Alex Philippidis Senior News Editor Genetic Engineering & Biotechnology News

Investment, Incentives, and IP Drive Growth in Clinical Development

Gene therapy’s numbers look promising. In a year full of biopharma mergers and acquisitions, one of the biggest deals has been Novartis’ planned $8.7 billion purchase of AveXis, whose lead candidate AVXS-101 has advanced into a pivotal trial following positive Phase I data published in November in The New England Journal of Medicine.

Additional figures furnished to GEN by Informa show the extent of gene-therapy development: Informa Pharma Intelligence’s Trialtrove database records 729 gene therapies as having been developed, of which nearly two-thirds (461) were preclinical. Those therapies have been assessed in 1,855 clinical trials, most in early phases: 657 in Phase I, 509 in Phase I/II, and 455 in Phase II. As for later development, 89 have reached Phase III, 32 are in Phase II/III, and 28 in Phase IV.

By far the most crowded therapeutic area is oncology, accounting for two-thirds or 1,254 of the 1,855 trials. The next-largest indication is cardiovascular with 214 trials (11.5%), followed by infectious disease (6.5%).

Clinical activity is expected to increase, in part due to the FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation, created through the 21st Century Cures Act. “There are a lot of incentives for sponsors who get RMAT, including early and frequent interactions with the FDA, as well as the ability to discuss early on any potential surrogate or intermediate endpoints in their clinical trials,” Amanda Micklus, principal analyst with Informa Pharma Intelligence, told GEN. “The RMAT designation potentially could really advance the progress of these therapies through the pipeline.”

Patricia Reilly, Informa Pharma Intelligence vice president, Intelligence Alliances and Product Unification, highlighted another sign of growth: The number of gene-therapy developers has ballooned from the 69 counted in 2014 by the Alliance for Regenerative Medicine (ARM), to 255 today.

Below is a list of 25 “up and coming” gene-therapy candidates that had reached Phase III and/or registrational trials as of the first quarter of 2018, based on figures from ARM, as well as and/or company announcements. Each candidate is listed by name, sponsor(s), indication(s), mechanism, late-stage trials, and updates.

Two gene-therapy candidates on ARM’s list not included here are TransVax™, whose co-developer Vical eliminated more than half its staff and halted development after it failed the Phase III HELIOS trial. The other is Amgen’s Imlygic® (talimogene laherparepvec), which is in trials for additional melanoma indications (NCT02263508 and NCT02211131) following initial approval in 2015.

Axalimogene filolisbac (ADXS11-001) and ADXS-DUAL

Sponsor(s): Advaxis

Indication: Metastatic cervical cancer

Mechanism: Targeted immunotherapy based on a platform technology that uses live attenuated Listeria monocytogenes (Lm) bioengineered to secrete antigen/adjuvant fusion proteins. ADXS-DUAL is second-generation of axalimogene filolisbac.

Trial(s) (Identifier): Study of ADXS11-001 in Subjects with High Risk Locally Advanced Cervical Cancer (AIM2CERV; NCT02853604; Phase III)

Updates: Advaxis is assessing axalimogene filolisbac in combinations with Bristol-Myers Squibb’s Opdivo® (nivolumab) and AstraZeneca’s Imfinzi® (durvalumab). On March 12, Advaxis acknowledged the FDA’s clinical hold on a Phase I/II study of axalimogene filolisbac/Imfinzi after a patient died in February.

AMG0101 (AMG0103)

Sponsor(s): AnGes, Shionogi (global license for dermal diseases), MEDRx, and Alfresa Pharma

Indication(s): Atopic Dermatitis [Japan]; Chronic discogenic low back pain (DLBP) [U.S.]

Mechanism: NF-kB Decoy Oligonucleotide, a specific inhibitor for NF-kB that acts as a switch to a gene cluster involved in the immune inflammatory response in the body. Administered via ointment in atopic dermatitis, and via injection in discogenic low back pain.

Trial(s) (Identifier): Phase III study in atopic dermatitis (details unavailable); AMG0103 in Subjects with Chronic Discogenic Lumbar Back Pain (NCT03263611; Phase Ib)

Updates: As of April 12, AnGes stated, the Phase III atopic dermatitis study was completed, and failed. On February 28, AnGes said the first patient was dosed in the Phase Ib study.


Sponsor(s): uniQure

Indication(s): Hemophilia B

Mechanism: AMT-061 consists of an AAV5 viral vector carrying a gene cassette with the Padua variant of Factor IX (FIX-Padua).

Trial(s) (Identifiers): Dose Confirmation Trial of AAV5-hFIXco-Padua (NCT03489291; Phase II)

Updates: Phase II study not yet recruiting patients. uniQure announced plans to advance AMT-061 into a pivotal study on October 19, 2017.


Sponsor(s): AveXis (to be acquired by Novartis)

Indication(s): Spinal muscular atrophy (SMA)

Mechanism: A non-replicating adeno-associated virus 9 (AAV9) capsid is used to deliver a functional copy of a human SMN gene to the nucleus of the patient's own cells. The gene is meant to supplement the cell's production of the SMN protein, increasing that production to an adequate level.

Trial(s) (Identifiers): Pre-Symptomatic Study of Intravenous AVXS-101 in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 (SPRINT; NCT03505099; Phase III); Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 (STRIVE-EU; NCT03461289; Phase III); Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 (STR1VE; NCT03306277; Phase III)

Updates: On April 25, AveXis said the first patient was dosed in the Phase III SPRINT trial. A day earlier, AveXis released initial results from the STR1VE trial showing that all six patients were alive and event-free as of April 11—plus 24-month follow-up data from a Phase I trial of AVXS-101 in SMA Type 1 showing that all 15 patients were alive and without need for permanent ventilation.


Sponsor(s): Cold Genesys

Indication(s): Non-muscle invasive bladder cancer (NMIBC); muscle invasive bladder cancer (MIBC)

Mechanism: Oncolytic immunotherapy based on a modified common cold adenovirus backbone that contains a cancer-specific promoter and a GM-CSF transgene. CG0070 is designed to replicate inside the tumor cells, causing tumor cell lysis and immunogenic cell death.

Trial(s) (Identifier): Safety and Efficacy of CG0070 Oncolytic Virus Regimen for High Grade NMIBC After BCG Failure (BOND2; NCT02365818; Phase II)

Updates: Cold Genesys last year reported topline results from BOND2 that included a six-month Complete Response rate of 46.7%.


Sponsor(s): Marsala Biotech, Guangzhou Dabo Biological Products

Indication(s): Squamous cell carcinoma of the head and neck

Mechanism: E10A uses the Endostatin gene to prevent angiogenesis and shut off the nutrient supply to tumors, starving the tumors and leading to tumor shrinkage. The gene is integrated into an adenoviral vector which is injected into the tumor. The vector infects the tumor cells, delivering the endostatin gene inside the cell.

Trial(s) (Identifier): E10A for the Treatment of Squamous Cell Carcinoma of the Head and Neck (NCT02630264; Phase III)

Updates: NCT02630264 status is unknown.

Generx® (alferminogene tadenovec, Ad5FGF-4)

Sponsor(s): Angionetics, Huapont Life Sciences, Gene Biotherapeutics (formerly Taxus Cardium Pharmaceuticals Group)

Indication(s): Angiogenic growth factor gene therapy for cardiac microvascular insufficiency in patients with myocardial ischemia and symptomatic chronic stable angina pectoris due to coronary microvascular dysfunction in association with advanced coronary artery disease.

Mechanism: Generx consists of the human fibroblast growth factor-4 (FGF-4) gene, a CMV promoter sequence, and a signal peptide, encapsulated in a human serotype 5 adenovirus. Generx is delivered to the heart using Angionetic’s optimized cardiac catheter technique, binds to heart cells via the coxsackie adenovirus receptors, and transfects the cells with the FGF-4 gene.

Trial(s) (Identifier): Ad5FGF-4 in Patients with Refractory Angina Due to Myocardial Ischemia (AFFIRM; NCT02928094; Phase III)

Updates: NCT02928094 active but not yet recruiting patients as of January 10.


Sponsor(s): GlaxoSmithKline (GSK) and Ospedale (Hospital) San Raffaele–Telethon Institute for Gene Therapy (OSR-TIGET)

Indication(s): Metachromatic Leukodystrophy (MLD)

Mechanism: Autologous cluster of differentiation 34+ (CD34+) cells transduced with lentiviral vector containing human arylsulfatase A (ARSA) complementary deoxyribonucleic acid (cDNA)

Trial(s) (Identifier): A Safety and Efficacy Study of Cryopreserved GSK2696274 for Treatment of Metachromatic Leukodystrophy (MLD; NCT03392987; Phase III)

Updates: On April 12, GSK said it will hand off its rare disease gene therapy portfolio to Orchard Therapeutics in return for a 19.9% stake in the acquirer, undisclosed milestone payments, royalties, and a seat on Orchard’s board. The portfolio includes GSK269274, another late-stage clinical candidate GSK269275, and the European-approved gene therapy Strimvelis™.


Sponsor(s): GlaxoSmithKline (GSK) and Ospedale (Hospital) San Raffaele-Telethon Institute for Gene Therapy (OSR-TIGET)

Indication(s): Wiskott Aldrich syndrome (WAS)

Mechanism: Genetically modified autologous hematopoietic stem cells that were collected from bone marrow and/or mobilized from peripheral blood and transduced with a lentiviral vector encoding for WAS.

Trial(s) (Identifier): Gene Therapy for Wiskott-Aldrich Syndrome (TIGET-WAS; NCT01515462; Phase II)

Updates: On April 12, GSK said it will hand off its rare disease gene therapy portfolio to Orchard Therapeutics in return for a 19.9% stake in the acquirer, undisclosed milestone payments, royalties, and a seat on Orchard’s board. The portfolio includes GSK269275, GSK269274, and the European-approved gene therapy Strimvelis™.


Sponsor(s): GenSight Biologics

Indication(s): Leber Hereditary Optic Neuropathy (LHON) caused by mutation of the ND4 gene

Mechanism: AAV2 gene-therapy vector that encodes the human wild-type ND4 protein

Trial(s) (Identifier): Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation (RESCUE, NCT02652767; Phase III); Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE, NCT02652780); Efficacy and Safety Study of Bilateral Intravitreal Injection of GS010 for the Treatment of Vision Loss up to 1 Year from Onset in LHON Due to the ND4 Mutation (REFLECT, NCT03293524; Phase III).

Updates: On April 2, GenSight said the Phase III REVERSE trial missed its primary endpoint, as the 48-weeks improvement of +11 ETDRS letters in GS010-treated eyes was similar to that of untreated eyes across the study’s 37 subjects.


Sponsor(s): bluebird bio

Indication(s): Cerebral adrenoleukodystrophy (CALD)

Mechanism: Transplantation with a patient’s own stem cells modified to contain a functioning copy of the ABCD1 gene, which is designed to result in production of functional adrenoleukodystrophy protein (ALDP).

Trial(s) (Identifier): A Phase II/III Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (Starbeam or ALD-102, NCT01896102; Phase II/III); Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product (NCT02698579).

Updates: bluebird bio said February 21 it plans to file for European approval of Lenti-D in CALD in 2019. The company in October published interim data in the New England Journal of Medicine from the Starbeam study showing that as of the April 25, 2017, data cutoff, 15 of 17 patients (88%) infused with Lenti-D remained alive and free of major functional disabilities after 24 months.

LentiGlobin™ (BB305)

Sponsor(s): bluebird bio

Indication(s): Transfusion-dependent β-thalassemia (TDT, also known as β-thalassemia major), severe sickle cell disease.

Mechanism: Insertion of a functional human β-globin gene into a patient’s own hematopoietic stem cells ex vivo, followed by autologous stem cell transplantation.

Trial(s) (Identifier): A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype (Northstar-2 or HGB-207, NCT02906202; Phase III); A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia, Who Have a β0/β0 Genotype (Northstar-3 or HGB-212, NCT03207009; Phase III).

Updates: On April 18, bluebird bio published interim data in the New England Journal of Medicine from Phase I/II clinical studies assessing LentiGlobin in TDT. Most of the studies’ 22 patients remained transfusion-free after two years. On February 21, bluebird said it plans to file for European approval of LentiGlobin in TDT and non-β0/β0 genotypes in the second half of 2018.


Sponsor(s): Lysogene

Indication(s): Mucopolysaccharidosis IIIA (MPS IIIA), also known as Sanfilippo type A syndrome

Mechanism: AAVrh10 virus containing healthy copy of N-sulfoglucosamine sulfohydrolase (SGSH) gene administered directly to the brain via injection, designed to stimulate production of enzyme that breaks down heparan sulfate, slowing down or halting progression of the disease.

Trial(s) (Identifier): Planned Phase II/III pivotal trial (not listed on at deadline); International Pivotal Observational Study (SAMOS)

Updates: Lysogene said April 16 it plans to start a pivotal Phase II/III trial of LYS-SAF302 in the second half of 2018, “a change compared to initial expectations due to a manufacturing delay.”

Nadofaragene firadenovec/Sny3 (rAd-IFN/Syn3, Instiladrin®)

Sponsor(s): FKD Therapies, Ferring Pharmaceuticals

Indication(s): Bacillus Calmette-Guérin (BCG)-unresponsive nonmuscle invasive bladder cancer (NMIBC)

Mechanism: Non-replicating recombinant adenovirus type 5 (Ad5)-vector encoding the interferon alpha-2b (IFNα2b) gene, combined with the excipient Syn3 (rAD-IFN/Syn3). It infects nearby tumor cells and expresses INF alpha-2b intracellularly which activates the transcription and translation of genes whose products mediate antiviral, antiproliferative, antitumor, and immune-modulating effects.

Trials(s) (Identifier): A Study to Evaluate Instiladrin® in Patients with High-Grade, Bacillus Calmette-Guerin (BCG) Unresponsive NMIBC (NCT02773849; Phase III)

Updates: On May 3, Ferring Pharmaceuticals acquired an option to secure global commercialization rights to Nadofaragene firadenovec/Syn3 from FKD.


Sponsor(s): OcuNexus Therapeutics

Indication(s): Persistent epithelial defects that are non-responsive to current standard of care treatment

Mechanism: Natural, unmodified oligonucleotide (30-mer) that downregulates expression of the key gap junction hemichannel protein Connexin43 (Cx43). Nexagon is formulated in a thermoreversible gel placed under a contact lens or amniotic membrane to ensure contact with the corneal/conjunctival surface for sufficient time to get the active drug into the cell. 

Trial(s) (Identifier): Details not available

Updates: Nexagon is in Phase III development, according to the company and a study published online last year. OcuNexus has disclosed discussions with the FDA aimed at finalizing a clinical development plan to advance Nexagon into a pivotal trial.


Sponsor(s): Nightstar Therapeutics

Indication(s): Choroideremia

Mechanism: AAV2 vector containing recombinant human complementary DNA (cDNA) that is designed to produce REP1 inside the eye. NSR-REP1 is administered surgically by injection into the sub-retinal space.

Trial(s) (Identifier): A Safety Study of Retinal Gene Therapy for Choroideremia (GEMINI; NCT03507686); fficacy and Safety of AAV2-REP1 for the Treatment of Choroideremia (STAR; NCT03496012)

Updates: Nightstar said April 3 that it expects the STAR trial will be fully enrolled by the first half of 2019.

OXB-301 (TroVax®; MVA-5T4)

Sponsor(s): Oxford BioMedica

Indication(s): Ovarian cancer, colorectal cancer

Mechanism: Cancer vaccine containing an attenuated modified vaccinia virus Ankara (MVA) as active ingredient. OXB-301 is engineered to deliver the 5T4 oncofetal antigen gene and is designed to destroy cancerous cells by stimulating the immune system.

Trial(s) (Identifier): The Activity of TroVax® Versus Placebo in Relapsed Asymptomatic Ovarian Cancer (TRIOC; NCT01556841; Phase II)

Updates: Last year, Oxford Biomedica announced “encouraging results” from a Phase I/II trial of TroVax and low-dose cyclophosphamide in advanced colorectal cancer, with “significant anti-5T4 immune responses” generated at treatment day 43.

Pexa-Vec (Pexastimogene devacirepvec; JX-594)

Sponsor(s): SillaJen, Transgene, Beijing Shenogen Pharma Group, Lee's Pharmaceutical, GC Pharma

Indication(s): Hepatocellular carcinoma

Mechanism: Wyeth strain vaccinia virus engineered to directly lyse tumor cells and stimulate anti-tumor immunity

Trial(s) (Identifier): Hepatocellular Carcinoma Study Comparing Vaccinia Virus Based Immunotherapy Plus Sorafenib vs Sorafenib Alone (PHOCUS; NCT02562755; Phase III)

Updates: Pexa-Vec is also in a Phase I/II study in refractory colorectal cancer with AstraZeneca’s Imfinzi® (durvalumab) and tremelimumab (NCT03206073); in a Phase I study in metastatic/avanced solid tumors with Bristol-Myers’ Squibb (BMS)’s Yervoy® (ipililumab; NCT02977156); a Phase I/II study in advanced hepatocellular carcinoma with BMS’ Opdivo® (nivolumab; NCT03071094); and a Phase I study in renal cell carcinoma with Regeneron Pharmaceuticals’ REGN2810 (NCT03294083).


Sponsor(s): Advantagene

Indication(s): Prostate cancer; hepatocellular carcinoma; pancreatic adenocarcinoma; non-small cell lung cancer (NSCLC)

Mechanism: ProstAtak is based on Advantagene’s Gene Mediated Cytotoxic Immunotherapy (GMCI™) tech platform and consists of aglatimagene besadenovec and valacyclovir. Initial injections at the tumor site deliver aglatimagene besadenovec (AdV-tk), a gene vector derived from an adenovirus and engineered to deliver the thymidine kinase (tk) gene, derived from the Herpes Simplex virus, to the target cells. The target tissues and cells then produce the TK protein.

Trial(s) (Identifier): Phase 3 Study of ProstAtak® Immunotherapy with Standard Radiation Therapy for Localized Prostate Cancer (PrTK03; NCT01436968; Phase III); Randomized Controlled Trial of ProstAtak® Immunotherapy During Active Surveillance for Prostate Cancer (ULYSSES; NCT02768363; Phase II/III); Multicenter RCT of ADV-TK Gene Therapy Improving the Outcome of Liver Transplantation for Advanced HCC (NCT03313596; Phase III)

Updates: On April 27, Advantagene said it will partner with Bristol-Myers Squibb (BMS) to assess the combination of ProstAtak and BMS’ Opdivo® (nivolumab) in newly diagnosed, malignant glioma patients receiving standard-of-care surgery and radiation with or without temozolomide.

RT-100 (AC6 Gene Transfer)

Sponsor(s): Renova Therapeutics

Indication(s): Heart failure and reduced ejection fraction (HFrEF)

Mechanism: RT-100 is infused during cardiac catheterization directly into the arteries that feed the heart. The gene encoding human AC6 is delivered via a modified adenovirus vector that is able to enter the cells but cannot reproduce itself (Ad5.hAC6).

Trial(s) (Identifier): AC6 Gene Transfer in Patients with Reduced Left Ventricular Ejection Fraction Heart Failure (FLOURISH; NCT03360448)(Phase III)

Updates: As of April 10, NCT03360448 was active but not yet recruiting patients.

Toca 511 (vocimagene amiretrorepvec) and Toca FC

Sponsor(s): Tocagen, ApolloBio

Indication(s): Recurrent high-grade glioma (Phase II/III); metastatic solid tumors, including colorectal cancer, renal cell cancer, melanoma, pancreatic cancer, lung cancer, and breast cancer (Phase I); newly diagnosed high-grade glioma (Preclinical)

Mechanism: Two-part cancer immunotherapy. Toca 511 is an injectable retroviral replicating vector (RRV) that encodes a prodrug activator enzyme, cytosine deaminase (CD). Toca FC is an oral extended-release formulation of the prodrug 5-fluorocytosine (5-FC), which is converted into 5-fluorouracil (5-FU), when it encounters CD.

Trial(s) (Identifier): The Toca 5 Trial: Toca 511 & Toca FC Versus Standard of Care in Patients With Recurrent High Grade Glioma (Toca 5; NCT02414165);  A Study of Toca 511, a Retroviral Replicating Vector, Combined With Toca FC in Patients With Solid Tumors or Lymphoma (Toca 6; NCT02576665); A Study of the Safety of Toca 511, a Retroviral Replicating Vector, Combined With Toca FC in Subjects With Newly Diagnosed High Grade Glioma Receiving Standard of Care (Toca 7; NCT02598011)

Updates: On April 19, Tocagen granted ApolloBio exclusive rights to Toca 511 and Toca FC in the greater China region, in a deal that could generate up to $127 million-plus for Tocagen.

Valoctocogene Roxaparvovec (formerly BMN 270)

Sponsor(s): BioMarin Pharmaceutical

Indication(s): Hemophilia A

Mechanism: AAV-factor VIII vector

Trial(s) (Identifier): Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN270-301, also called GENEr8-1; NCT03370913); Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg (BMN270-302, also called GENEr8-2; NCT03392974)

Updates: BioMarin said April 25 it planned to dose “in the near future” the first GENEr8-1 patient with valoctocogene roxaparvovec manufactured in the company’s new commercial gene therapy facility in Novato, CA.

VB-111 (ofranergene obadenovec)

Sponsor(s): Vascular Biogenics, operating as VBL Therapeutics; NanoCarrier (Japan rights)

Indication(s): Solid tumors, including recurrent platinum-resistant ovarian cancer (Phase III)

Mechanism: Targeted anti-cancer gene-based biologic administered as an IV infusion once every two months. VB-111 was developed through VBL’s Vascular Targeting System (VTSTM). VBL says the mechanism combines blockade of tumor vasculature with an anti-tumor immune response.

Trial(s) (Identifier): A Study of VB-111 With Paclitaxel vs Paclitaxel for Treatment of Recurrent Platinum-Resistant Ovarian Cancer (OVAL; NCT03398655) (Phase III)

Updates: On March 8, VBL acknowledged that VB-111 missed its primary endpoint of overall survival in the Phase III GLOBE study assessing the treatment in combination with Roche/Genentech’s Avastin® (bevacizumab) in patients with recurrent glioblastoma (NCT02511405).


Sponsor(s): Inovio Pharmaceuticals, ApolloBio

Indication(s): Cervical high-grade squamous intraepithelial lesion (HSIL)

Mechanism: Immunotherapy designed to treat precancers and cancers caused by human papillomavirus (HPV); Inovio says VGX-3100 is the first immunotherapy for HPV-related cervical precancer. Using ASPIRE™ (Antigen SPecific Immune REsponses) technology, VGX-3100 includes DNA plasmids targeting the E6 and E7 proteins of HPV types 16 and 18. VGX-3100 is delivered intramuscularly using CELLECTRA® 5PSP electroportation technology.

Trial(s) (Identifier): REVEAL 1 (Evaluation of VGX-3100 and Electroporation for the Treatment of Cervical HSIL) (REVEAL 1; NCT03185013) (Phase III)

Updates: In March, ApolloBio closed an agreement with Inovio to develop VGX-3100 within China, Hong Kong, Macao, and Taiwan. ApolloBio agreed to pay Inovio $23 million upfront, up-to-$20 million in milestone payments, and double-digit tiered royalty payments. VGX-3100 is also in two Phase II trials.


Sponsor(s): ViroMed, Beijing Nuosilandi Biotechnology, Reyon Pharmaceutical

Indication(s): Painful diabetic peripheral neuropathy [DPN; U.S. and South Korea]; Chronic non-healing ischemic foot ulcer in diabetes [U.S.]; Critical limb ischemia [China]; Amyotrophic lateral sclerosis [ALS; U.S.]; Acute myocardial infarction [South Korea]

Mechanism: DNA-based medicine designed to express two isoforms of HGF (hepatocyte growth factor), HGF728 and HGF723

Trial(s): Safety and Efficacy Study of VM202 in the Treatment of Chronic Non-Healing Foot Ulcers (NCT02563522; Phase III); Phase III Gene Therapy for Painful Diabetic Neuropathy (NCT02427464); Hepatocyte Growth Factor to Improve Functioning in PAD (HI-PAD; NCT03363165; Phase II)

Updates: On April 17, ViroMed said a second Phase III trial in DPN was approved in the U.S. A day earlier, ViroMed published results from a study showing that VM202 reduced neuropathic pain symptoms in a mouse chronic constriction injury model.

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