Alex Philippidis
Alex Philippidis

Data released May 9 by the Alliance for Regenerative Medicine (ARM) illustrates the growth of clinical activity where gene therapy is concerned.

ARM’s Quarterly Regenerative Medicine Global Data Report for the first quarter of 2019 showed 372 gene therapy clinical trials were in progress as of the end of Q1. Interestingly, a majority (217 or 58%) were studies in Phase II, followed by Phase I (123 or 33%), and Phase III (32 or 9%).

The number of gene therapy clinical trials inched up by 10 from the 362 recorded as of the end of 2018. More significantly, the number of clinical studies jumped 17% year-over-year from the 319 trials in progress as of the first three months of 2018.

Also heating up is the pace of gene therapy activity outside of trials. On April 15, Catalent signaled its intent to expand into gene therapy when it said it had agreed to acquire Paragon Bioservices for $1.2 billion. Thermo Fisher Scientific will expand its capabilities in the field once it completes its planned $1.7 billion purchase of viral vector CDMO Brammer Bio, planned for the end of the second quarter.

The year’s biggest gene therapy M&A deal to date came in February, when Roche said it planned to acquire Spark Therapeutics for $4.8 billion. However, resistance from Spark shareholders seeking a higher price has compelled Roche to extend its tender-offer deadline twice; the offer is set to expire on June 3.

As for approvals of new gene therapies, two candidates considered close to approvals appear on this list. One is Zynteglo™ (formerly LentiGlobin™), a bluebird bio gene therapy for thalassemia and sickle cell disease that has received a positive recommendation for approval by the European Medicines Agency’s Committee for Human Medicinal Products—a usually-reliable indicator of final approval by the European Commission.

The other candidate in the homestretch of regulatory review is Zolgensma® (formerly AVXS-101) from AveXis, a Novartis Company. Zolgensma is a spinal muscular atrophy gene therapy that has won more news coverage for its planned multi-million-dollar price tag than for its positive Phase III trial data.

Zynteglo and Zolgensma are joined by 23 other “up-and-coming” gene-therapy candidates that have reached Phase III and/or registrational trials as of the first quarter of 2019, based on figures from ARM, as well as and/or company announcements. As with last year’s A-List, each candidate is listed by name, sponsor(s), indication(s), mechanism, late-stage trials, and updates.

Two gene therapy candidates on ARM’s list are not included. One is CMB305, a prime-boost cancer vaccine designed to target tumors that express NY-ESO-1. Immune Design halted a Phase III combination trial with Roche/Genentech’s Tecentriq® (atezolizumab) after disappointing Phase II results in synovial sarcoma. At the time, Immune Design said it would seek an external development partner for CMB305. Instead, the company was acquired by Merck & Co. for approximately $300 million, a deal completed April 2.

Also not included below is Amgen’s Imlygic® (talimogene laherparepvec). It was initially approved in 2015, and is now in trials for additional melanoma indications (NCT02263508 and NCT02211131).

Among promising candidates that bear watching is Audentes Therapeutics’ AT132, an X-linked myotubular myopathy candidate. Audentes plans third-quarter talks with the FDA and EMA on license application submission pathways following positive Phase I/II data—most recently on May 1, when the company announced “significant and sustained” improvements in neuromuscular function in both dose cohorts.

Another promising candidate, Avrobio’s AVR-RD-01 for Fabry disease, said April 29 it will move ahead with its Phase II FAB-201 trial (NCT03454893) and dosing of patients at U.S. clinical sites, following IND clearance from the FDA.


Axalimogene filolisbac (AXAL; 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 the 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); ADXS 11-001 Vaccination Prior to Robotic Surgery, HPV-Positive Oropharyngeal Cancer (NCT02002182; Phase II)

Updates: On May 15, the FDA imposed a partial clinical hold on AIM2CERV, saying Advaxis satisfactorily addressed questions on “certain AXAL chemistry, manufacturing, and controls (CMC) matters.” Advaxis is also assessing axalimogene filolisbac in combinations with Merck & Co.’s Keytruda® (pembrolizumab), Bristol-Myers Squibb’s Opdivo® (nivolumab), and AstraZeneca’s Imfinzi® (durvalumab).



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): NCT03569891 (HOPE-B, Phase III); NCT03489291 (Phase IIb)

Updates: uniQure on May 10 presented updated clinical data showing that clinically significant elevations of Factor IX (FIX) activity were sustained in all three patients up to six months after a single administration of AMT-061. The first patient achieved FIX activity of 51% of normal, the second patient, 33% of normal and the third patient, 57% of normal.


Fidanacogene elaparvovec (formerly SPK-9001 and PF-06838435)

Sponsor(s): Pfizer

Indication: Hemophilia B

Mechanism: Bio-engineered adeno-associated virus (AAV) capsid (protein shell) and a high-activity human coagulation factor IX gene.

Trials (Identifier): NCT03587116 (Phase III)

Updates: Spark Therapeutics transitioned development of what was SPK-9001 to Pfizer last year. Pfizer initiated a Phase III program in July 2018, starting with a lead-in study designed to assess the efficacy and safety of current factor IX prophylaxis replacement therapy, followed by evaluation of fidanacogene elaparvovec in hemophilia B.


Generx® (alferminogene tadenovec, Ad5FGF-4)

Sponsor(s): Angionetics, Huapont Life Sciences (exclusive territorial distribution and license agreement for China)

Indication(s): Refractory angina (Phase III); Cardiac syndrome X (Phase I/II); Congestive heart failure (Phase I/II); Moyamoya disease (IND-enabling), Cerebral ischemia (Candidate selection)

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): NCT02928094 (AFFIRM, Phase III)

Updates:  NCT02928094 is expected to start June 1, 2019, with an estimated primary completion date of June 1, 2021. Angionetics is a majority-owned subsidiary of Gene Biotherapeutics (formerly Taxus Cardium Pharmaceuticals Group), but operates independently. Last year, Angionetics said it plans to externally finance clinical development and commercialization of Generx, which could include an initial public offering in 2019.



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): NCT02652780 (REVERSE, Phase III); NCT02652767 (RESCUE, Phase III); NCT03293524 (REFLECT, Phase III).

Updates: On May 15, GenSight presented positive first 96-week results from the REVERSE trial showing continued efficacy of GS010 two years past injection. At Week 96, GS010-treated eyes showed a mean improvement of -0.308 LogMAR compared to baseline, equivalent to +15.4 ETDRS letters or three lines on the ETDRS vision chart. The company presented positive 72-week results on May 6.


Instiladrin® (nadofaragene firadenovec/Sny3; rAd-IFN/Syn3)

Sponsor(s):  FKD Therapies, Ferring Pharmaceuticals

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

Mechanism: Nonreplicating 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): NCT02773849 (Phase III)

Updates: On May 5, Colin Dinney, MD, of MD Anderson Cancer Center presented updated three-year Phase II data for Instiladrin in BCG unresponsive NMIBC patients. Of 34 men available for follow-up study, 24 were still alive three years after treatment. Of the other 10, 3 died of bladder cancer while 7 died of other causes. Ferring last year acquired an option for global commercialization rights to Instiladrin.



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):  NCT03852498 (ALD-104, Phase III); NCT01896102 (Starbeam or ALD-102, Phase II/III); NCT02698579 (Long-term Follow-up)

Updates: On May 2, bluebird bio said it treated the first patient in ALD-104 in April. The trial’s primary endpoint is the proportion of patients who are alive and do not have any of the six major functional disabilities at Month 24.



Sponsor(s): Lysogene, Regenxbio, Sarepta Therapeutics

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 the enzyme that breaks down heparan sulfate, slowing down or halting progression of the disease.

Trial(s) (Identifier): NCT03612869 (AAVance, Phase II/III); NCT02746341 (SAMOS, natural history study)

Updates:  Lysogene said on April 29 it expects to complete patient recruitment for AAVance in the first half of 2020.The first patient was recruited in December 2018, and treated two months later. In October 2018, Sarepta Therapeutics acquired U.S. and other ex-European rights to LYS-SAF302, which uses Regenxbio’s NAV AAVrh10 vector, in a deal that could generate more than $125 million for Lysogene.



Sponsor(s): Eyevance Pharmaceuticals, OcuNexus Therapeutics

Indication(s): Persistent epithelial defects (PED) that are nonresponsive 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 of current trials are not available. Nexagon was the subject of eight earlier studies, of which five were completed, two terminated, and one withdrawn.

Updates: Eyevance licensed worldwide rights to Nexagon from OcuNexus in October 2018 for an undisclosed price, saying it was funding a pivotal trial anticipated to start this year. The Phase IIb study would assess Nexagon as a potential treatment for PED caused by thermal or chemical burns, Eyevance CSO Mark Jasek, PhD, said on a video posted on YouTube November 5, 2018, by the organizer of the Ophthalmology Innovation Summit.



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): NCT03496012 (STAR, Phase III); NCT03507686 (GEMINI, Phase II); NCT03584165 (SOLSTICE, long-term follow up)

Updates: Nightstar agreed to be acquired by Biogen for approximately $877 million, the companies said March 4, in a deal designed to replenish the buyer’s ophthalmology drug pipeline with gene therapies led by NSR-REP1. On May 8, Nightstar shareholders approved the acquisition, expected to be completed June 7. Data from NCT03496012 is expected in the second half of 2020.



Sponsor(s): Orchard Therapeutics, Great Ormond Street Hospital for Children NHS Foundation Trust

Indication(s): Adenosine deaminase severe combined immune deficiency (ADA-SCID)

Mechanism: Autologous, ex vivo, hematopoietic stem cell gene therapy.

Trial(s) (Identifier): NCT03765632 (Phase I/II)

Updates: On February 22, Orchard presented positive two-year follow-up data in 20 patients from the fresh formulation registrational trial of OTL-101. Orchard reported 100% overall survival and 100% event free survival in patients treated with OTL-101 compared to 88% and 56% with historical hematopoietic stem cell transplant overall at 24 months. Orchard CMO Andrea Spezzi, MD, said the company is planning a BLA filing in 2020.


OTL-103 (formerly GSK2696275)

Sponsor(s): Orchard Therapeutics, 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): NCT01515462 (TIGET-WAS, Phase II); NCT03837483 (Phase II)

Updates: Orchard said March 21 it remained on track with 2019 plans to publish three-year follow-up data from the eight patients enrolled in NCT01515462, and begin enrollment in NCT03837483. Orchard acquired OTL-103 from GlaxoSmithKline last year.


OTL-200 (formerly GSK2696274)

Sponsor(s): Orchard Therapeutics, 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): NCT03392987 (Phase III)

Updates: On March 26, Orchard presented registrational data from a Phase I/II trial (NCT01560182): Late infantile patients showed 65 and 72 percentage point increases in gross motor function scores at 2- and 3-years post treatment, while early juvenile patients showed a 40 percentage point leap. Orchard intends to submit previously-planned regulatory filings in 2020. Orchard acquired OTL-200 from GlaxoSmithKline last year.


OXB-301 (TroVax®; MVA-5T4)

Sponsor(s): Oxford BioMedica, University College London, Cancer Research UK

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): NCT01556841 (TRIOC, Phase II)

Updates: NCT01556841 was completed as of May 9. In September 2018, Oncoimmunology published results from the Phase II SKOPOS trial (NCT01569919) showing 95.6% of patients developing cellular or humoral immune response to 5T4, meeting the study’s primary endpoint of at least 64%. Disease control was seen in 87% of patients (consisting of 69.6% showing stable disease and 17.4% partial response).


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): NCT02562755 (PHOCUS, Phase III); NCT03071094 (Phase I/II)

Updates: SillaJen said March 20 it would issue KRW 110 billion ($97.3 million) in convertible bonds toward clinical trials for Pexa-Vec. Studies will assess Pexa-Vec in liver and breast cancer, head and neck cancer, and neuroendocrine tumors. Initial data from PHOCUS is expected later this year, Transgene said in January—as is interim data from NCT03071094. Transgene holds European rights to Pexa-Vec.


ProstAtak® (aglatimagene besadenovec, AdV-tk)

Sponsor(s): Advantagene (d/b/a Candel Therapeutics)

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):  NCT01436968 (PrTK03, Phase III); NCT02768363 (ULYSSES, Phase II/III); NCT03313596 (LT-03, Phase III); NCT03576612 (Phase I)

Updates: On April 1, Advantagene (doing business as Candel Therapeutics) closed on a $22.5 million Series C preferred stock financing led by Northpond Ventures. Candel said March 21 it had dosed the first high-grade glioma patients in NCT03576612.


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): NCT03360448 (FLOURISH, Phase III)

Updates: NCT03360448 was not yet recruiting patients as of the most recent update posted on, dated December 18, 2018. Further updates not available.



Sponsor(s): Spark Therapeutics (acquisition planned by Roche)

Indication(s): Hemophilia A

Mechanism: Bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid

Trial(s) (Identifier): NCT03003533 (Phase I/II); NCT03432520 (long-term follow-up study); NCT03876301 (prospective, observational study)

Updates: Spark advanced SPK-8011 into Phase III last year, starting with an observational Phase III six-month run-in study. In December 2018, Spark presented updated preliminary data from 12 participants in an ongoing Phase I/II trial showing a 94% reduction in bleeds and a 95% reduction in FVIII infusions across all three doses, beginning four weeks after vector infusion.


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): NCT02414165 (Toca 5, Phase II/III); NCT02576665 (Toca 6, Phase I); NCT02598011 (Toca 7, Phase I)

Updates: In April, Tocagen presented preclinical data showing increased survival from the Toca 511/Toca FC combination with temozolomide or cyclophosphamide. Tocagen said the results support the planned Phase II/III NRG-BN006 trial, designed to evaluate Toca 511/Toca FC with standard of care in patients with newly diagnosed glioblastoma. NRG-BN006 is expected to begin enrollment in the second half of 2019.


Valoctocogene Roxaparvovec (formerly BMN 270)

Sponsor(s): BioMarin Pharmaceutical

Indication(s): Hemophilia A

Mechanism: AAV-factor VIII vector

Trial(s) (Identifier): NCT03370913 (BMN270-301 or GENEr8-1, Phase III); NCT03392974 (BMN270-302, also called GENEr8-2; Phase III)

Updates: BioMarin said April 25 that NCT03370913 is expected to be fully enrolled in the third quarter of 2019. The company has enrolled the subset of subjects needed to pursue a potential accelerated approval—a pathway made possible by the FDA’s draft guidance for hemophilia gene therapy products published last year.


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 anticancer 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): NCT03398655 (OVAL, Phase III)

Updates: Vascular Biogenics said March 28 that OVAL continues to enroll patients, with an interim efficacy readout expected by year’s end. During the second half of this year, the company also expects to launch a Phase III trial with the National Cancer Institute, exploring VB-111 in colon cancer in combination with a checkpoint inhibitor.



Sponsor(s): Inovio Pharmaceuticals, ApolloBio

Indication(s): Cervical high-grade squamous intraepithelial lesion (HSIL) associated with HPV-16 and HPV-18 infection (Phase III); pre-cancerous lesions of the vulva and anus (Phase II).

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): NCT03185013 (REVEAL 1, Phase III); NCT03721978 (REVEAL 2, Phase III)

Updates: Updates: Inovio on May 16 joined QIAGEN to announce they will co-develop a diagnostic test to identify patients most likely to respond to VGX-3100. On March 4, Inovio said REVEAL 2  had been launched, with plans for a BLA filing in 2021 as the first immunotherapy for women with cervical dysplasia. Later this year, Inovio expects interim Phase II results from NCT03603808, as well as NCT03499795 and NCT03180684.



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

Indication(s): Painful diabetic peripheral neuropathy [DPN; U.S. and South Korea]; Chronic nonhealing 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): NCT02563522 (VMNHU-003, Phase III); NCT02427464 (VMDN-003, Phase III); NCT03363165 (HI-PAD, Phase II); NCT03404024 (VMCAD-002, Phase II)

Updates: In a March 29 letter to investors, ViroMed CEO Sunyoung Kim, DPhil, said VM202 would be complemented or supplanted by one of three preclinical candidates set to reach Phase III by 2025. On February 1, Kim said ViroMed will receive results this summer from its first Phase III trial of VM202 for PDPN.


Zolgensma® (formerly AVXS-101)

Sponsor(s): AveXis, a Novartis company

Indication(s): Spinal muscular atrophy (SMA)

Mechanism: A nonreplicating 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): NCT03505099 (SPR1NT, Phase III); NCT03461289 (STRIVE-EU, Phase III); NCT03306277 (STR1VE, Phase III)

Updates: AveXis on May 5 trumpeted positive data for Zolgensma from several trials, including the first Phase III SPR1NT trial showing motor milestone achievement consistent with normal development in SMA patients treated pre-symptomatically. On May 8, AveXis President David Lennon, PhD, said Novartis is considering a Zolgensma price of $1.5 million to $5 million.


Zynteglo™ (formerly LentiGlobin™, BB305; autologous CD34+ cells encoding β A-T87Q-globin gene)

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): NCT02906202 (Northstar-2 or HGB-207, Phase III); NCT03207009 (Northstar-3 or HGB-212, Phase III)

Updates: bluebird on March 29 received a positive recommendation for conditional marketing authorization for Zynteglo from the European Medicines Agency’s Committee for Medicinal Products for Human Use. Bluebird said May 2 it expects to submit a BLA to the FDA by the end of 2019.

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