Dabocemagene Autoficel (D-Fi/FCX-007): An Investigational Gene Therapy for Dystrophic Epidermolysis Bullosa

1. Executive Summary

Dabocemagene autoficel, also known as D-Fi and FCX-007, is an investigational ex vivo, autologous cell-based gene therapy developed by Castle Creek Biosciences Inc. It is designed to treat Dystrophic Epidermolysis Bullosa (DEB), a group of severe inherited skin disorders characterized by extreme skin fragility and blistering. The therapy involves genetically modifying a patient's own dermal fibroblasts using a lentiviral vector to express functional type VII collagen (COL7), the protein deficient in DEB. This approach aims to restore anchoring fibril formation at the dermal-epidermal junction, thereby improving wound healing and skin integrity.[1]

Early-phase clinical trials provided initial positive signals regarding COL7 expression and wound healing.[4] However, the clinical development program faced a significant setback when a pivotal Phase 3 trial (DeFi-RDEB, NCT04213261) was terminated in 2023 due to the unavailability of the lentiviral vector, leading to a period where development appeared stalled.[6] Despite this challenge, the program has demonstrated notable resilience. In early 2025, Castle Creek Biosciences secured $75 million in royalty financing, primarily to support a renewed Phase 3 effort for D-Fi.[5] This financial backing has enabled the initiation of a new Phase 3 study, NCT06892639, which commenced recruitment in March 2025.[1] This sequence of events, from a critical manufacturing-related trial failure to substantial new investment and the launch of a new pivotal study, highlights both the inherent complexities in gene therapy development, particularly concerning viral vector supply chains, and a persistent conviction in the therapeutic potential of dabocemagene autoficel.

Dabocemagene autoficel has received multiple U.S. Food and Drug Administration (FDA) designations, including Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) status, underscoring the significant unmet medical need in DEB.[4] The therapy is entering an evolving treatment landscape with recently approved therapies for DEB, and its ultimate success will depend on robust data from the new Phase 3 trial, consistent manufacturing, and a favorable long-term safety profile.

2. Introduction to Dabocemagene autoficel (D-Fi/FCX-007) and Dystrophic Epidermolysis Bullosa (DEB)

2.1. Overview of Dystrophic Epidermolysis Bullosa (DEB)

Dystrophic Epidermolysis Bullosa (DEB) is an ultra-rare, inherited genetic skin disorder, representing one of the four main subtypes of Epidermolysis Bullosa (EB).[9] It is characterized by profound skin fragility, where even minor mechanical friction or trauma to the skin or mucous membranes results in the formation of painful blisters and chronic wounds.[9] DEB encompasses a spectrum of severity, with Recessive Dystrophic Epidermolysis Bullosa (RDEB) typically manifesting as a more generalized and severe condition compared to Dominant Dystrophic Epidermolysis Bullosa (DDEB).[9] RDEB is widely regarded as the most critical and life-limiting form of EB.[10]

The clinical manifestations of RDEB are extensive and debilitating. Patients often suffer from chronic, non-healing wounds, extensive scarring, malnutrition due to oral and esophageal involvement, anemia, esophageal strictures requiring dilation, and growth retardation.[9] Musculoskeletal complications include pseudosyndactyly (the fusion of fingers and toes into "mitten" deformities) and joint contractures, leading to significant functional impairment. Dental anomalies, microstomia (restricted mouth opening), and corneal abrasions further compound the disease burden.[9] A particularly grave complication for individuals with severe RDEB is an elevated risk of developing aggressive cutaneous squamous cell carcinoma (SCC), which often arises in areas of chronic inflammation and scarring, typically in the second decade of life, and is a major cause of premature mortality.[9] The profound impact of these manifestations on patients' quality of life and the limited availability of effective, disease-modifying treatments underscore a significant unmet medical need.[8] The prevalence of DEB is estimated at approximately 1.35 cases per 1 million live births, and it accounts for roughly 25% of all EB cases.[9]

2.2. The Genetic Basis: COL7A1 Gene and Type VII Collagen (COL7)

The underlying cause of DEB lies in mutations within the COL7A1 gene.[4] This gene encodes the instructions for synthesizing type VII collagen (COL7), a large protein that is the principal structural component of anchoring fibrils.[2] Anchoring fibrils are specialized macromolecular structures located at the dermal-epidermal junction (DEJ), the interface between the epidermis (the outermost layer of the skin) and the dermis (the underlying connective tissue layer).[2] Their primary function is to secure the epidermis to the dermis, providing mechanical stability and resilience to the skin.[2]

In individuals with DEB, pathogenic mutations in COL7A1 lead to a deficiency or complete absence of functional COL7 protein. This, in turn, results in poorly formed or absent anchoring fibrils.[2] Consequently, the adhesion between the epidermal and dermal layers is compromised, leading to tissue cleavage and the characteristic blistering, erosions, and extreme skin fragility observed in DEB upon minimal trauma.[2] Patients with RDEB, the more severe form, typically exhibit a near-total or complete absence of functional COL7, correlating with the severity of their clinical phenotype.[4]

2.3. Introduction to Dabocemagene autoficel (D-Fi/FCX-007)

Dabocemagene autoficel, also known by its developmental codes D-Fi and FCX-007, is an investigational ex vivo, autologous cell-based gene therapy.[1] It is under development by Castle Creek Biosciences Inc. [1] as a potential treatment for DEB.

The therapeutic concept involves harvesting a patient's own (autologous) dermal fibroblasts, which are then genetically modified in a laboratory setting. This modification is achieved using a lentiviral vector to introduce a functional copy of the human COL7A1 gene into these cells.[2] The primary goal of dabocemagene autoficel is to address the root cause of DEB by enabling these modified fibroblasts, once administered back to the patient, to produce and secrete functional COL7 protein directly at the site of wounds.[2] This localized production of COL7 is intended to promote the formation of new anchoring fibrils, restore integrity to the DEJ, facilitate wound healing, and improve the overall stability of the skin.[2] The use of autologous cells is a key feature of this personalized medicine approach, designed to minimize the risk of immunological rejection of the transplanted cells. However, as with any gene therapy introducing a new or corrected protein, the potential for an immune response to the newly expressed COL7 was a consideration monitored in early clinical trials.[13]

3. Pharmacological Profile and Manufacturing

3.1. Drug Identification and Basic Characteristics

Dabocemagene autoficel is a complex biological investigational product. Its key identifiers and characteristics are summarized in Table 1.

Table 1: Dabocemagene autoficel - Key Characteristics

Characteristic	Details	Reference(s)
Generic Name	Dabocemagene autoficel	1
Other Names/Codes	D-Fi, FCX-007	2
DrugBank ID	DB17878	[User Query]
CAS Number	2365353-63-3	1
UNII	0S9A4XK1WT	1
Developer	Castle Creek Biosciences Inc.	1
Drug Type	Biotech; Ex vivo, autologous cell-based gene therapy; Cell transplant therapy (autologous)	1
Full Description	Autologous human dermal fibroblast cells derived from skin biopsies, and transduced with a non-replicating, self-inactivating lentivirus vector that encodes human collagen type VII alpha 1 (COL7A1) under the control of a human cytomegalovirus (CMV) promoter	3
Target Gene	COL7A1	2
Therapeutic Protein	Type VII Collagen (COL7)	2
Indication	Dystrophic Epidermolysis Bullosa (DEB), with a primary focus on Recessive DEB (RDEB)	4
Administration Route	Intradermal injection	2
Vector Type	Self-inactivating (SIN) Lentiviral Vector	2

3.2. Mechanism of Action

Dabocemagene autoficel is engineered to directly address the molecular defect underlying DEB: the deficiency of functional type VII collagen (COL7).[2] The therapy utilizes a patient's own dermal fibroblasts, which are harvested and then genetically modified ex vivo. This genetic modification involves the use of a self-inactivating (SIN) lentiviral vector to deliver a correct and functional copy of the human COL7A1 gene into these fibroblasts.[2] Lentiviral vectors are chosen for their ability to efficiently transduce both dividing and non-dividing cells and to integrate the therapeutic gene into the host cell's genome, potentially leading to long-term expression of the transgene.[17] The use of a SIN vector is a critical safety feature; these vectors are designed with deletions in the U3 region of the 3' long terminal repeat (LTR), which, after reverse transcription and integration, results in a transcriptionally attenuated LTR. This design minimizes the risk of the vector's own regulatory elements activating nearby host genes, particularly proto-oncogenes, thereby reducing the potential for insertional oncogenesis.[2] The expression of the COL7A1 transgene is driven by a human cytomegalovirus (CMV) promoter, a strong constitutive promoter selected to ensure robust production of the COL7 protein by the modified cells.[3]

Once these genetically engineered autologous fibroblasts are administered back to the patient via intradermal injection into wound areas, they are intended to locally produce and secrete functional COL7 protein.[2] This newly synthesized COL7 is expected to assemble into anchoring fibrils at the dermal-epidermal junction (DEJ). Anchoring fibrils are essential for the structural integrity of the skin, as they physically link the epidermal basement membrane to the underlying papillary dermis.[2] By restoring functional COL7 and promoting the formation of new anchoring fibrils, dabocemagene autoficel aims to strengthen the DEJ, improve adhesion between skin layers, prevent the recurrent blistering characteristic of DEB, facilitate the healing of chronic wounds, and enhance overall skin stability.[2]

3.3. Manufacturing Process

The manufacturing of dabocemagene autoficel is a personalized, multi-step process based on Castle Creek Biosciences' proprietary autologous fibroblast technology and a patented manufacturing protocol.[2] The process begins with the collection of small skin biopsies from the DEB patient.[2] From these biopsies, dermal fibroblast cells are isolated and separated from other cutaneous cell types. These isolated fibroblasts are then cultured and expanded in number under controlled laboratory conditions using specialized growth media.[2]

Following sufficient expansion, the patient's autologous fibroblasts are transduced with the self-inactivating (SIN) lentiviral vector carrying the therapeutic COL7A1 gene.[2] This transduction step results in the integration of the functional COL7A1 gene into the fibroblasts' DNA, enabling them to produce type VII collagen. A critical aspect of this manufacturing strategy is the creation of a personalized cell bank from a single set of biopsies. These genetically modified autologous fibroblasts are cryogenically preserved, establishing a master cell bank that can serve as a long-term repository for the individual patient's therapeutic needs.[2] This approach facilitates the potential for future redosing or treatment of new wound areas without requiring repeated biopsies and de novo manufacturing cycles from scratch.

The complexity of this ex vivo autologous gene therapy manufacturing process, involving patient-specific cell handling, viral vector production, genetic modification, cell expansion, and stringent quality control, is substantial. Such processes are inherently resource-intensive and demand specialized facilities and expertise. The termination of the initial Phase 3 trial (NCT04213261) explicitly due to "vector unavailability" highlights a critical vulnerability within this complex supply chain.[6] Viral vectors, particularly those of clinical grade, are sophisticated biological products whose consistent and scalable production can pose significant challenges. This past disruption underscores that even with a promising therapeutic rationale, ensuring a reliable and scalable supply of all components, especially the viral vector, is paramount for the successful clinical development and potential commercialization of such advanced therapies.

3.4. Administration Route

Dabocemagene autoficel is formulated for local administration and is delivered via intradermal injections.[2] The genetically modified autologous fibroblasts are injected directly into chronic, non-healing, or recurrent wounds characteristic of DEB.[2] Specifically, injections are targeted to the papillary dermis, the superficial layer of the dermis situated just beneath the epidermis.[2] This localized delivery aims to concentrate the therapeutic cells at the site of pathology, where the newly expressed COL7 protein can most effectively contribute to the formation of anchoring fibrils and restore integrity to the DEJ.[2]

The clinical trial protocols specify an injection volume of 0.25 mL per injection site, with a maximum total volume of 15 mL (corresponding to up to 60 individual injections) allowed per treatment session.[22] This suggests that multiple small-volume injections are administered to cover the affected wound areas. The therapy is designed to be redosable, a feature supported by the cryopreservation of a personalized cell bank.[2] This redosability is a potentially significant advantage for a chronic condition like DEB, where new wounds can continuously form or existing wounds may require further treatment over time. The Phase 3 clinical trial protocol for NCT04213261, for instance, included at least two main treatment sessions with D-Fi, with provisions for additional treatments at later time points for unclosed wounds.[20] This strategy contrasts with some gene therapies designed as single-administration treatments and acknowledges the persistent nature of DEB.

4. Clinical Development Program

The clinical development of dabocemagene autoficel has encompassed early-phase studies to establish initial safety and efficacy signals, followed by a pivotal Phase 3 program aimed at securing regulatory approval. The program has faced significant challenges, including the termination of its initial pivotal trial, but has since been revived with new funding and a new Phase 3 study.

Table 2: Summary of Key Clinical Trials for Dabocemagene autoficel

Trial ID (NCT #)	Phase	Official Title / Brief Description	Status (Key Dates)	Participants (Planned/Actual)	Age Range	Key Efficacy Endpoints/Aims	Brief Summary of Key Findings/Status	Reference(s)
NCT02493816	1	A Phase I Study of FCX-007 (Lenticol-F) for RDEB	Completed (Published June 2019)	4 adults (5 subjects enrolled)	≥18 years	Safety, C7 expression, AF morphology, transgene presence	Well-tolerated. Increased C7 MFI in 3/4 subjects. Transgene detected. No new mature AFs by TEM, but wisp-like structures present. One patient reported faster biopsy site healing.	13
NCT02810951	1/2	A Phase I/II Study of FCX-007 for RDEB	Terminated (April 2022, interim data Jan 2020, listed as terminated Feb 2023)	6 RDEB patients in efficacy analysis	>7 years	Safety, COL7 expression, AF presence, wound healing	Castle Creek: 80% (8/10) treated wounds ≥90% healing at 12 weeks vs 0% untreated; well-tolerated. Review: Correct C7 localization, enhanced wound healing, AF formation reported (contrasting with NCT02493816 initial AF findings).	4
NCT04213261 (DeFi-RDEB)	3	A Pivotal Phase 3 Study of FCX-007 for RDEB	Terminated (due to vector unavailability; primary completion Jan 2023; PCORI archived July 2024)	Approx. 24 evaluable (up to 40 screened)	≥2 years	Primary: Complete wound closure of First Wound Pair at Week 24. Secondary: Closure at other timepoints/wound pairs, durability.	Terminated before completion due to vector unavailability. No final efficacy data available from snippets.	6
NCT06892639	3	Evaluation of D-Fi for the Treatment of Wounds Due to DEB	Recruiting (First posted Mar 2025)	Target: 32	Not specified (DEB patients)	Wound healing (details not specified in snippets)	New Phase 3 trial initiated following new financing.	1

4.1. Early-Phase Clinical Trials (Phase 1/2)

4.1.1. NCT02810951 (FCX-007 for RDEB)

This Phase 1/2 study (NCT02810951) was designed to assess the safety of FCX-007, the expression of type VII collagen (COL7), the presence of anchoring fibrils, and clinical wound healing in patients with RDEB aged 7 years and older.[23] The study received funding support from the FDA's Office of Orphan Products Development (OOPD).[23] While some sources list the trial as "NOT RECRUITING" [23], others indicate it was "Terminated" with a publish date of February 8, 2023.[24] PCORI data further clarifies that this Phase 1/2 trial was terminated in April 2022, with interim data having been reported in January 2020.[6]

According to information released by Castle Creek Biosciences, data from six RDEB patients treated in this study showed that 80% (8 out of 10) of chronic wounds treated with D-Fi (FCX-007) achieved at least 90% healing by 12 weeks after the first injection. In contrast, none of the untreated wounds in these patients healed during the same period. The therapy was reported to be well-tolerated, with only minor and temporary adverse events such as redness or discoloration at the injection site noted.[4] A separate account mentioned that 8 out of 10 treated wounds showed at least 90% healing within approximately three months.[5]

A 2024 review by Has et al., referencing earlier publications, indicated that this trial (NCT02810951) utilized a lentiviral vector to deliver COL7A1 cDNA into autologous fibroblasts. The outcomes reported included correct localization of C7 protein at the DEJ and enhanced wound healing. Notably, this trial was reported to have demonstrated anchoring fibril formation, a finding that contrasts with some initial reports from the related NCT02493816 study.[17]

4.1.2. NCT02493816 (Lenticol-F - precursor/related study)

The NCT02493816 trial, also known as Lenticol-F, was a Phase 1, single-center, open-label study conducted in four adult RDEB patients (five subjects were enrolled, with one subject acting as a manufacturing run-in but not receiving injections due to intercurrent illness, though their cells were manufactured).[13] This study evaluated intradermal injections of autologous fibroblasts genetically modified with a codon-optimized COL7A1 cDNA using a self-inactivating lentiviral vector.[13] The primary objective was to assess the safety of this approach, including the potential for autoimmune reactions against the recombinant C7. Secondary objectives included evaluating C7 expression at the DEJ, anchoring fibril morphology, and the presence of the transgene in treated skin biopsies over a 12-month follow-up period.[13] This trial, funded by several organizations including Cure EB and DEBRA UK, is considered a foundational study for the D-Fi development program by Castle Creek Biosciences.[14]

Safety Results (Lwin et al., JCI Insight 2019): The administration of gene-modified fibroblasts was well tolerated by all four treated patients, with no serious adverse reactions (SARs) or evidence of autoimmune reactions against the newly expressed C7 protein reported.[13] Seven mild (Grade 1) adverse reactions were noted, all deemed related to the injection procedure itself (e.g., injection site erythema, pain, bruising, or itch at the biopsy site) and resolved spontaneously.[13] A total of 45 adverse events (AEs) were recorded across the subjects, categorized as mild (n=25), moderate (n=10), and severe (n=10). The most frequent AEs were related to underlying EB skin conditions (n=15). One subject experienced ten serious AEs (SAEs) requiring hospitalization, but these were attributed to pre-existing comorbidities and not to the investigational therapy. No life-threatening events or deaths occurred. Routine clinical monitoring, including vital signs, physical examinations, and laboratory blood analyses, revealed no clinically significant changes from baseline, and no malignancies or tumorous cells were observed in treated areas during the 12-month monitoring period.[13] Importantly, immunological assessments (anti-C7 IgG ELISA, C7 ELISPOT, and indirect immunofluorescence for EBA-like autoantibodies) showed no evidence of pathogenic autoimmune responses to the recombinant C7.[13]

Efficacy Results (Lwin et al., JCI Insight 2019): In terms of C7 protein expression, quantitative immunofluorescence analysis showed a statistically significant (P < 0.05) increase in C7 mean fluorescence intensity (MFI) at the DEJ in injected skin compared to noninjected skin in three of the four subjects. This increase ranged from 1.26-fold to 26.10-fold and was sustained for up to 12 months in two of the four subjects.[13] The presence of the COL7A1 transgene (the codon-optimized cDNA) was confirmed in skin biopsies from an injected site in one subject at the 12-month time point.[13] However, a critical finding from this study was that transmission electron microscopy (TEM) did not detect the formation of new, mature, fan-shaped anchoring fibrils in any of the biopsies from injected sites at any time point up to 12 months. Only wisp-like structures, interpreted as possible immature or rudimentary anchoring fibrils similar to those seen at baseline, were observed.[13] While quantitative clinical measures of wound healing were not primary endpoints due to the limited treatment area, one subject (subject 5) voluntarily reported that a biopsy site from an injected skin area healed much faster than a biopsy site from a noninjected area, an observation consistent with the significant increase in full-length C7 expression noted in her treated skin.[13]

The discrepancy regarding anchoring fibril (AF) formation between the initial report of NCT02493816 (no new mature AFs) and later summaries suggesting NCT02810951 did show AF formation is noteworthy.[14] Mature AFs are the ultimate structural evidence of functional COL7 restoration and are critical for long-term skin integrity. This apparent inconsistency could arise from various factors, including differences in the specific D-Fi product batches used, variations in patient characteristics, differing biopsy timings or analytical sensitivities, or an evolution in the interpretation of ultrastructural findings (e.g., whether "wisp-like structures" were later considered indicative of early/ongoing fibrillogenesis). Resolving this and demonstrating consistent, robust formation of mature anchoring fibrils is a crucial objective for the ongoing Phase 3 development.

4.2. Pivotal Phase 3 Clinical Trial Program

4.2.1. DeFi-RDEB Study (NCT04213261)

The DeFi-RDEB study (NCT04213261) was designed as a pivotal Phase 3, multi-center, open-label trial to evaluate FCX-007 in patients with RDEB.[8] The study employed an intra-patient randomized, controlled design, where each participant served as their own control.[8] Up to three pairs of target wounds (each 10-50 cm², or 5-50 cm² for children aged 2-6 years) were to be identified per subject; one wound in each pair was randomized to receive intradermal injections of FCX-007, while the other (control) wound received standard-of-care treatment only.[20] The trial aimed to enroll approximately 24 evaluable subjects aged 2 years or older with a confirmed clinical diagnosis of RDEB and a documented COL7A1 genetic mutation.[12]

The treatment regimen involved initial FCX-007 administrations at Day 1 and Week 12/Month 3. Provisions were made for potential re-treatment of any treated wounds that remained unclosed, and for the treatment of previously unclosed control wounds, at Week 24/Month 6 and Week 36/Month 9, at the investigator's discretion.[20] The primary efficacy endpoint was defined as complete wound closure of the first (primary) designated wound pair, assessed by the investigator at Week 24.[8] Secondary efficacy endpoints included complete wound closure of the first wound pair at Week 12, complete wound closure of all treated wound pairs at Weeks 12 and 24, and various measures of the durability of wound closure.[8] Safety and efficacy assessments were scheduled to continue through Week 48/Month 12, after which participants who received FCX-007 would enter a long-term safety follow-up period extending up to 15 years.[20] This long follow-up is typical for gene therapies involving integrating vectors to monitor for any late-emerging safety concerns. Recruitment sites for this study included prominent U.S. institutions such as Stanford University, Children's Hospital Colorado, Dell Children's Medical Group, Solutions Through Advanced Research, Inc., and Mayo Clinic.[8]

The intra-patient control design is a robust approach for rare diseases like RDEB, as it minimizes the impact of inter-patient variability in disease severity and response, thereby enhancing statistical power and allowing for meaningful conclusions with a smaller number of participants. This design is particularly well-suited for evaluating locally administered therapies.

Trial Status of NCT04213261: The trial was initially listed as "Recruiting" [25] and subsequently as "Active_not_recruiting" [24] or "NOT RECRUITING".[26] However, a critical development occurred: according to a PCORI update on July 16, 2024, the Phase 3 DeFi-RDEB trial (NCT04213261) was terminated due to unavailability of the viral vector.[6] The primary completion date had been listed as January 2023.[6] This termination represented a major setback for the D-Fi program, halting its progression towards potential regulatory submission at that time.

4.2.2. New Phase 3 Study (NCT06892639): "Evaluation of D-Fi for the Treatment of Wounds Due to DEB"

Following the termination of NCT04213261 and subsequent new financing (detailed in Section 6.2), Castle Creek Biosciences has initiated a new Phase 3 clinical trial for D-Fi, registered as NCT06892639.[1] This trial is titled "Evaluation of D-Fi for the Treatment of Wounds Due to DEB."

Key details for NCT06892639 available from the provided information include:

Sponsor: Castle Creek Biosciences, LLC.[1]
Status: Recruiting.[1]
First Posted Date: March 25, 2025; Last Posted Date: May 15, 2025.[1]
Target Enrollment: 32 participants.[1]
Intervention: Biological: D-Fi (dabocemagene autoficel).[1]
Condition: Dystrophic Epidermolysis Bullosa.[1]
Locations: Initial listed locations in the United States include Stanford University School of Medicine (Redwood City, CA), Mission Dermatology (Santa Margarita, CA), and Children's Hospital Colorado (Aurora, CO).[1]

Specifics regarding the study design, primary and secondary endpoints, and detailed treatment regimens for NCT06892639 are not extensively detailed in the currently available materials beyond its designation as a Phase 3 evaluation of D-Fi for DEB wounds. It is plausible that this new trial will incorporate learnings from the previous DeFi-RDEB study, potentially with modifications to the protocol or, crucially, with a secured and validated vector supply chain. The initiation of this new trial, backed by significant funding, signals a determined effort to advance dabocemagene autoficel towards regulatory approval despite previous obstacles. The success of this new trial will be paramount for the future of D-Fi.

5. Regulatory Landscape and Designations

5.1. U.S. Food and Drug Administration (FDA) Designations

Dabocemagene autoficel (D-Fi/FCX-007) has been granted several special designations by the U.S. FDA, reflecting its potential to address a serious and life-threatening rare disease with significant unmet medical needs. These designations are intended to facilitate and expedite the development and review process.

Table 3: FDA Regulatory Designations for Dabocemagene autoficel

Designation	Granting Body	Significance/Implication	Reference(s)
Orphan Drug Designation	FDA	For the treatment of Dystrophic Epidermolysis Bullosa (DEB). Provides incentives such as tax credits for clinical trials, exemption from user fees, and potential for 7 years of market exclusivity upon approval.	4
Rare Pediatric Disease (RPD) Designation	FDA	For the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB). If a marketing application for D-Fi is approved for RDEB, Castle Creek Biosciences may be eligible to receive a Priority Review Voucher (PRV). A PRV can be redeemed to obtain priority review for a subsequent human drug application or can be sold to another company.	4
Fast Track Designation	FDA	For the treatment of RDEB. Designed to facilitate the development and expedite the review of drugs intended to treat serious conditions and fill an unmet medical need. Allows for more frequent meetings with FDA, eligibility for Accelerated Approval and Priority Review, if relevant criteria are met, and Rolling Review of the BLA.	4
Regenerative Medicine Advanced Therapy (RMAT) Designation	FDA	For the treatment of RDEB. This designation is for cell and gene therapies intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and where preliminary clinical evidence indicates the drug has the potential to address unmet medical needs. It provides all the benefits of Fast Track and Breakthrough Therapy designations, including intensive FDA guidance on efficient development and eligibility for accelerated approval.	4

In addition to these designations, Castle Creek Biosciences was awarded an FDA Orphan Products Development (OOPD) Grant in October 2021. This grant, amounting to $1.825 million over four years, was intended to support a meaningful portion of the clinical development costs for the (now terminated) Phase 3 DeFi-RDEB study (NCT04213261).[8]

5.2. European Medicines Agency (EMA) Status

The provided research materials contain very limited information regarding the regulatory status or interactions of dabocemagene autoficel with the European Medicines Agency (EMA) or its Committee for Orphan Medicinal Products (COMP). While competitor products like beremagene geperpavec (Vyjuvek®) have received Orphan Drug Designation from the EMA and are progressing through the Marketing Authorisation Application (MAA) review process [29], similar specific milestones for D-Fi in Europe are not detailed. An SEC filing by Castle Creek Biosciences from mid-2021, related to a potential initial public offering and projecting topline data from the original Phase 3 trial in the second half of 2022, did not include information about EMA regulatory strategy or status.[19] This information gap suggests that the primary development focus for D-Fi may have been concentrated on the U.S. pathway, or that European regulatory activities were less mature or not publicly disclosed at the time the source materials were generated. For a therapy targeting an ultra-rare disease with a global patient population, clarity on the European regulatory strategy would be an important factor for future global access.

5.3. Anticipated Regulatory Pathway, Challenges, and Outlook

The array of FDA special designations awarded to dabocemagene autoficel indicates a supportive regulatory environment in the United States, acknowledging the therapy's potential to address a critical unmet need in RDEB. These designations can significantly streamline development and review timelines. However, the ultimate path to regulatory approval is critically dependent on the generation of robust and convincing safety and efficacy data from the newly initiated Phase 3 trial, NCT06892639.

The termination of the preceding Phase 3 trial, NCT04213261, due to "vector unavailability" represented a substantial setback and a significant challenge that the program had to overcome.[6] This event undoubtedly delayed the development timeline. Indeed, a PCORI record updated in July 2024 noted that "FDA Submission Planned: N/A" and that the development of D-Fi "appears to have stalled," with no new developer press releases found since August 2022.[6] This assessment, however, predates the announcement of new financing and the initiation of the new Phase 3 trial (NCT06892639) in early 2025.

There have been some potentially conflicting signals regarding the regulatory timeline. For instance, an article from June 2024 suggested that FDA approval for dabocemagene autoficel was "expected in the coming months".[31] This projection appears inconsistent with the known termination of the pivotal trial and the subsequent very recent commencement of a new Phase 3 study. Such discrepancies may arise from rapidly evolving program statuses or reliance on outdated information. The initiation of NCT06892639 effectively resets the timeline for generating the necessary pivotal data for a Biologics License Application (BLA).

One potentially favorable factor is the FDA's prior experience with gene therapies for DEB that employ a similar underlying mechanism of action, such as the approved Vyjuvek®, which also delivers the COL7A1 gene. This familiarity might contribute to a more streamlined review process for D-Fi, provided the clinical data from NCT06892639 are positive and address all safety and efficacy concerns.[32] Nevertheless, dabocemagene autoficel remains an investigational product and has not yet received marketing approval from the FDA or any other global regulatory authority.[2] The successful resolution of previous manufacturing challenges, particularly concerning consistent vector supply, will be crucial for regulatory confidence and future commercial viability.

6. Developer, Funding, and Commercial Context

6.1. Castle Creek Biosciences

Castle Creek Biosciences, Inc. is a clinical-stage (also referred to as late-stage [33]) biopharmaceutical company dedicated to developing and commercializing innovative cell and gene therapies for rare diseases with limited or no treatment options.[2] The company's core focus has been on dermatological and connective tissue disorders, leveraging its proprietary autologous fibroblast technology platform, which forms the basis for its lead candidate, dabocemagene autoficel (D-Fi).[2] Castle Creek Biosciences operates an in-house, current Good Manufacturing Practice (cGMP) compliant, commercial-scale manufacturing facility located in Exton, Pennsylvania. This facility is equipped with validated systems and processes, some of which were previously implemented for an FDA-approved cell therapy product, suggesting established manufacturing capabilities.[12]

In a strategic expansion beyond dermatology, Castle Creek acquired Novavita Thera in January 2022. This acquisition was aimed at broadening its innovative cell and gene therapy platform into in vivo gene therapies for metabolic liver diseases.[29] Through this expansion, the company is developing LV-FAH, a lentiviral vector-based therapy targeting hereditary tyrosinemia type 1 (HT1), a rare and life-threatening metabolic disorder.[33] This diversification indicates a broader ambition to become a multi-platform gene therapy company addressing various rare diseases.

6.2. Recent Funding and Collaborations

The D-Fi program received a significant financial boost in early 2025. Castle Creek Biosciences secured $75 million through a royalty financing agreement, announced in February 2025.[5] This financing was specifically structured to support the ongoing Phase 3 clinical trial of D-Fi for DEB.[5] The transaction was led by Ligand Pharmaceuticals, which invested $50 million, with the remaining $25 million contributed by a syndicate of co-investors, including Paragon Biosciences, Valor Equity Partners, and XOMA Royalty Corporation (which contributed $5 million).[5] Under the terms of the agreement, the investors are entitled to receive a high single-digit royalty on the worldwide net sales of D-Fi if the therapy is successfully commercialized.[5]

This funding event is particularly noteworthy as it occurred after the known termination of the previous Phase 3 trial (NCT04213261) due to vector supply issues. Securing substantial investment, especially through a royalty-based structure (which implies investor confidence in future revenue generation), after such a significant setback, suggests a strong belief among investors in the continued therapeutic potential of D-Fi and in Castle Creek's ability to overcome the prior manufacturing hurdles. Both XOMA Royalty and Ligand Pharmaceuticals reported this transaction in their respective Q1 2025 financial disclosures. Ligand, for instance, recorded a $44.3 million one-time research and development charge in Q1 2025 related to this royalty financing agreement.[32]

Prior to this, in October 2021, Castle Creek Biosciences had also been awarded a $1.825 million research grant from the FDA's Office of Orphan Products Development (OOPD) to support the clinical development of FCX-007 in the DeFi-RDEB study (NCT04213261).[8]

6.3. Market Outlook and Competitive Landscape

Dabocemagene autoficel is being developed for a market characterized by a high unmet medical need but also an increasingly competitive therapeutic landscape, with several novel treatments for DEB having recently gained approval or nearing regulatory submission. Understanding this context is crucial for assessing D-Fi's potential positioning.

Table 4: Overview of Selected Therapies for Dystrophic Epidermolysis Bullosa

Therapy Name (Brand/Generic)	Developer	Mechanism of Action/Therapy Type	Administration	Regulatory Status (US/EU) & Key Dates	Key Distinguishing Features/Reported Efficacy Highlights	Reference(s)
Dabocemagene autoficel (D-Fi, FCX-007)	Castle Creek Biosciences	Ex vivo autologous fibroblast gene therapy (lentiviral vector delivering COL7A1)	Intradermal injection into wounds; redosable	Investigational (Phase 3 - NCT06892639 recruiting); Multiple FDA designations. No EMA approval.	Personalized cell bank for long-term use. Phase 1/2: 80% of treated wounds ≥90% healing at 12 weeks.	1
Beremagene geperpavec-svdt (Vyjuvek®)	Krystal Biotech	Topical, non-replicating HSV-1 vector-based gene therapy delivering two copies of COL7A1	Topical gel applied to wounds by HCP (can be in home setting); redosable weekly	FDA Approved (May 2023) for DEB (≥6 months). EMA MAA under review; Orphan & PRIME designations.	First topical, redosable gene therapy for DEB. Clinical trials: 67% wounds complete healing vs 22% placebo.	9
Prademagene zamikeracel (pz-cel / EB-101 / Zevaskyn™)	Abeona Therapeutics	Autologous, COL7A1 gene-corrected epidermal sheets (keratinocytes transduced with retroviral vector)	Surgical grafting of cell sheets onto wounds; intended as one-time application per wound	FDA Approved (Zevaskyn, May 2025). BLA submitted Q3 2023.	Cell-based sheets. Long-term data suggests sustained wound healing up to 8 years.	9
Birch triterpenes topical gel (Filsuvez®)	Amryt Pharma (acquired by Chiesi Farmaceutici)	Botanical product (birch bark extract)	Topical gel applied to wound surfaces at each dressing change (every 1-4 days)	FDA Approved (Dec 2023) for JEB & DEB (≥6 months). EMA Approved (June 2022).	Botanical, non-gene therapy. Clinical trials: 41.3% patients achieved wound closure within 45 days vs 28.9% placebo.	6
Allo-APZ2-OTS	Rheacell	Allogeneic ABCB5+ mesenchymal stromal cells (MSCs)	Intravenous infusion	Investigational (Phase 3 for RDEB)	Systemic cell therapy with immunomodulatory and anti-inflammatory properties.	9
PTR-01	BridgeBio Pharma (previously PellePharm)	Topical recombinant human Collagen Type VII	Topical application	Investigational (Phase 2 completed for RDEB)	Protein replacement therapy.	9

Dabocemagene autoficel's approach, involving intradermally injected, genetically modified autologous fibroblasts, offers a distinct modality compared to the topical gel-based gene therapy (Vyjuvek®), the surgically applied cell sheets (Zevaskyn™/pz-cel), or the botanical topical gel (Filsuvez®). The potential for D-Fi to be redosed from a personalized cell bank could be an advantage in managing the chronic and recurrent nature of DEB wounds.[2]

The market for DEB therapies, while addressing an ultra-rare condition, is valued significantly due to the severity of the disease and the high cost associated with advanced therapies. Ligand Pharmaceuticals has estimated that D-Fi could have a market potential exceeding $1 billion worldwide.[32] Interestingly, Ligand management has also suggested that D-Fi and Vyjuvek® might be complementary and could potentially be used in combination, which could expand the therapeutic options rather than leading to direct displacement.[32] The FDA's familiarity with the COL7A1 gene as a target, following the review and approval of Vyjuvek®, might also provide a somewhat smoother registrational pathway for D-Fi, assuming positive clinical outcomes from the new Phase 3 trial.[32]

The success of D-Fi will ultimately depend on its ability to demonstrate a compelling clinical profile, including robust and durable wound healing, a favorable safety record, improvements in patient-reported outcomes (such as pain and itch), and potentially a convenient administration regimen compared to or in complement with existing and emerging alternatives.

7. Discussion and Future Perspectives

The development trajectory of dabocemagene autoficel offers a compelling case study in the promise and perils of pioneering advanced gene therapies for rare genetic disorders. Initial Phase 1/2 clinical investigations (NCT02810951 and the related NCT02493816) provided encouraging preliminary evidence of biological activity, including increased expression of type VII collagen (COL7) at the dermal-epidermal junction (DEJ) and positive signals for wound healing in patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB).[4] However, these early studies also highlighted areas requiring further clarification, particularly regarding the consistent formation of mature, functional anchoring fibrils—the ultimate hallmark of structural correction in DEB. While one review suggested anchoring fibril formation was observed in NCT02810951 [17], the detailed publication from the earlier NCT02493816 trial reported the absence of new mature anchoring fibrils, although increased C7 protein was present.[13] This discrepancy underscores the importance of rigorous, standardized assessment of such critical biological endpoints in later-stage trials.

The D-Fi program encountered a formidable obstacle with the termination of its pivotal Phase 3 DeFi-RDEB study (NCT04213261) due to "vector unavailability".[6] This event, occurring after significant investment and patient enrollment, served as a stark illustration of the vulnerabilities inherent in the complex manufacturing and supply chains for gene therapy components, particularly viral vectors. Such a disruption can have profound implications for development timelines, costs, and investor confidence. The period following this termination saw the program's momentum stall, as noted by PCORI in mid-2024.[6]

Remarkably, the program has since demonstrated significant resilience. The infusion of $75 million in new royalty financing in early 2025, specifically to support a renewed Phase 3 effort, and the subsequent initiation of a new Phase 3 trial (NCT06892639) in March 2025, signify a strong continued belief in the therapeutic hypothesis underpinning dabocemagene autoficel.[1] This revival suggests that the preclinical and early clinical data were compelling enough to attract substantial new investment, contingent upon addressing the critical vector supply issues. It is imperative that Castle Creek Biosciences has implemented a robust and reliable vector manufacturing and sourcing strategy to prevent recurrence of such disruptions, as this will be a key factor for the success of the new trial and any future commercialization efforts.

Dabocemagene autoficel is poised to enter a dynamic and evolving therapeutic landscape for DEB. The recent FDA approvals of Vyjuvek® (beremagene geperpavec), a topical gene therapy, and Filsuvez®, a botanical topical gel, along with the approval of Zevaskyn™ (prademagene zamikeracel), an autologous cell sheet therapy, mean that D-Fi will need to clearly differentiate itself.[9] Potential differentiating factors for D-Fi could include its mode of administration (intradermal injection), the specific cell type used (autologous dermal fibroblasts), the creation of a personalized cell bank allowing for redosing without repeated biopsies [2], and potentially its efficacy or durability profile in specific wound types or patient populations. The suggestion that D-Fi might be used in combination with other therapies like Vyjuvek® also opens avenues for a complementary role rather than purely direct competition.[32]

Several critical questions remain for dabocemagene autoficel. The foremost is whether the new Phase 3 trial (NCT06892639) will definitively demonstrate statistically significant and clinically meaningful efficacy, particularly in terms of durable wound closure and, crucially, consistent evidence of mature anchoring fibril formation. The long-term safety profile, especially concerning the use of an integrating lentiviral vector, will also be under scrutiny. While SIN lentiviral vectors are designed to minimize risks of insertional oncogenesis, extended follow-up (as planned with the 15-year monitoring in the original Phase 3 protocol [20]) is essential to confirm long-term safety. This will be particularly relevant as it is compared against therapies using different vector systems (e.g., the generally non-integrating HSV-1 vector in Vyjuvek®) or non-viral approaches.

Furthermore, as the treatment paradigm for DEB matures, the definition of "success" is likely to expand beyond simple wound closure. Therapeutic impact on patient-reported outcomes such as pain, itch, overall quality of life, functional abilities (e.g., hand function, mobility), and potentially the incidence of complications like SCC, will become increasingly important differentiators. Future research should prioritize comprehensive data collection on these broader outcomes. Comparative effectiveness research, if feasible in such a rare disease, would be invaluable, although challenging to conduct. Continued investigation into the molecular and cellular mechanisms underlying the response to D-Fi, particularly regarding the quality and longevity of skin repair, will also be important.

8. Conclusion

Dabocemagene autoficel (D-Fi/FCX-007) represents a sophisticated, personalized gene therapy approach with the potential to address the underlying genetic defect in Dystrophic Epidermolysis Bullosa by restoring functional type VII collagen. Early clinical studies have provided encouraging signals of biological activity and wound healing. However, the program has navigated a challenging development path, marked by a significant setback with the termination of its initial pivotal Phase 3 trial due to vector manufacturing issues.

The recent infusion of substantial new financing in early 2025 and the prompt initiation of a new Phase 3 clinical trial (NCT06892639) demonstrate renewed commitment and investor confidence in the therapy's potential. This revival underscores the perceived value of D-Fi's therapeutic concept, provided that manufacturing and supply chain vulnerabilities have been definitively resolved.

The ultimate success of dabocemagene autoficel hinges on several critical factors: the unambiguous demonstration of robust and durable efficacy in the ongoing Phase 3 trial, particularly concerning complete wound closure and the formation of functional anchoring fibrils; a continued favorable safety profile over the long term, especially given its nature as an integrating lentiviral vector-based therapy; and the establishment of a reliable and scalable manufacturing process.

As dabocemagene autoficel progresses, it enters an increasingly competitive therapeutic landscape for DEB. Its differentiation and value proposition will be determined by its overall clinical performance relative to existing and other emerging treatments, considering not only wound healing but also broader impacts on patient quality of life, long-term durability, and safety. The journey of D-Fi highlights both the transformative potential of gene therapy for severe rare diseases and the complex scientific, manufacturing, and regulatory hurdles that must be overcome to bring such innovations to patients.

9. References

[User Query]

Works cited

Dabocemagene autoficel | MedPath, accessed May 28, 2025, https://trial.medpath.com/drug/e15e9ca8c1616617/dabocemagene-autoficel
Castle Creek: Re-Dosable Gene Therapies for Localized Treatment ..., accessed May 28, 2025, https://castlecreekbio.com/technology/autologous-fibroblast-technology/
Dabocemagene autoficel: Uses, Interactions, Mechanism of Action | DrugBank Online, accessed May 28, 2025, https://go.drugbank.com/drugs/DB17878
D-Fi Gene Therapy for Dystrophic Epidermolysis Bullosa (DEB) - Castle Creek Biosciences, accessed May 28, 2025, https://castlecreekbio.com/pipeline/gene-therapy-dystrophic-epidermolysis-bullosa/
Castle Creek gets $75M for DEB gene therapy Phase 3 trial, accessed May 28, 2025, https://epidermolysisbullosanews.com/news/castle-creek-gets-75m-deb-gene-therapy-phase-3-trial/
Dabocemagene autoficel (D-Fi) to treat recessive dystrophic epidermolysis bullosa - Horizon Scanning Database, accessed May 28, 2025, https://horizonscandb.pcori.org/report/topics/185
Ligand Pharmaceuticals Leads $75M Royalty Financing for D-Fi Gene Therapy in Rare Skin Disease Trial - MedPath, accessed May 28, 2025, https://trial.medpath.com/news/0253eecee851bd33/ligand-pharmaceuticals-leads-75m-royalty-financing-for-d-fi-gene-therapy-in-rare-skin-disease-trial
FDA Grant Supports Recessive Dystrophic Epidermolysis Bullosa ..., accessed May 28, 2025, https://www.cgtlive.com/view/fda-grant-supports-recessive-dystrophic-epidermolysis-bullosa-gene-therapy-study
High-Cost Therapy Profile: April - Prime Therapeutics - Portal, accessed May 28, 2025, https://www.primetherapeutics.com/high-cost-therapy-profile-april
Epidermolysis Bullosa Treatment Market Size, Drugs, Companies - DelveInsight, accessed May 28, 2025, https://www.delveinsight.com/report-store/epidermolysis-bullosa-market
Statistical Analysis Plan - Clinical Trials, accessed May 28, 2025, https://cdn.clinicaltrials.gov/large-docs/61/NCT04213261/SAP_003.pdf
Castle Creek Biosciences Awarded FDA Orphan Products Development Grant to Support DeFi-RDEB, a Pivotal Phase 3 Study of FCX-007 Investigational Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa - PR Newswire, accessed May 28, 2025, https://www.prnewswire.com/news-releases/castle-creek-biosciences-awarded-fda-orphan-products-development-grant-to-support-defi-rdeb-a-pivotal-phase-3-study-of-fcx-007-investigational-gene-therapy-for-recessive-dystrophic-epidermolysis-bullosa-301405636.html
Safety and early efficacy outcomes for lentiviral ... - JCI Insight, accessed May 28, 2025, https://insight.jci.org/articles/view/126243
Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa - JCI Insight, accessed May 28, 2025, https://insight.jci.org/articles/view/126243/figure/1
Dabocemagene autoficel API Price per kg: B2B transaction history - Pharmaoffer.com, accessed May 28, 2025, https://pharmaoffer.com/api-excipient-supplier/transactions/dabocemagene-autoficel
ZEVASKYN Approval Positions Abeona as Emerging Player in ..., accessed May 28, 2025, https://www.prnewswire.com/news-releases/zevaskyn-approval-positions-abeona-as-emerging-player-in-dystrophic-epidermolysis-bullosa-market--delveinsight-302462822.html
Treatment of Epidermolysis Bullosa and Future Directions: A Review - PMC, accessed May 28, 2025, https://pmc.ncbi.nlm.nih.gov/articles/PMC11333680/
Treatment of Epidermolysis Bullosa and Future Directions: A Review | springermedizin.de, accessed May 28, 2025, https://www.springermedizin.de/treatment-of-epidermolysis-bullosa-and-future-directions-a-revie/27423484
castle creek biosciences, inc. - SEC.gov, accessed May 28, 2025, https://www.sec.gov/Archives/edgar/data/1756702/000119312521217390/d167954ds1.htm
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa, accessed May 28, 2025, https://ctv.veeva.com/study/a-study-of-fcx-007-for-recessive-dystrophic-epidermolysis-bullosa
vyjuvek-ndfb.docx, accessed May 28, 2025, https://dss.mo.gov/mhd/cs/advisory/rdac/doc/vyjuvek-ndfb.docx
B531A477-FD63-4932-ADBF-CAF955375D11 - FCX-007/US IND# 16582 - ClinicalTrials.gov, accessed May 28, 2025, https://cdn.clinicaltrials.gov/large-docs/61/NCT04213261/Prot_002.pdf
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa ..., accessed May 28, 2025, https://stanfordhealthcare.org/trials/a/NCT02810951.html
Anna L. Bruckner | Dermatology | Aurora, CO - MediFind, accessed May 28, 2025, https://www.medifind.com/doctors/anna-l-bruckner/12938945
FCX-007 (dabocemagene autoficel ( DrugBank - DDrare: Database ..., accessed May 28, 2025, [https://ddrare.nibn.go.jp/ddrare_Mar2023/ddrare_Mar2022/cgi-bin/drug_who_e.cgi?query=FCX-007%20%28dabocemagene%20autoficel&disease_id=36](https://www.google.com/url?q=https://ddrare.nibn.go.jp/ddrare_Mar2023/ddrare_Mar2022/cgi-bin/drug_who_e.cgi?query%3DFCX-007%2B(dabocemagene%2Bautoficel%26disease_id%3D36&sa=D&source=editors&ust=1748434761136070&usg=AOvVaw1YAvdxhJK3BvjtljX7CDG6)
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa, accessed May 28, 2025, https://stanfordhealthcare.org/trials/a/NCT04213261.html
Recessive Dystrophic Epidermolysis Bullosa (DBCOND0064204) | DrugBank Online, accessed May 28, 2025, https://go.drugbank.com/conditions/DBCOND0064204
Castle Creek Biosciences Awarded FDA Orphan Products Development Grant to Support DeFi-RDEB, a Pivotal Phase 3 Study of FCX-007 Investigational Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa, accessed May 28, 2025, https://castlecreekbio.com/castle-creek-biosciences-awarded-fda-orphan-products-development-grant-to-support-defi-rdeb-a-pivotal-phase-3-study-of-fcx-007-investigational-gene-therapy-for-recessive-dystrophic-epidermolysis-bull/
Krystal's Dystrophic Epidermolysis Bullosa Gene Therapy B-VEC Under Review in EU, accessed May 28, 2025, https://www.cgtlive.com/view/krystal-dystrophic-epidermolysis-bullosa-gene-therapy-b-vec-under-review-eu
Understanding the ABC of Epidermolysis Bullosa Treatment - Roots Analysis, accessed May 28, 2025, https://www.rootsanalysis.com/key-insights/abc-of-epidermolysis-bullosa-treatment.html
Gene Therapy: From science fiction to medical reality | RGA, accessed May 28, 2025, https://www.rgare.com/knowledge-center/article/gene-therapy--from-science-fiction-to-medical-reality
s27.q4cdn.com, accessed May 28, 2025, https://s27.q4cdn.com/906368049/files/News/2025/Zacks_SCR_Research_05122025_LGND_Vandermosten.pdf
Top 10 Emerging East Coast Biotech Companies to Watch in 2025 - TFS HealthScience, accessed May 28, 2025, https://tfscro.com/resources/the-emerging-east-10-biotechs-powering-the-next-wave-of-innovation/
XOMA Royalty Reports First Quarter 2025 Financial Results and ..., accessed May 28, 2025, https://investors.xoma.com/news-events/press-releases/detail/474/xoma-royalty-reports-first-quarter-2025-financial-results
Ligand Reports First Quarter 2025 Financial Results, accessed May 28, 2025, https://investor.ligand.com/news-and-events/press-releases/news-details/2025/Ligand-Reports-First-Quarter-2025-Financial-Results/default.aspx
The Rise of Gene Therapies in Epidermolysis Bullosa Treatment - DelveInsight, accessed May 28, 2025, https://www.delveinsight.com/blog/gene-therapies-for-epidermolysis-bullosa-treatment
Our Current Clinical Trials - Castle Creek Biosciences, accessed May 28, 2025, https://castlecreekbio.com/pipeline/clinical-trials/
Injected Gene Therapy for RDEB | Dermatology - Stanford Medicine, accessed May 28, 2025, https://med.stanford.edu/dermatology/research/research_1/currentstudies/fibrocell.html
Drug and biologic pipeline update Q2 2024 - CarelonRx, accessed May 28, 2025, https://www.carelonrx.com/content/dam/digital/carelon/crx-assets/documents/Q2-2024-drug-pipeline-update.pdf
FCX-013 - Synapse - Global Drug Intelligence Database, accessed May 28, 2025, https://synapse.patsnap.com/drug/42b489e547f74c4e87481e27a159459f
Toward Combined Cell and Gene Therapy for Genodermatoses - PMC - PubMed Central, accessed May 28, 2025, https://pmc.ncbi.nlm.nih.gov/articles/PMC7197428/
Development of Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa - UCL Discovery, accessed May 28, 2025, https://discovery.ucl.ac.uk/1477579/1/C.Georgiadis%20thesis%2006.04.16%20Library%20version.pdf
dabocemagene autoficel (FCX-007) / Precigen, Paragon Biosci - LARVOL DELTA, accessed May 28, 2025, https://delta.larvol.com/Products/?ProductId=ebda7760-4a79-4fe4-a65e-9fdaafef4d7c
Full article: Industry Updates from the Field of Stem Cell Research and Regenerative Medicine in September 2019, accessed May 28, 2025, https://www.tandfonline.com/doi/full/10.2217/rme-2019-0131
XOMA Royalty Reports First Quarter 2025 Financial Results and Highlights Business Achievements | XOMAP Stock News, accessed May 28, 2025, https://www.stocktitan.net/news/XOMAP/xoma-royalty-reports-first-quarter-2025-financial-results-and-ivv7p4qidvjt.html
Clindamycin Phosphate - Drug Targets, Indications, Patents - Patsnap Synapse, accessed May 28, 2025, https://synapse.patsnap.com/drug/9a2068e154dd4300afceec3ec9a6df88
Ligand Reports First Quarter 2025 Financial Results - GlobeNewswire, accessed May 28, 2025, https://www.globenewswire.com/news-release/2025/05/08/3077051/0/en/Ligand-Reports-First-Quarter-2025-Financial-Results.html
Zacks Small-Cap Research, accessed May 28, 2025, https://s27.q4cdn.com/906368049/files/News/2023/Zacks_SCR_Research_04182023_ABEO_Bautz.pdf
Current Status of Biomedical Products for Gene and Cell Therapy of Recessive Dystrophic Epidermolysis Bullosa - MDPI, accessed May 28, 2025, https://www.mdpi.com/1422-0067/25/19/10270
Ligand Reports First Quarter 2025 Financial Results - BioSpace, accessed May 28, 2025, https://www.biospace.com/press-releases/ligand-reports-first-quarter-2025-financial-results

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