Regulatory Approvals

Regulatory milestones achieved by our customers supported
by RES QSP modeling

Regulatory Approvals

Regulatory milestones achieved by our customers supported by RES QSP modeling

Regulatory Approvals

Regulatory milestones achieved by our customers supported by
RES QSP modeling

Title (3-6 words)

PROGRAM

MODALITY

PIPELINE STAGE

LFD-200

Antibody Drug Conjugate

Autoimmune and inflammatory disorders

Lifordi Immunotherapeutics Presents Preclinical Data on Glucocorticoid Antibody Drug Conjugate LFD-200 to Treat Autoimmune and Inflammatory Diseases at EULAR 2025

01 Discovery

02 Pre-Clinical

03 Clinical

LFD-200

Antibody Drug Conjugate

Autoimmune and inflammatory disorders

Lifordi Immunotherapeutics Presents Preclinical Data on Glucocorticoid Antibody Drug Conjugate LFD-200 to Treat Autoimmune and Inflammatory Diseases at EULAR 2025

01 Discovery

02 Pre-Clinical

03 Clinical

LFD-200

Antibody Drug Conjugate

Autoimmune and inflammatory disorders

Lifordi Immunotherapeutics Presents Preclinical Data on Glucocorticoid Antibody Drug Conjugate LFD-200 to Treat Autoimmune and Inflammatory Diseases at EULAR 2025

01 Discovery

02 Pre-Clinical

03 Clinical

SGT-501

Gene therapy

CPVT

FDA IND and Health Canada CTA Approval for First-in-Class Cardiac Gene Therapy to Treat Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

01 Discovery

02 Pre-Clinical

03 Clinical (IND)

SGT-501

Gene therapy

CPVT

FDA IND and Health Canada CTA Approval for First-in-Class Cardiac Gene Therapy to Treat Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

01 Discovery

02 Pre-Clinical

03 Clinical (IND)

SGT-501

Gene therapy

CPVT

FDA IND and Health Canada CTA Approval for First-in-Class Cardiac Gene Therapy to Treat Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

01 Discovery

02 Pre-Clinical

03 Clinical (IND)

SGT-212

Gene therapy

Friedreich’s Ataxia

FDA clearance of IND application for Solid Bio’s SGT-212, a gene therapy to treat Friedreich’s Ataxia. January 7, 2025.

01 Discovery

02 Pre-Clinical

03 Clinical (IND)

SGT-212

Gene therapy

Friedreich’s Ataxia

FDA clearance of IND application for Solid Bio’s SGT-212, a gene therapy to treat Friedreich’s Ataxia. January 7, 2025.

01 Discovery

02 Pre-Clinical

03 Clinical (IND)

SGT-212

Gene therapy

Friedreich’s Ataxia

FDA clearance of IND application for Solid Bio’s SGT-212, a gene therapy to treat Friedreich’s Ataxia. January 7, 2025.

01 Discovery

02 Pre-Clinical

03 Clinical (IND)

STAR-0310

Monoclonal antibody

Atopic Dermatitis

FDA clearance of IND application for Astria’s STAR-0310, a monoclonal antibody OX40 antagonist for the treatment of atopic dermatitis.

01 Discovery

02 Pre-Clinical

03 Clinical (Phase I/II)

STAR-0310

Monoclonal antibody

Atopic Dermatitis

FDA clearance of IND application for Astria’s STAR-0310, a monoclonal antibody OX40 antagonist for the treatment of atopic dermatitis.

01 Discovery

02 Pre-Clinical

03 Clinical (Phase I/II)

STAR-0310

Monoclonal antibody

Atopic Dermatitis

FDA clearance of IND application for Astria’s STAR-0310, a monoclonal antibody OX40 antagonist for the treatment of atopic dermatitis.

01 Discovery

02 Pre-Clinical

03 Clinical (PI/II)

BE-101

Cell therapy (Engineered B-cell)

Hemophilia B

FDA clearance of IND application for Be Biopharma’s BE-101, engineered B cell medicine for the treatment of Hemophilia B.

01 Discovery

02 Pre-Clinical

03 Clinical (IND)

BE-101

Cell therapy (Engineered B-cell)

Hemophilia B

FDA clearance of IND application for Be Biopharma’s BE-101, engineered B cell medicine for the treatment of Hemophilia B.

01 Discovery

02 Pre-Clinical

03 Clinical (IND)

BE-101

Cell therapy (Engineered B-cell)

Hemophilia B

FDA clearance of IND application for Be Biopharma’s BE-101, engineered B cell medicine for the treatment of Hemophilia B.

01 Discovery

02 Pre-Clinical

03 Clinical (IND)

STAR-0215

Monoclonal antibody

Hereditary Angioedema

FDA clearance for Phase 1b/2 trial of Astria’s Navenibart (STAR-0215), a monoclonal antibody inhibitor of plasma kallikrein for the treatment of hereditary angioedema.

01 Discovery

02 Pre-Clinical

03 Clinical (Phase I/II)

STAR-0215

Monoclonal antibody

Hereditary Angioedema

FDA clearance for Phase 1b/2 trial of Astria’s Navenibart (STAR-0215), a monoclonal antibody inhibitor of plasma kallikrein for the treatment of hereditary angioedema.

01 Discovery

02 Pre-Clinical

03 Clinical (Phase I/II)

STAR-0215

Monoclonal antibody

Hereditary Angioedema

FDA clearance for Phase 1b/2 trial of Astria’s Navenibart (STAR-0215), a monoclonal antibody inhibitor of plasma kallikrein for the treatment of hereditary angioedema.

01 Discovery

02 Pre-Clinical

03 Clinical (PI/II)

TAK-861

Small molecule

Narcolepsy

FDA clearance for Phase 3 trial of Takeda’s TAK-861, a small molecule inhibitor therapy for treatment of narcolepsy.

01 Discovery

02 Pre-Clinical

03 Clinical (Phase I/II/III)

TAK-861

Small molecule

Narcolepsy

FDA clearance for Phase 3 trial of Takeda’s TAK-861, a small molecule inhibitor therapy for treatment of narcolepsy.

01 Discovery

02 Pre-Clinical

03 Clinical (Phase I/II/III)

TAK-861

Small molecule

Narcolepsy

FDA clearance for Phase 3 trial of Takeda’s TAK-861, a small molecule inhibitor therapy for treatment of narcolepsy.

01 Discovery

02 Pre-Clinical

03 Clinical (PI/II/III)

SGT-003

Gene therapy

Duchenne Muscular Dystrophy

FDA clearance of IND application for Solid Bio’s SGT-003, a gene therapy for Duchenne Muscular Dystrophy.

01 Discovery

02 Pre-Clinical

03 Clinical (IND)

SGT-003

Gene therapy

Duchenne Muscular Dystrophy

FDA clearance of IND application for Solid Bio’s SGT-003, a gene therapy for Duchenne Muscular Dystrophy.

01 Discovery

02 Pre-Clinical

03 Clinical (IND)

SGT-003

Gene therapy

Duchenne Muscular Dystrophy

FDA clearance of IND application for Solid Bio’s SGT-003, a gene therapy for Duchenne Muscular Dystrophy.

01 Discovery

02 Pre-Clinical

03 Clinical (IND)

ADZYNMA (ADAMTS13)

Recombinant Protein

cTTP

FDA approval of Takeda’s ADZYNMA (ADAMTS13, recombinant-krhn), the first and only FDA-approved recombinant protein designed to address an unmet medical need in people with cTTP by replacing the deficient ADAMTS13 enzyme.

01 Discovery

02 Pre-Clinical

03 Clinical (Phase III)

ADZYNMA (ADAMTS13)

Recombinant Protein

cTTP

FDA approval of Takeda’s ADZYNMA (ADAMTS13, recombinant-krhn), the first and only FDA-approved recombinant protein designed to address an unmet medical need in people with cTTP by replacing the deficient ADAMTS13 enzyme.

01 Discovery

02 Pre-Clinical

03 Clinical (Phase III)

ADZYNMA (ADAMTS13)

Recombinant Protein

cTTP

FDA approval of Takeda’s ADZYNMA (ADAMTS13, recombinant-krhn), the first and only FDA-approved recombinant protein designed to address an unmet medical need in people with cTTP by replacing the deficient ADAMTS13 enzyme.

01 Discovery

02 Pre-Clinical

03 Clinical (PIII)

M9140

Antibody Drug Conjugate

Advanced Solid Tumors

FDA clearance for Phase I trial for EMD Serono’s M9140, an anti-CEACAM5 Antibody Drug Conjugate for the treatment of advanced solid tumors.

01 Discovery

02 Pre-Clinical

03 Clinical (Phase I/II/III)

M9140

Antibody Drug Conjugate

Advanced Solid Tumors

FDA clearance for Phase I trial for EMD Serono’s M9140, an anti-CEACAM5 Antibody Drug Conjugate for the treatment of advanced solid tumors.

01 Discovery

02 Pre-Clinical

03 Clinical (Phase I/II/III)

M9140

Antibody Drug Conjugate

Advanced Solid Tumors

FDA clearance for Phase I trial for EMD Serono’s M9140, an anti-CEACAM5 Antibody Drug Conjugate for the treatment of advanced solid tumors.

01 Discovery

02 Pre-Clinical

03 Clinical (PI/II/III)