants for 700,000 more shares, priced slightly lower at $6.139995 per unit.
Expected Proceeds: The gross proceeds from the offering are anticipated to be around $75 million before deducting underwriting discounts and expenses.
Additional Information: The announcement is part of Precision BioSciences' ongoing financial activities, including presentations at investment conferences and historical earnings data.
FDA Approval Enables Initiation of DMD Clinical Trial
- Clinical Trial Launch: The FDA's 'Study May Proceed' notification allows Precision BioSciences to initiate the FUNCTION-DMD Phase 1/2 clinical trial, aimed at evaluating the safety and efficacy of PBGENE-DMD in patients with Duchenne muscular dystrophy (DMD), marking a significant advancement in the gene editing field.
- Gene Editing Innovation: PBGENE-DMD employs a gene excision approach designed to permanently correct the dystrophin gene in approximately 60% of DMD patients, restoring near full-length functional dystrophin protein, which is expected to significantly improve functional outcomes for patients and offer potential long-term therapeutic effects.
- Investor Event Arrangement: The company plans to host a virtual investor event in March following the Muscular Dystrophy Association meeting to discuss the PBGENE-DMD program and the FUNCTION-DMD clinical study, featuring participation from multiple key opinion leaders, thereby enhancing collaboration with patients and the medical community.
- Future Outlook: The first clinical trial site is expected to be activated in the first half of 2026, with initial data anticipated by the end of 2026; if supportive data is obtained, the company will engage with the FDA to align on the regulatory path forward, advancing treatment options for DMD.
Precision BioSciences Receives FDA Approval for Clinical Trial
- FDA Clinical Trial Approval: Precision BioSciences received a 'Study May Proceed' notification from the FDA, allowing the initiation of the FUNCTION-DMD Phase 1/2 clinical trial to evaluate the safety and efficacy of PBGENE-DMD in patients with Duchenne muscular dystrophy, marking a significant advancement in the gene editing field.
- Gene Editing Innovation: PBGENE-DMD utilizes a gene excision approach targeting mutations in exons 45-55 affecting 60% of DMD patients, aiming to restore near full-length functional dystrophin protein, which is expected to significantly improve muscle function and address existing treatment gaps.
- Clinical Trial Progress: The company plans to activate its first clinical trial site in the U.S. in the first half of 2026, with initial data expected by year-end 2026; if results are supportive, the company will engage with the FDA to discuss the regulatory pathway forward, demonstrating its commitment to accelerating treatment progress.
- Investor Event Arrangement: Precision plans to host a virtual investor event in March following the Muscular Dystrophy Association meeting to discuss the PBGENE-DMD program and the FUNCTION-DMD clinical study, featuring participation from key opinion leaders in the medical and patient advocacy communities, enhancing communication with stakeholders.
Precision BioSciences Advances PBGENE-HBV and PBGENE-DMD Trials, Data Updates Expected in 2026
- Clinical Trial Progress: Precision BioSciences' PBGENE-HBV is currently undergoing the Phase 1/2a ELIMINATE-B trial, which has shown dose-dependent antiviral activity, with data updates expected at medical conferences in 2026, potentially offering a cure for chronic hepatitis B patients.
- Strong Financial Position: As of December 31, 2025, Precision anticipates having approximately $137 million in cash and cash equivalents, which is expected to fund key data milestones for PBGENE-HBV and PBGENE-DMD, ensuring operational stability through 2028.
- New DMD Treatment Approach: The PBGENE-DMD Phase 1/2 FUNCTION-DMD trial is expected to receive IND clearance in Q1 2026, aiming to provide durable functional muscle improvement for 60% of DMD patients, with initial data anticipated by the end of 2026.
- Collaborative Project Advancements: The ECUR-506 project with iECURE has reached key alignment with the FDA, supporting its potential as a curative treatment for neonatal OTC deficiency, with clinical trial data expected to be released in the first half of 2026, further enhancing Precision's market position.
iECURE Secures FDA RMAT Designation for ECUR-506, Advancing OTC Deficiency Treatment
- Regulatory Milestone: iECURE's ECUR-506 has received FDA's Regenerative Medicine Advanced Therapy (RMAT) designation, which will provide accelerated review and intensive guidance for its development in treating neonatal onset ornithine transcarbamylase deficiency, thereby expediting the clinical development process.
- Clinical Study Alignment: The company has aligned with the FDA on primary and secondary efficacy endpoints, comparators, and sample size for the ongoing OTC-HOPE study, laying the groundwork for a future Biologics License Application (BLA) and showcasing its potential in the rare disease space.
- Innovation Passport: ECUR-506 has also been awarded an Innovation Passport under the UK's Innovative Licensing and Access Pathway (ILAP), making it one of only three selected investigational medicinal products, underscoring its potential to address significant unmet medical needs.
- Clinical Data Support: Encouraging clinical data from the OTC-HOPE study, including a complete clinical response observed in the first treated infant, further validates the efficacy of ECUR-506 and may inform the development of other indications in the pipeline.
Biotech Stocks Surge in After-Hours Trading with Gains from BioAtla, Entero, and Sonnet
BioAtla Inc. Gains: BioAtla's shares surged 13.94% in after-hours trading, attributed to investor reactions to recent financing agreements, despite no new company-specific news.
Metagenomi Inc. and PAVmed Inc. Movements: Metagenomi's stock rose 5.71% with no new updates, while PAVmed advanced 5.72% after reporting a significant net loss and a sharp decline in revenue.
Precision BioSciences' Positive Outlook: Precision BioSciences gained 2.94% after announcing it will present Phase 1 trial data at an upcoming conference, boosting investor optimism.
Entero Therapeutics Rebranding: Entero Therapeutics' shares increased 3.68% following its announcement of a rebranding to GridAI Technologies Corp., effective December 1, 2025, along with a new ticker symbol.
Precision BioSciences Initiates $75 Million Stock and Warrant Sale
Stock Offering Announcement: Precision BioSciences announced an underwritten offering of approximately 10.8 million shares of common stock, along with warrants for an additional 5.4 million shares, priced at $6.14 per share.
Pre-Funded Warrants Details: Certain investors will receive pre-funded warrants for up to 1.4 million shares and half-warrants for 700,000 more shares, priced slightly lower at $6.139995 per unit.
Expected Proceeds: The gross proceeds from the offering are anticipated to be around $75 million before deducting underwriting discounts and expenses.
Additional Information: The announcement is part of Precision BioSciences' ongoing financial activities, including presentations at investment conferences and historical earnings data.
