A complete medical intelligence index tracking every known cure, every functional cure, every treatment in development, and every future pathway to disease elimination across all major disease categories.
Direct-acting antivirals (sofosbuvir/ledipasvir) achieve >95% cure rates in 8–12 weeks. One of the greatest drug development successes in history. Cure exists — access is the remaining problem.
GILEAD · ABBVIE · MERCKCasgevy (CRISPR/Cas9) FDA-approved December 2023. Patients producing normal haemoglobin, pain crises eliminated. First CRISPR medicine ever approved — a historic milestone.
CRISPR THERAPEUTICS · VERTEX · BLUEBIRD BIOZolgensma (onasemnogene) — single-dose gene therapy dramatically changing trajectory of the most common genetic cause of infant death. Children reaching normal motor milestones.
NOVARTIS · BIOGENCheckpoint inhibitors (nivolumab, pembrolizumab) producing durable multi-year remissions in melanoma, lung, bladder, kidney cancers. CAR-T achieving >90% remission in some blood cancers.
BMS · MERCK · NOVARTIS · KITE · BRISTOLSemaglutide and tirzepatide producing 15–22% body weight loss with profound metabolic benefits. Type 2 diabetes remission achieved. Reducing cardiovascular events by 20%+.
NOVO NORDISK · ELI LILLY · PFIZERCell-free DNA, circulating tumour DNA (ctDNA), and exosome panels detected in routine blood draws identify cancer at Stage I with 70–90% sensitivity across 50+ cancer types. GRAIL Galleri, Exact Sciences, and Guardant Health offer multi-cancer early detection tests. FDA approval for ctDNA monitoring granted 2024.
GRAIL · EXACT SCIENCES · GUARDANT HEALTHSecond-generation chimeric antigen receptor T-cell therapies add armour domains, logic-gated dual-target recognition, and in vivo manufacturing protocols. Eliminates cytokine release syndrome risk through IL-6 circuit breakers. 2025 trials target solid tumours, previously resistant to CAR-T, using GD2, EGFR, and mesothelin antigens.
NOVARTIS · KITE PHARMA · ALLOGENEBeyond COVID vaccines, mRNA encoding cancer neoantigens (Moderna mRNA-4157/V940 with Merck), cardiovascular proteins (AstraZeneca heart failure), and rare disease enzymes (Translate Bio) enter Phase II/III. Lipid nanoparticle delivery achieves 85% hepatic transfection efficiency. Modular production enables personalised therapies in under 6 weeks.
MODERNA · BIONTECH · ASTRAZENECA · TRANSLATE BIOSemaglutide (Ozempic/Wegovy) and tirzepatide (Mounjaro) demonstrate 15–22% body weight reduction with cardiovascular mortality benefits confirmed in SELECT trial (2023). Pipeline includes oral small-molecule GLP-1 agonists, triple agonists (GLP-1/GIP/glucagon), and once-monthly injectable formulations entering trials 2025–2026.
NOVO NORDISK · ELI LILLY · STRUCTURE THERAPEUTICSIntellia Therapeutics NTLA-2001 (TTR amyloidosis) and Regeneron in vivo CRISPR show durable 90%+ protein reduction after single IV dose. Casgevy (exa-cel) received FDA and EMA approval December 2023 for sickle cell disease — first CRISPR therapy approved. In vivo lipid nanoparticle delivery to liver, lung, and muscle now demonstrated.
INTELLIA · REGENERON · CRISPR THERAPEUTICS · VERTEXPersonalised neoantigen vaccines for melanoma and pancreatic cancer showing 44–49% reduction in recurrence in Phase 2. Moderna + Merck partnership leading. Platform could address any tumour.
MODERNA · MERCK · BIONTECHFDA-approved esketamine (Spravato) for treatment-resistant depression. Psilocybin COMP360 Phase 3 trial shows 29% remission after a single dose. MDMA-assisted therapy (MAPS) in Phase 3 for PTSD. Ibogaine (Stanford 2023 veterans study) showing dramatic addiction reset. $2B+ rapidly legitimising pharmaceutical sector undergoing fastest regulatory approvals since mRNA vaccines.
COMPASS PATHWAYS · MAPS · ATAI LIFE SCIENCES · SMALL PHARMA · BECKLEY PSYTECHLatency reversal + broad neutralising antibodies + gene editing of viral reservoirs. Multiple trials showing patients maintaining undetectable viral load years after stopping ART.
GILEAD · ATEA · DEFYMED · UNIVERSITY OF OXFORDVertex's VX-880 — stem-cell-derived islet cells showing full insulin independence in early trial patients. Potentially eliminating insulin dependency for 8.5M people with T1D worldwide.
VERTEX · SERNOVA · SIGILON · NOVO NORDISKOne-time gene therapy showing curative factor levels lasting 5+ years in trials. Roctavian (haemophilia A) approved. Multiple next-gen therapies in Phase 3 with better durability.
BIOMARIN · SPARK · SANGAMO · PFIZER · CSL BEHRINGAnti-amyloid antibodies (lecanemab, donanemab) slowing decline 35%. Next wave: amyloid + tau + neuroinflammation combination. Prediction + prevention protocols emerging for at-risk patients.
BIOGEN · ELI LILLY · ROCHE · AC IMMUNECAR-Treg therapies and antigen-specific tolerance protocols reprogramming the immune system to stop attacking self — potentially curing rheumatoid arthritis, MS, lupus, and T1D at root.
SANGAMO · SONOMA BIOTHERAPEUTICS · CABALETTAMoving beyond symptom management to biological mechanism targeting. Ketamine analogue development (non-dissociative), targeted mGluR5 modulators for Fragile X and autism, AMPA potentiators for cognitive enhancement, and closed-loop neuromodulation implants for refractory OCD and depression. AI-driven biomarker discovery identifying treatment responders before prescribing.
JANSSEN · SAGE THERAPEUTICS · CEREVEL THERAPEUTICS · NEUROCRINE · ALTO NEUROSCIENCEMulti-system rejuvenation reversing biological age by decades — coordinated epigenetic reprogramming, senolytic clearance, and systemic factor restoration making 80 feel like 40.
ALTOS LABS · CALICO · RETRO BIOSCIENCES · LONGEVICADirect injection of gene-editing machinery — base editors, prime editors, CRISPR — that finds and corrects mutations in living tissue without removing cells from the body.
BEAM THERAPEUTICS · PRIME MEDICINE · INTELLIA · EDITReal-time computational model of your individual biology — genome, proteome, microbiome, metabolome — enabling fully personalised drug design, dosing, and disease prediction.
SIEMENS HEALTHINEERS · DASSAULT · MICROSOFT · GOOGLEEngineered immune systems that detect and eliminate any tumour cell before it grows. Annual immune reset preventing all cancers — cancer as a chronic, manageable condition rather than a death sentence.
IOVANCE · ACHILLES · NEON THERAPEUTICS · NCI| Disease / Condition | Category | Status | Progress | Current Best Treatment | Next Breakthrough |
|---|---|---|---|---|---|
|
Smallpox ▾
|
Infectious | ✓ ERADICATED | Vaccine (1980) | Biodefence monitoring | |
|
WHO Global Programme
📍 Geneva, Switzerland
Global eradication coordinated 1967–1980. Last case
1977. Two WHO-certified stockpiles remain at CDC (Atlanta)
and VECTOR Institute (Russia).
ERADICATED
CDC Biodefence
📍 Atlanta, GA, USA
Maintains certified smallpox stockpile and tecovirimat
(TPOXX) antiviral. Develops vaccines for mpox
cross-protection and bioterror scenarios.
ONGOING SURVEILLANCE
|
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|
Hepatitis C ▾
|
Infectious / Viral | ✓ CURED | Sofosbuvir (8–12 wks) | Access & screening scale-up | |
|
Gilead Sciences
📍 Foster City, CA, USA
Sofosbuvir (Sovaldi), Harvoni, Epclusa — DAA regimens
achieving 97–99% SVR12 cure rates in 8–12 weeks across
all HCV genotypes.
APPROVED — MARKET
AbbVie
📍 North Chicago, IL, USA
Mavyret (glecaprevir + pibrentasvir) — 8-week
pan-genotypic regimen, approved for CKD patients, cheapest
option in many markets.
APPROVED — MARKET
Merck (MSD)
📍 Rahway, NJ, USA
Zepatier (elbasvir/grazoprevir) — approved for genotypes
1 & 4. Strong real-world evidence across 90+
countries.
APPROVED — MARKET
|
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|
Sickle Cell Disease ▾
|
Genetic / Haematology | ◑ NEAR CURE | Casgevy (CRISPR, 2023) | In-vivo editing, cheaper LNP delivery | |
|
CRISPR Therapeutics
📍 Zug, Switzerland (R&D: Cambridge, MA)
Casgevy (exagamglogene autotemcel) — CRISPR/Cas9 editing
of BCL11A enhancer, re-activating fetal haemoglobin. FDA
& EMA approved Dec 2023. First CRISPR medicine ever
approved.
APPROVED 2023
Vertex Pharmaceuticals
📍 Boston, MA, USA
Co-developer of Casgevy. Joint commercialisation with
CRISPR Therapeutics. Also pursuing next-gen in-vivo
delivery to eliminate ex-vivo cell extraction step.
APPROVED 2023
bluebird bio
📍 Somerville, MA, USA
Lyfgenia (lovotibeglogene autotemcel) — lentiviral gene
addition approved Dec 2023. Anti-sickling HbAT87Q
haemoglobin eliminates vaso-occlusive crises. BBW for
haematologic malignancy.
APPROVED 2023
Intellia Therapeutics
📍 Cambridge, MA, USA
In-vivo CRISPR (NTLA-2002) delivered via LNP directly to
liver cells — no need to remove, edit, and reinfuse
patient cells. Targeting BCL11A locus in liver. Phase 1/2.
PHASE 1/2
|
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|
SMA Type 1 ▾
|
Genetic / Neuromuscular | ◑ NEAR CURE | Zolgensma gene therapy | Broader in-vivo delivery, earlier NBS | |
|
Novartis Gene Therapies
📍 Basel, Switzerland
Zolgensma (onasemnogene abeparvovec) — single IV dose of
AAV9 vector carrying SMN1 gene. Children reach normal
motor milestones. $2.1M per dose, approved 2019. Most
expensive drug in history.
APPROVED 2019
Biogen
📍 Cambridge, MA, USA
Spinraza (nusinersen) — intrathecal ASO therapy
increasing SMN2 exon 7 inclusion. First SMA treatment ever
approved (2016). Dosed 4× in year 1, 3× annually
thereafter.
APPROVED 2016
Roche / Genentech
📍 Basel, Switzerland / South San Francisco, CA
Evrysdi (risdiplam) — oral small-molecule SMN2 splicing
modifier. Once-daily syrup. FDA approved 2020. Uniquely,
penetrates CNS and peripheral motor neurons orally — no
injection needed.
APPROVED 2020
|
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|
Cystic Fibrosis ✦ ▾
|
Genetic / Pulmonary | ◑ NEAR CURE | Trikafta (Vertex, 2019) | Next-gen modulators for remaining 10% | |
|
Vertex Pharmaceuticals
📍 Boston, MA, USA
Trikafta (elexacaftor/tezacaftor/ivacaftor) — approved
2019. Triple CFTR modulator correcting the underlying
protein defect. FEV1 +14 percentage points,
hospitalisations -63%, sweat chloride normalised in 87% of
patients. Works for ~90% of CF patients with F508del
mutation.
APPROVED 2019 — TRANSFORMATIVE
Vertex Pharmaceuticals (next-gen)
📍 Boston, MA, USA
VX-522 — mRNA therapy delivering functional CFTR to lung
cells. Targeting the ~10% of CF patients with nonsense
mutations not responsive to current modulators. Phase 1/2
initiation planned. Could be universal CF treatment.
PRECLINICAL / PHASE 1
Cystic Fibrosis Foundation
📍 Bethesda, MD, USA
Funded Trikafta development via $150M milestone-based
partnership with Vertex. Now funding next-gen modulator
research and gene repair approaches for rare CF mutations
excluded from modulators.
RESEARCH FUNDER
|
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|
Childhood ALL Leukaemia ▾
|
Cancer / Blood | ◑ HIGH REMISSION | CAR-T + chemo (>90%) | Universal CAR-T, lower toxicity | |
|
Novartis
📍 Basel, Switzerland
Kymriah (tisagenlecleucel) — first-ever FDA-approved
CAR-T therapy (2017). CD19-targeting for paediatric and
young adult B-cell ALL. 81% complete remission at 3 months
in pivotal trial.
APPROVED 2017
Kite Pharma (Gilead)
📍 Santa Monica, CA, USA
Yescarta (axicabtagene ciloleucel) — CD19 CAR-T for
large B-cell lymphoma and FL. 73% ORR. Manufactured in 16
days. Also in paediatric ALL trials (KTE-C19).
APPROVED — EXPANDING
Allogene Therapeutics
📍 South San Francisco, CA, USA
AlloCAR-T — allogeneic (off-the-shelf) CAR-T from
healthy donors. ALLO-501A for B-cell NHL in Phase 2.
Targets: no 16–22 week manufacturing wait, immediate
treatment.
PHASE 2
|
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|
NAFLD / NASH (Fatty Liver) ▾
|
Metabolic / Hepatic | ◑ FIRST APPROVED 2024 | Resmetirom (Rezdiffra, FDA March 2024) | GLP-1 agonists, FXR agonists, ACC inhibitors | |
|
Madrigal Pharmaceuticals
📍 West Conshohocken, PA, USA
Rezdiffra (resmetirom) — liver-targeted THR-β agonist.
FDA approved March 2024 — first-ever approved treatment
for NASH/MASH with moderate-to-advanced fibrosis.
MAESTRO-NASH trial: NASH resolution in 26%, fibrosis
improvement in 24% at 52 weeks.
APPROVED MARCH 2024
Novo Nordisk
📍 Bagsværd, Denmark
Semaglutide Phase 3 ESSENCE trial in NASH cirrhosis —
Phase 2 data showed 59% NASH resolution and 43% fibrosis
improvement. GLP-1 mechanism reduces liver fat via weight
loss and direct hepatic effects. Results expected 2025.
PHASE 3
Intercept Pharmaceuticals (Alfasigma)
📍 Morristown, NJ, USA
Obeticholic acid (OCA) — FXR agonist. Phase 3
REGENERATE: fibrosis improvement in 23% of patients. FDA
Complete Response Letter — not yet approved. Acquired by
Alfasigma 2023. Still the most-studied FXR agonist in
NASH.
PHASE 3 (CRL RECEIVED)
|
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|
Haemophilia A/B ▾
|
Genetic / Coagulation | ◑ IN TRIALS | Roctavian, Hemgenix (FDA approved) | Next-gen AAV, better durability | |
|
BioMarin Pharmaceutical
📍 San Rafael, CA, USA
Roctavian (valoctocogene roxaparvovec) — AAV5 gene
therapy for Haemophilia A. FDA approved Aug 2023. Single
IV infusion. Factor VIII levels >50% in majority of
patients at 3 years.
APPROVED 2023
CSL Behring / uniQure
📍 King of Prussia, PA, USA / Lexington, MA, USA
Hemgenix (etranacogene dezaparvovec) — AAV5 gene therapy
for Haemophilia B. FDA approved Nov 2022. Factor IX levels
normalise in most patients after single infusion. $3.5M
per dose.
APPROVED 2022
Pfizer / Sangamo
📍 New York, USA / Brisbane, CA, USA
Beqvez (fidanacogene elaparvovec) — AAV vector for
Haemophilia B. FDA approved Apr 2024. Zinc-finger
nuclease–corrected AAV cassette showing durable Factor
IX expression.
APPROVED 2024
Spark Therapeutics (Roche)
📍 Philadelphia, PA, USA
SPK-8011 for Haemophilia A — AAVrh10 vector. Phase 3
pivotal trial. Unique capsid aimed at patients with
pre-existing AAV5 immunity (excluded from Roctavian).
PHASE 3
|
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|
Duchenne Muscular Dystrophy ▾
|
Genetic / Neuromuscular | ◑ PARTIAL TREATMENT | Elevidys (Sarepta, FDA 2023) | Full-length dystrophin gene therapy | |
|
Sarepta Therapeutics
📍 Cambridge, MA, USA
Elevidys (delandistrogene moxeparvovec) — FDA
accelerated approval June 2023 for ambulatory DMD patients
aged 4–5. AAV-rh74 delivering micro-dystrophin gene.
Produces functional dystrophin protein in muscle. Full
approval 2024 expanded to age 4+.
APPROVED 2023/2024
Solid Biosciences
📍 Cambridge, MA, USA
SGT-003 — next-gen micro-dystrophin gene therapy with
improved capsid (AAVMYO) for better muscle penetration.
Phase 1/2. Addresses exon mutations not covered by
Elevidys and systemic delivery to cardiac muscle.
PHASE 1/2
Sarepta / Nationwide Children's
📍 Cambridge, MA / Columbus, OH, USA
Exon-skipping antisense oligos — eteplirsen (approved
2016), golodirsen, viltolarsen, casimersen — skipping
specific exons to restore partial reading frame. Combined
exon-skipping + gene therapy pipeline targeting all
mutation types.
MULTIPLE APPROVED
|
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|
Malaria ▾
|
Infectious / Parasitic | ◑ PARTIAL VACCINE | R21/Matrix-M vaccine (77% efficacy) | Anti-PfSPZ mAbs, mRNA malaria vaccine | |
|
Oxford / Serum Institute of India
📍 Oxford, UK / Pune, India
R21/Matrix-M — 77% efficacy in Phase 3 trials. WHO
prequalified 2023. First vaccine to meet WHO's 75%
efficacy target. Serum Institute producing 100M+
doses/year at <$3/dose.
WHO APPROVED 2023
Sanaria / NIAID
📍 Rockville, MD, USA
PfSPZ vaccine — live attenuated whole-sporozoite
vaccine. Phase 2: 100% protection over 6 months in
controlled trials. IV administration challenge; working on
injectable formulation.
PHASE 2
Moderna
📍 Cambridge, MA, USA
mRNA malaria vaccine targeting PfCSP antigen —
preclinical/early Phase 1. Platform advantage: rapid
adaptation to new strains, including artemisinin-resistant
variants emerging in SE Asia.
PHASE 1
|
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|
Hepatitis B ▾
|
Infectious / Viral | ◑ FUNCTIONAL CURE TRIALS | Tenofovir/entecavir (viral suppression) | ABI-H0731 + RNAi combos, HBsAg loss | |
|
Assembly Biosciences
📍 South San Francisco, CA, USA
ABI-H0731 (vebicorvir) — core inhibitor blocking HBV
capsid assembly. In combination with NrtI backbone showing
HBsAg decline in Phase 2. Target: HBsAg loss = functional
cure endpoint.
PHASE 2
Arbutus Biopharma / Genevant
📍 Warminster, PA, USA / Vancouver, BC, Canada
AB-729 — RNAi agent reducing HBsAg levels. LNP-delivered
siRNA silencing HBV transcripts. Phase 2 combination
studies with ABI-H0731 showing additive HBsAg suppression.
PHASE 2
Gilead Sciences
📍 Foster City, CA, USA
Multiple HBV pipeline candidates including selgantolimod
(TLR8 agonist), GSK836 (antisense oligonucleotide), and
subcutaneous lenacapavir extension to HBV. Largest
pipeline targeting functional cure.
PHASE 2
|
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|
HIV / AIDS ▾
|
Infectious / Viral | ◑ CONTROLLED | ART (undetectable VL) | Latency reversal + gene editing cure | |
|
Gilead Sciences
📍 Foster City, CA, USA
Biktarvy (bictegravir/FTC/TAF) — #1 ART globally.
Lenacapavir (Sunlenca) — twice-yearly injectable capsid
inhibitor approved 2022 for multi-drug resistant HIV.
PURPOSE 1 trial: 100% HIV prevention in women.
APPROVED — MARKET
ViiV AI applications have shown promising results in the
early detection of diseases such as Alzheimer's through
medical imagery analysis. AI has been integrated into
patient care systems, facilitating diagnosis and
personalized treatment plans. (GSK)
📍 Brentford, London, UK
Cabotegravir LA + rilpivirine LA — monthly/bi-monthly
injectable ART. Cabotegravir injectable PrEP (Apretude)
— twice-yearly prevention injection, 88% more effective
than daily Truvada.
APPROVED — MARKET
Excision BioTherapeutics
📍 San Francisco, CA, USA
EBT-101 — CRISPR-based gene editing to excise integrated
HIV provirus from CD4+ T cells and macrophages. Phase 1
— first human CRISPR HIV cure attempt. Two patients
showed functional cure signals 2023.
PHASE 1
IAVI (non-profit)
📍 New York, NY, USA
Broadly neutralising antibodies (bNAbs) — VRC01,
3BNC117, 10-1074 in Phase 2. Combination bNAb cocktails
targeting multiple Env epitopes showing 100-week viral
suppression after ART cessation.
PHASE 2
|
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|
Type 2 Diabetes ▾
|
Metabolic / Endocrine | ◑ REMISSION POSSIBLE | GLP-1 agonists + lifestyle | Tirzepatide + bariatric protocols | |
|
Novo Nordisk
📍 Bagsværd, Denmark
Ozempic/Wegovy (semaglutide) — GLP-1 receptor agonist.
Weekly injection achieving 15% body weight loss and T2D
remission in many patients. Also reduces CV events 20%
(SUSTAIN-6). Global shortage ongoing.
APPROVED — GLOBAL
Eli Lilly
📍 Indianapolis, IN, USA
Mounjaro/Zepbound (tirzepatide) — dual GLP-1/GIP
agonist. Phase 3 SURMOUNT: 22.5% weight loss, T2D
remission in 51% of patients at 72 weeks. Faster, stronger
than semaglutide alone.
APPROVED 2022/2023
Structure Therapeutics
📍 South San Francisco, CA, USA
GSBR-1290 — oral GLP-1RA, once-daily pill. Phase 2
results: −9% body weight at 12 weeks with no nausea.
First potentially competitive oral alternative to weekly
injections.
PHASE 2
|
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|
Type 1 Diabetes ▾
|
Autoimmune / Metabolic | ◑ IN TRIALS | Insulin + CGM | Vertex stem-cell islets (VX-880) | |
|
Vertex Pharmaceuticals
📍 Boston, MA, USA
VX-880 — stem-cell-derived islet cells delivered IV.
Phase 1/2: first 3 patients achieved full insulin
independence at 90 days (including one at 2 years).
Potentially curative single infusion.
PHASE 1/2
Sernova Corp
📍 London, Ontario, Canada
Cell Pouch System — implantable scaffold creating
vascularised tissue site for donor islet transplant. Phase
2: insulin independence achieved in 70% of patients at 1
year. No immunosuppression variant in development.
PHASE 2
Provention Bio (Sanofi)
📍 Red Bank, NJ, USA / Paris, France
Tzield (teplizumab) — anti-CD3 mAb FDA-approved 2022.
Delays T1D onset by median 2 years in high-risk
individuals. First disease-modifying T1D therapy —
prevention rather than cure.
APPROVED 2022
|
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|
Melanoma (advanced) ▾
|
Cancer / Skin | ◑ LONG REMISSION | Checkpoint inhibitors | mRNA personalised neoantigen vaccine | |
|
Bristol Myers Squibb
📍 New York, NY, USA
Opdivo (nivolumab) + Yervoy (ipilimumab) — dual PD-1 +
CTLA-4 blockade. 5-year OS 52% in advanced melanoma.
Previously median survival was 6–9 months. Now 49% alive
at 6.5 years.
APPROVED — STANDARD OF CARE
Merck
📍 Rahway, NJ, USA
Keytruda (pembrolizumab) — anti-PD-1. Adjuvant use
post-resection reducing recurrence 44%. In combination
with mRNA-4157 vaccine (Phase 3) showing 49% reduction in
relapse vs Keytruda alone.
APPROVED + PHASE 3 COMBO
Moderna
📍 Cambridge, MA, USA
mRNA-4157/V940 — personalised neoantigen cancer vaccine.
Manufactured in 8 weeks from patient tumour biopsy. Phase
3 (KEYNOTE-942): 49% reduction in recurrence or death vs
Keytruda alone. Pivotal filing planned 2025.
PHASE 3 — BREAKTHROUGH
BioNTech
📍 Mainz, Germany
BNT111 (FixVac) — fixed-combo mRNA vaccine encoding
melanoma-specific antigens (NY-ESO-1, MAGEA3, tyrosinase,
TPTE). Phase 2 combined with cemiplimab.
PHASE 2
|
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|
Rheumatoid Arthritis ▾
|
Autoimmune | ◑ WELL CONTROLLED | Biologics, JAK inhibitors | CAR-Treg tolerance therapies | |
|
AbbVie
📍 North Chicago, IL, USA
Humira (adalimumab) — anti-TNF, world's bestselling drug
($20B/yr). Rinvoq (upadacitinib) — selective JAK1
inhibitor outperforming adalimumab in head-to-head
SELECT-COMPARE trial.
APPROVED — STANDARD OF CARE
Sangamo Therapeutics
📍 Brisbane, CA, USA
TX200 — CAR-Treg therapy engineering regulatory T cells
to home to joint synovium and suppress autoimmune attack.
Phase 1 (kidney transplant tolerance first, RA next).
First antigen-specific tolerance approach.
PHASE 1
Cabaletta Bio
📍 Philadelphia, PA, USA
CABA-201 — anti-CD19 CAR-T depleting autoreactive B
cells. Phase 1 RESET-RA initiated 2024. Logic: if
autoreactive B cells are deleted and the immune system
resets, disease remission without ongoing
immunosuppression.
PHASE 1
|
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|
Lupus (SLE) ▾
|
Autoimmune | ◑ BREAKTHROUGH TRIAL | Hydroxychloroquine, belimumab, anifrolumab | Anti-CD19 CAR-T B-cell depletion | |
|
University Hospital Erlangen / Heidelberg
📍 Erlangen & Heidelberg, Germany
Anti-CD19 CAR-T B-cell depletion — 5/5 patients in
complete drug-free remission in 2022–2024 trials.
Patients taken off all immunosuppression. Median follow-up
17 months — all remain in remission. Potentially
curative for refractory SLE. Landmark Nature Medicine
publications.
PHASE 1 — LANDMARK RESULTS
AstraZeneca / Viela Bio
📍 Cambridge, UK / Gaithersburg, MD, USA
Anifrolumab (Saphnelo) — anti-interferon-α receptor
mAb. FDA approved 2021. First new mechanism for SLE in a
decade. Reduces flares 36% in TULIP-2 trial. Best in class
for interferon-high SLE patients.
APPROVED 2021
GlaxoSmithKline
📍 Brentford, London, UK
Benlysta (belimumab) — anti-BLyS mAb, FDA approved 2011.
IV and subcutaneous forms. Reduces flares, protects
kidneys. First biologic approved for lupus in 50 years.
CAR-T approaches from Heidelberg group now being
commercialised.
APPROVED — STANDARD
|
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|
Multiple Sclerosis ▾
|
Autoimmune / Neuro | ◑ PROGRESSION SLOWED | DMTs (ocrelizumab) | Remyelination, BTK inhibitors | |
|
Roche / Genentech
📍 Basel, Switzerland / South San Francisco, CA
Ocrevus (ocrelizumab) — anti-CD20 mAb depleting B cells.
Standard of care for relapsing and primary progressive MS.
Reduces relapses 47% vs interferon. First therapy approved
for PPMS.
APPROVED — STANDARD OF CARE
Sanofi
📍 Paris, France
Tolebrutinib — oral BTK inhibitor preventing
CNS-resident microglial and B-cell driven inflammation.
Phase 3 HERCULES (SPMS): first drug to slow SPMS
disability in 10 years. NDA filing expected 2025.
PHASE 3 — NDA PENDING
Myelin Repair Foundation
📍 Saratoga, CA, USA
opicinumab (anti-LINGO-1) remyelination program —
promotes oligodendrocyte differentiation and myelin sheath
re-growth after damage. Phase 2b AFFINITY study. First
therapy addressing repair rather than just halting damage.
PHASE 2b
|
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|
Treatment-Resistant Depression
▾
|
Neuropsychiatric | ◑ PSYCHEDELIC REVOLUTION | Esketamine (Spravato, FDA 2019) | Psilocybin COMP360, MDMA therapy, ibogaine | |
|
Johnson & Johnson (Janssen)
📍 Titusville, NJ, USA
Spravato (esketamine) nasal spray — FDA approved 2019
for treatment-resistant depression and 2020 for MDD with
acute suicidal ideation. 29% remission at 4 weeks vs 20%
placebo. First new TRD mechanism in 25 years. Given in
certified healthcare settings.
APPROVED 2019
COMPASS Pathways
📍 London, UK
COMP360 (synthetic psilocybin) — Phase 3 COMP360-P3 in
TRD. Phase 2b: 29% remission rate after a single 25mg dose
at 3 weeks. Largest psilocybin trial ever run. FDA
Breakthrough Therapy designation. Primary endpoint results
expected 2025.
PHASE 3
MAPS (Multidisciplinary Assoc. for Psychedelic Studies)
📍 San Jose, CA, USA
MDMA-assisted therapy for PTSD — Phase 3 MAPP1 and
MAPP2: 67% of participants no longer met PTSD criteria vs
32% placebo. FDA advisory committee raised concerns about
trial blinding; Complete Response Letter received 2024.
Regulatory path continues.
PHASE 3 / CRL
Stanford University / ATAI Life Sciences
📍 Stanford, CA, USA / Berlin, Germany
Ibogaine study (Stanford, 2023) — veterans with TBI and
PTSD: single ibogaine dose produced dramatic reductions in
PTSD (88%), anxiety (87%), and depression (87%) at one
month. No control — but striking effect sizes prompting
formal trials.
OBSERVATIONAL → PHASE 1
|
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Huntington's Disease ▾
|
Genetic / Neurological | DISEASE-MODIFYING TRIALS | Tetrabenazine (symptom only) | Tominersen HTT-ASO, Wave WVE-003 | |
|
Roche / Ionis Pharmaceuticals
📍 Basel, Switzerland / Carlsbad, CA, USA
Tominersen — intrathecal ASO reducing huntingtin protein
40–60%. Phase 3 GENERATION HD1 paused 2021 due to
adverse events; redesigned dose-selection Phase 3 restart
ongoing. Brain delivery via lumbar puncture every 2
months.
PHASE 3 RESTART
Wave Life Sciences
📍 Cambridge, MA, USA
WVE-003 — allele-selective ASO targeting mutant
huntingtin only, preserving wild-type protein. Phase 1b/2a
SELECT-HD: significant reduction in mHTT. Advantage over
tominersen: spares normal huntingtin needed for neuronal
function.
PHASE 1b/2a
uniQure
📍 Lexington, MA, USA / Amsterdam, Netherlands
AMT-130 — AAV5 gene therapy delivering miHTT
(microRNA-based silencing) directly to striatum via
stereotactic injection. Phase 1/2: huntingtin protein
reduced 60–75% in spinal fluid. One-time brain surgery.
PHASE 1/2
|
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Tuberculosis (TB) ▾
|
Infectious / Bacterial | ACTIVE RESEARCH | BPaL regimen (6-month XDR-TB) | mRNA TB vaccine, new drug combos | |
|
Janssen (J&J)
📍 Beerse, Belgium
Bedaquiline — backbone of BPaL regimen (bedaquiline +
pretomanid + linezolid). 6-month course curing 89% of
XDR-TB cases. FDA approved. The first new TB drug class in
50 years.
APPROVED — BPaL REGIMEN
Moderna
📍 Cambridge, MA, USA
mRNA-1095 TB vaccine — Phase 1 initiated 2023. First
mRNA vaccine for TB. Targeting MTBVAC and M72/AS01E
antigens. Could overcome BCG's limited efficacy in
pulmonary TB.
PHASE 1
Gates Medical Research Institute
📍 Cambridge, MA, USA
M72/AS01E adjuvanted vaccine — Phase 2b results: 50%
efficacy against TB disease in HIV-negative adults. Phase
3 funded. Non-profit accelerating regimens inaccessible to
pharma.
PHASE 3 PENDING
|
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Alzheimer's Disease ▾
|
Neurodegeneration | ◑ EARLY INTERVENTION | Lecanemab (slows 35%) | Amyloid + tau + inflammation combo | |
|
Biogen / Eisai
📍 Cambridge, MA, USA / Tokyo, Japan
Leqembi (lecanemab) — anti-amyloid antibody. FDA
approved Jan 2023 (accelerated) → full approval Jul
2023. Slows cognitive decline 27% at 18 months (CLARITY
AD). Targets protofibrils — more specific than
aducanumab.
APPROVED 2023
Eli Lilly
📍 Indianapolis, IN, USA
Kisunla (donanemab) — FDA approved Jul 2024. Plaque
removal in 84% of patients. TRAILBLAZER-ALZ2: 35% slowing
of decline in early AD. Stops dosing once plaques cleared
— unique among all anti-amyloids.
APPROVED 2024
AC Immune
📍 Lausanne, Switzerland
ACI-35.030 — tau vaccine (targeting phospho-tau
oligomers). Phase 2 JACI-35.054 with Janssen. Also
semorinemab (anti-tau antibody) Phase 2. Tau targeting
needed for second-wave therapy alongside amyloid.
PHASE 2
Neurimmune / UCB
📍 Schlieren, Switzerland / Brussels, Belgium
NI006 — novel amyloid fibril–clearing antibody
entering Phase 2. Reverse translational approach derived
from cognitively normal elderly human antibodies. UCB0107
anti-tau Phase 1.
PHASE 1/2
|
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|
Parkinson's Disease ▾
|
Neurodegeneration | MANAGED | L-DOPA + DBS | Alpha-synuclein targeting, stem cells | |
|
Prothena Corporation
📍 Dublin, Ireland / South San Francisco, CA
Prasinezumab (PRX002/RO7046015) — anti-alpha-synuclein
mAb. Phase 2b PADOVA: slowed motor progression 35% in
fast-progressing patients. Phase 2b extension ongoing.
Roche partnership.
PHASE 2b
Neuropore Therapies / UCB
📍 San Diego, CA, USA / Brussels, Belgium
NPT520-34 / UCB0599 — small-molecule inhibitor of
alpha-synuclein aggregation (oral). Phase 2 PROPEL study.
First oral disease-modifying approach — pills vs
injections.
PHASE 2
BlueRock Therapeutics (Bayer)
📍 New York, NY, USA / Berlin, Germany
DA01 — iPSC-derived midbrain dopaminergic neurons
implanted into putamen. Phase 1 open-label: first patient
implanted 2023, showing cell survival and early motor
benefit signals at 12 months.
PHASE 1
|
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|
ALS (Lou Gehrig's) ▾
|
Neurodegeneration | RESEARCH | Tofersen (SOD1-ALS, 2023) | Broader ASO, gene silencing | |
|
Biogen / Ionis Pharmaceuticals
📍 Cambridge, MA, USA / Carlsbad, CA, USA
Tofersen (Qalsody) — ASO silencing mutant SOD1. FDA
approved Apr 2023 for SOD1-ALS. Reduces SOD1 protein 38%,
slows biomarker progression. First ALS therapy addressing
a genetic cause.
APPROVED 2023 (SOD1-ALS)
Ionis Pharmaceuticals
📍 Carlsbad, CA, USA
ION363 (jacifusen) — intrathecal ASO for FUS-ALS. FDA
Expanded Access. Showed 58% reduction in disease
progression in compassionate use. Pivotal trial design
underway.
PHASE 2 / EAP
QurAlis
📍 Cambridge, MA, USA
QRL-201 — ASO targeting STMN2 to restore truncated STMN2
protein (TDP-43 loss-of-function pathway). Addresses the
most common molecular defect in sporadic ALS. Phase 1
initiation 2024.
PHASE 1
|
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|
Solid Tumours (various) ▾
|
Cancer / Oncology | VARIES BY TYPE | Targeted therapy, immunotherapy | Personalised vaccines, ADCs | |
|
Daiichi Sankyo / AstraZeneca
📍 Tokyo, Japan / Cambridge, UK
Enhertu (trastuzumab deruxtecan) — HER2-targeting ADC. 5
approvals across breast, gastric, lung, colorectal cancers
in 3 years. 28% ORR in HER2-low patients (previously
untreatable). The poster-child for next-gen ADCs.
5 APPROVALS
Moderna
📍 Cambridge, MA, USA
mRNA-4157 personalised cancer vaccine — tumour biopsy
→ neoantigen sequencing → custom mRNA manufactured in
8 weeks → combined with Keytruda. Phase 3 across
melanoma, NSCLC, bladder, kidney cancers.
PHASE 3 MULTIPLE TUMOURS
Iovance Biotherapeutics
📍 Philadelphia, PA, USA
Amtagvi (lifileucel) — FDA approved Feb 2024 for
metastatic melanoma. TIL therapy: tumour-infiltrating
lymphocytes extracted, expanded 6,000×, reinfused. 31.5%
ORR in patients failed all other therapies.
APPROVED 2024
|
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|
Aging ▾
|
Systemic / Universal | RESEARCH STAGE | Lifestyle, metformin, rapamycin | Epigenetic reprogramming, senolytics | |
|
Altos Labs
📍 Cambridge, UK + San Diego, CA + Bay Area, CA
Yamanaka factor (OSKM) partial reprogramming to reverse
epigenetic age without inducing pluripotency. $3B raised
2022. Shinya Yamanaka, Jennifer Doudna, and Juan Carlos
Izpisua Belmonte leading. Tissue-specific rejuvenation in
2025–2027 target.
PRECLINICAL
Calico Life Sciences (Google/Alphabet)
📍 South San Francisco, CA, USA
Multi-target longevity biology — studying naked mole
rats, AbbVie partnership. Focusing on hallmarks of aging:
proteostasis, mitochondrial function, and cellular
senescence. Research publication cadence accelerating.
RESEARCH
Retro Biosciences
📍 Redwood City, CA, USA
$180M from Sam Altman. Three programmes: plasma dilution
(removing pro-aging circulating factors), autophagy
enhancement (chaperone-mediated autophagy), and partial
reprogramming. Human trials target 2026.
PRECLINICAL
Unity Biotechnology
📍 Brisbane, CA, USA
UBX1325 — senolytic (ABT-263-class) clearing senescent
cells in the retina. Phase 2 for diabetic macular oedema
and AMD showing meaningful visual acuity improvement at 24
weeks. Pivot from systemic to local senolytic.
PHASE 2
|
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|
Long COVID ▾
|
Infectious / Post-Viral | ✗ NO TREATMENT | Symptom management only | BC007 antibody, extended Paxlovid trials | |
|
Berlin Cures
📍 Berlin, Germany
BC007 — G-protein-coupled receptor antibody neutraliser.
Targets autoantibodies implicated in autonomic dysfunction
and vascular dysregulation in Long COVID and ME/CFS. Phase
2 initiated 2023.
PHASE 2
Pfizer / NIAID (NIH)
📍 New York, USA / Bethesda, MD, USA
RECOVER-VITAL trial — extended Paxlovid
(nirmatrelvir/ritonavir) 15-day course for Long COVID
fatigue and cognitive symptoms. 155 sites across US.
Results expected 2025.
PHASE 3 TRIAL
Weill Cornell / Stanford Medicine
📍 New York, NY / Stanford, CA, USA
ME/CFS-protocol research applying fibromyalgia and
dysautonomia management to Long COVID. Identifying viral
persistence (spike protein in tissues) as likely driver.
Antiviral + immunomodulatory combos.
RESEARCH
|
|||||
|
Frontotemporal Dementia (FTD)
▾
|
Neurodegeneration | ✗ NO TREATMENT | Symptom management only | GRN gene therapy, C9orf72 ASOs, TDP-43 | |
|
Passage Bio
📍 Philadelphia, PA, USA
PBFT02 — AAV1 gene therapy delivering GRN (progranulin)
gene to treat GRN-FTD. Phase 1/2 SUNRISE-FTLD: progranulin
levels restored in CSF. GRN mutations cause 5–10% of all
FTD via progranulin haploinsufficiency.
PHASE 1/2
Ionis Pharmaceuticals
📍 Carlsbad, CA, USA
IONIS-C9Rx — ASO targeting C9orf72 repeat expansion
(most common FTD/ALS mutation, 25–40% of familial
cases). Delivered intrathecally. Phase 1 in C9orf72-FTD
and ALS. First potential disease-modifying therapy for
this genetic form.
PHASE 1
Alector
📍 South San Francisco, CA, USA
AL001 (latozinemab) — anti-SORT1 antibody increasing
progranulin levels. Phase 3 INFRONT-3 in GRN-FTD: did not
meet primary endpoint. Signals of slowing in biomarkers.
Next-gen approach targeting TMEM106B ongoing.
PHASE 3 (MIXED RESULTS)
|
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|
Pancreatic Cancer ▾
|
Cancer / Solid Tumour | ✗ POOR PROGNOSIS | FOLFIRINOX, gem/nab-paclitaxel | KRAS inhibitors, mRNA vaccines, liquid biopsy | |
|
Mirati Therapeutics (now BMS)
📍 San Diego, CA, USA
MRTX1133 — KRAS G12D inhibitor. First potent, selective
KRAS G12D inhibitor. Phase 1/2 in pancreatic cancer —
the mutation present in 40% of all pancreatic cancers.
KRAS was "undruggable" for 40 years.
PHASE 1/2
Revolution Medicines
📍 Redwood City, CA, USA
RMC-6236 — RAS(ON) multi-selective inhibitor hitting
KRAS G12D, G12V, and G13C. Phase 1/2 results: 40% ORR in
pancreatic cancer. Novel tri-complex mechanism vs standard
competitive inhibitors.
PHASE 1/2
Moderna / Merck
📍 Cambridge, MA, USA / Rahway, NJ, USA
mRNA-4157 (V940) personalised neoantigen vaccine +
Keytruda — MESO-01 trial in resected pancreatic cancer.
Pancreatic cancer has high mutational burden and poor
immune infiltration — vaccines may overcome immune
evasion.
PHASE 2
Grail (Illumina)
📍 Menlo Park, CA, USA
Galleri multi-cancer early detection liquid biopsy —
detects 50+ cancers including pancreatic from a single
blood draw. The key unmet need: 85% of pancreatic cancers
diagnosed at Stage IV. Early detection could transform
survival rates.
COMMERCIAL — NHS TRIAL
|
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|
Glioblastoma (GBM) ▾
|
Cancer / Brain | ✗ NO CURE | Temozolomide + radiation (15 mo survival) | CAR-T, tumour-treating fields, oncolytic virus | |
|
Novocure
📍 Haifa, Israel / New York, USA
Optune (tumour treating fields, TTFields) — device
delivering low-intensity alternating electric fields
disrupting tumour cell division. FDA approved for newly
diagnosed and recurrent GBM. Adds ~2 months median OS on
top of standard chemo; some long-term survivors.
APPROVED — ADJUNCT
Bristol Myers Squibb / BioNTech
📍 New York, USA / Mainz, Germany
EGFRvIII-targeting CAR-T and mRNA neoantigen vaccines.
Blood-brain barrier penetration via convection-enhanced
delivery (CED). Multiple Phase 1 trials in recurrent GBM.
Intracavitary delivery post-resection showing early
signals.
PHASE 1
DNAtrix / DelMar Pharma
📍 Houston, TX, USA
DNX-2401 — oncolytic adenovirus injected into tumour
post-resection, selectively replicating in cancer cells
and triggering immune response. Phase 2 showing 20%
long-term survivors at 36 months — unprecedented for
GBM.
PHASE 2
|
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Beyond COVID-19 vaccines, mRNA platforms now target influenza, RSV, HIV, and personalised cancer neoantigen vaccines. Moderna and BioNTech have over 40 mRNA programmes in clinical trials as of 2025. Lipid nanoparticle delivery has been optimised for liver, muscle, and tumour tissue targeting.
Circulating tumour DNA (ctDNA) tests like Grail Galleri, Foundation Medicine's FoundationOne Liquid CDx, and Guardant360 detect 50+ cancer types from a single blood draw before symptoms emerge. Multi-cancer early detection (MCED) trials are underway in NHS and US healthcare systems with 75-85% sensitivity at stage I.
Six FDA-approved CAR-T products treat B-cell leukaemias, lymphomas, and multiple myeloma with remission rates of 40-80% in previously terminal patients. Next-generation allogeneic (off-the-shelf) CAR-T from Allogene and Precision BioSciences eliminates the 3-4 week autologous manufacturing delay.
Semaglutide (Ozempic, Wegovy) and tirzepatide (Mounjaro) achieve 15-22% body weight reduction in clinical trials, surpassing bariatric surgery benchmarks. Cardiovascular mortality reduction of 20% confirmed in SELECT trial. Emerging data suggest neuroprotective and addiction-suppressing effects, expanding the addressable indication set dramatically.
Partial cellular reprogramming using Yamanaka factors (OSK or OSKM) reverses epigenetic age markers in animal models. Altos Labs, Retro Biosciences, and Calico are in early human safety studies. Bryan Johnson's Blueprint protocol demonstrates measurable biomarker reversal. Full tissue-specific reprogramming in humans targeted for 2028-2032.
Microfluidic devices lined with human cells replicate organ physiology for drug testing, reducing animal trial dependency. Emulate Bio and CN Bio platforms model liver, lung, gut, and kidney. FDA has accepted organ-chip data as a valid alternative to some animal studies under the FDA Modernization Act 2.0 (2023).
Systematic clearance of senescent cells across all tissue types using precision senolytics (navitoclax variants, CAR-T senolytics) combined with stem cell replenishment and epigenetic reset. Animal studies show lifespan extension of 25-35%. Human translation requires solving tissue-specific delivery, immune modulation, and long-term safety over decades. Timeline: 2035-2050.