Live from Open Targets Platform, fetched 2026-11-11 22:28 UTC. Every row links to its source record.

HBB hemoglobin subunit beta

ENSG00000244734 · protein coding · Open Targets profile

Involved in oxygen transport from the lung to the various peripheral tissues. .

2 of the 4 drugs recorded against HBB are approved

Each band is a group of drugs. It runs from the kind of drug on the left to the furthest stage that drug has reached in any disease on the right. Thicker bands mean more drugs.

Approved bands are highlighted. Withdrawn means the drug was pulled from the market or from development. A combined phase counts as the later one (Phase 1/2 is shown as Phase 2). Hover a band for the drug names. Source: Open Targets Platform, fetched live.

HBB: inherited and tumour-mutation evidence across 15 cancers

Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.

Inherited (germline) variantsMutations in tumours (somatic)

Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.

Tractability

Small molecule: Approved Drug, Structure with Ligand, High-Quality Ligand, High-Quality Pocket, Druggable Family
Antibody: GO CC high conf
PROTAC: Small Molecule Binder
Other modalities: Approved Drug

gnomAD loss of function constraint: o/e 1.8590999841690063 (upper bound 2.7567999362945557). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.

Who runs trials of these drugs

Principal investigators and study chairs on 2 registry trials, updated in the last three years, of the most advanced drugs against HBB (efaproxiral, lovotibeglogene autotemcel), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.

Anjulika Chawla
bluebird bio, Inc. · 2 of 2 trials Phase 2+
Ph1/Ph2NCT02140554 · A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease brief
Ph3NCT04293185 · A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease brief

2 of 2 trials of drugs against HBB have sites only in United States

Registry trials of efaproxiral, lovotibeglogene autotemcel updated in the last three years, counted by the country group of their listed sites. A trial with sites in several groups counts in each; 2 of 2 have all sites in one country.

Sites only in this groupSites here and elsewhere

Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where sites are is a fact about the record, not a judgement of its quality. Europe is the EU and EEA, the UK and Switzerland. Source: ClinicalTrials.gov, fetched live.

2 trials of drugs against HBB were stopped early

Terminated, withdrawn and suspended interventional trials of efaproxiral, lovotibeglogene autotemcel, newest update first, live from ClinicalTrials.gov. Each reason is quoted exactly as the registry gives it; we do not sort reasons into safety, efficacy or business. Dates are the last registry update. Showing the 2 most recent of 2.

N/ANCT00433472 brief why it stopped
withdrawn · 2015-07-29 · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · matched by the registry's drug synonyms
“not enough interest”
Ph3NCT00055887 brief why it stopped
withdrawn · 2013-05-10 · Spectrum Pharmaceuticals, Inc · efaproxiral
“Study never started. No patients were enrolled.”

Paediatric incentives and obligations for HBB's drugs

For efaproxiral, lovotibeglogene autotemcel: what EMA and FDA publish about paediatric plans, orphan status and pediatric approvals, and how many registered studies take children. Each line is a yes, no or not published fact from the record linked under it; none of it is a forecast.

FDA pediatric target list: no · HBB is not on the list
Rule: Target is on FDA's Pediatric Molecular Target List (relevant targets). Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
EU paediatric investigation plan: no · No EMA paediatric investigation plan decision names efaproxiral, lovotibeglogene autotemcel
Rule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA data download (dated snapshot), 2026-11-11
EU orphan designation: no · No EU orphan designation names efaproxiral, lovotibeglogene autotemcel
Rule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA data download (dated snapshot), 2026-11-11
Authorised in the EU: no · EMA lists no centrally authorised medicine of efaproxiral, lovotibeglogene autotemcel
Rule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA data download (dated snapshot), 2026-11-11
FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the link
Rule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
FDA pediatric oncology approval: no · FDA's table lists no pediatric approval of efaproxiral, lovotibeglogene autotemcel
Rule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-11
Registered studies open to children: yes · 2 interventional studies of efaproxiral, lovotibeglogene autotemcel include patients under 18; 1 is recruiting, not yet recruiting or active
Rule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-11

Rare pediatric disease voucher. FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs

All 4 drugs and clinical candidates as a table

Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.

Drug	Type	Highest stage	Indications	Source records
EFAPROXIRAL	Small molecule	approval	neoplasm, non-small cell lung carcinoma, breast cancer, malignant glioma	Open Targets · ChEMBL
LOVOTIBEGLOGENE AUTOTEMCEL	Gene	approval	sickle cell disease	Open Targets · ChEMBL
VOXELOTOR	Small molecule	withdrawal	anemia, Hemolytic anemia, sickle cell disease, idiopathic pulmonary fibrosis, Hypoxemia, acute lung injury, chronic kidney disease	Open Targets · ChEMBL
BETIBEGLOGENE AUTOTEMCEL	Gene	withdrawal	beta thalassemia	Open Targets · ChEMBL