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Gain Therapeutics, Inc.

Gain Therapeutics, Inc. logo
Market Cap: Lowest
Employees: Lowest

Site-Directed Enzyme Enhancement Therapy platform

Gain Therapeutics, Inc. is a biotechnology company developing therapies for diseases caused by protein misfolding, focusing on rare genetic diseases and neurological disorders. It utilizes its proprietary technology to discover and develop molecules targeting misfolded proteins.

Tags: allosteric regulation, biotechnology, neurological disorders, protein misfolding, rare genetic diseases

Symbol: GANX

Recent Price: $2.18

Industry: Biotechnology

CEO: Mr. Gene Mack M.B.A.

Sector: Healthcare

Employees: 29

Address: 4800 Montgomery Lane, Bethesda, MD 20814

Phone: 301 500 1556

Last updated: 2024-12-31

Capricor Therapeutics, Inc.

Capricor Therapeutics, Inc. logo
Market Cap: Medium
Employees: Low

CAP-1002

Capricor Therapeutics, Inc. is a clinical-stage biotechnology company specializing in developing transformative cell and exosome-based therapeutics aimed at treating and preventing a range of diseases and disorders. Their lead candidate, CAP-1002, is designed for Duchenne muscular dystrophy and cytokine storm interventions.

Tags: COVID-19, Duchenne muscular dystrophy, biotechnology, cell therapy, therapeutics

Symbol: CAPR

Recent Price: $13.76

Industry: Biotechnology

CEO: Dr. Linda Marbán Ph.D.

Sector: Healthcare

Employees: 101

Address: 10865 Road to the Cure, San Diego, CA 92121

Phone: 310 358 3200

Leadership

  • Linda Marbán, Ph.D., Chief Executive Officer and Director
  • Kristi A. H. Elliott, Ph.D., Chief Science Officer
  • AJ Bergmann, M.B.A., Chief Financial Officer
  • Karen G. Krasney, J.D., Executive Vice President and General Counsel
  • Mark Awadalla, Vice President of Clinical Operations
  • Jonathan Tayco, Vice President of Program Management and Business Operations
  • Minghao Sun, Ph.D., Vice President of Research and Product Development
  • Yushi Feng, Ph.D., Vice President of Regulatory Affairs
  • Frank Litvack M.D., FACC, Executive Chairman
  • Paul G. Auwaerter, M.D., M.B.A., FIDSA, Director
  • Earl M. (Duke) Collier Jr., Director
  • George W. Dunbar, Jr., Director
  • Philip J. Gotwals, Ph.D., Director
  • Michael Kelliher, Director
  • David B. Musket, Director
  • Karimah Es Sabar, Director

Last updated: 2024-12-31

Cyclerion Therapeutics, Inc.

Cyclerion Therapeutics, Inc. logo
Market Cap: Lowest
Employees: Lowest

CY6463

Cyclerion Therapeutics is a clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing medicines for serious CNS diseases, with products like CY6463 for mitochondrial encephalomyopathy and Alzheimer's disease.

Tags: CNS diseases, CY6463, biopharmaceutical, clinical-stage, therapy development

Symbol: CYCN

Recent Price: $3.45

Industry: Biotechnology

CEO: Dr. Regina Graul Ph.D.

Sector: Healthcare

Employees: 1

Address: 245 First Street, Cambridge, MA 02142

Phone: 857 327 8778

Last updated: 2024-12-31

Dyne Therapeutics, Inc.

Dyne Therapeutics, Inc. logo
Market Cap: High
Employees: Low

FORCE platform

Dyne Therapeutics, Inc. is a biotechnology company focused on advancing therapeutics for genetically driven muscle diseases, including myotonic dystrophy type 1, duchenne muscular dystrophy, and facioscapulohumeral dystrophy, using its FORCE platform.

Tags: biotechnology, duchenne muscular dystrophy, facioscapulohumeral dystrophy, genetic therapy, muscle diseases, myotonic dystrophy type 1, nucleic acid therapies

Symbol: DYN

Recent Price: $23.65

Industry: Biotechnology

CEO: Mr. John G. Cox M.B.A.

Sector: Healthcare

Employees: 152

Address: 1560 Trapelo Road, Waltham, MA 02451

Phone: 781 786 8230

Last updated: 2024-12-31

Catalyst Pharmaceuticals, Inc.

Catalyst Pharmaceuticals, Inc. logo
Market Cap: High
Employees: Low

Firdapse

Catalyst Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company focused on developing and commercializing therapies for rare neuromuscular and neurological diseases in the United States, including products like Firdapse and Ruzurgi for Lambert-Eaton Myasthenic Syndrome.

Tags: Firdapse, Ruzurgi, biopharmaceutical, neurological diseases, neuromuscular diseases, rare diseases

Symbol: CPRX

Recent Price: $21.07

Industry: Biotechnology

CEO: Mr. Richard John Daly M.B.A.

Sector: Healthcare

Employees: 167

Address: 355 Alhambra Circle, Coral Gables, FL 33134

Phone: 305 420 3200

Last updated: 2024-12-31

Reneo Pharmaceuticals, Inc.

Reneo Pharmaceuticals, Inc. logo
Market Cap: Lowest
Employees: Lowest

REN001

Reneo Pharmaceuticals, Inc., a clinical-stage pharmaceutical company, focuses on developing therapies for patients with rare genetic mitochondrial diseases. Its primary product, REN001, is a selective peroxisome proliferator-activated receptor delta agonist currently in clinical trials.

Tags: Clinical Trials, Genetic Disorders, Mitochondrial Diseases, Pharmaceuticals, Therapeutics

Symbol: RPHM

Recent Price: $18.20

Industry: Biotechnology

CEO: Mr. Gregory J. Flesher

Sector: Healthcare

Employees: 8

Address: 18575 Jamboree Road, Irvine, CA 92612

Phone: 619 733 3852

Last updated: 2024-12-31

Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc. logo
Market Cap: Highest
Employees: Medium

EXONDYS 51, VYONDYS 53, AMONDYS 45, SRP-5051, SRP-9001, SRP-9003

Sarepta Therapeutics, Inc. is a biopharmaceutical company focused on developing RNA-targeted therapeutics and gene therapies for rare diseases, including treatments for Duchenne muscular dystrophy.

Tags: Duchenne muscular dystrophy, RNA-targeted therapeutics, biopharmaceutical, gene therapy, rare diseases

Symbol: SRPT

Recent Price: $121.19

Industry: Biotechnology

CEO: Mr. Douglas S. Ingram Esq.

Sector: Healthcare

Employees: 1314

Address: 215 First Street, Cambridge, MA 02142

Phone: 617 274 4000

Leadership

  • Douglas S. Ingram, President and Chief Executive Officer
  • Bilal Arif, Executive Vice President, Chief Technical Operations Officer
  • Diane Berry, PhD, Executive Vice President, Chief Global Policy & Advocacy Officer
  • Ryan E. Brown, JD, Executive Vice President, Chief General Counsel
  • Ian M. Estepan, Executive Vice President, Chief Financial Officer
  • Dallan Murray, Executive Vice President, Chief Customer Officer
  • Alison Nasisi, Executive Vice President, Chief People Officer
  • Louise R. Rodino-Klapac, PhD, Executive Vice President, Head of R&D, Chief Scientific Officer
  • Will Tilton, Senior Vice President, Head of Strategy, Chief of Staff
  • M. Kathleen Behrens, PhD, Chairwoman
  • Richard J. Barry,
  • Kathryn Boor, PhD,
  • Michael Chambers,
  • Deirdre Connelly,
  • Stephen L. Mayo, PhD,
  • Claude Nicaise, MD,
  • Hans Wigzell, MD, PhD,
  • John C. Martin, PhD (In Memoriam),
  • Annemieke Aartsma-Rus, PhD,
  • Carsten Bönnemann, MD,
  • Joy Cavagnaro, PhD, DABT, Fellow ATS,
  • Kay Davies, PhD, DBE FMedSci FRS,
  • Steven Gray, PhD,
  • Angela J. Russell, DPhil,

Last updated: 2024-12-31

Beam Therapeutics Inc.

Beam Therapeutics Inc. logo
Market Cap: High
Employees: Medium

precision genetic medicines

Beam Therapeutics Inc., a biotechnology company, develops precision genetic medicines for patients suffering from serious diseases in the United States, with a focus on conditions such as sickle cell disease, beta thalassemia, and Glycogen Storage Disease Type Ia.

Tags: Glycogen Storage Disease Type Ia, beta thalassemia, biotechnology, collaboration, genetic medicine, sickle cell disease

Symbol: BEAM

Recent Price: $25.28

Industry: Biotechnology

CEO: Mr. John M. Evans M.B.A.

Sector: Healthcare

Employees: 472

Address: 238 Main Street, Cambridge, MA 02142

Phone: 857 327 8775

Last updated: 2024-12-31

Design Therapeutics, Inc.

Design Therapeutics, Inc. logo
Market Cap: Low
Employees: Lowest

Gene TAC

Design Therapeutics, Inc. is a preclinical-stage biopharmaceutical company developing therapies for genetic diseases caused by nucleotide repeat expansions. Their portfolio includes products targeting Friedreich Ataxia and Myotonic Dystrophy Type-1, as well as Gene TAC candidates for other similar diseases.

Tags: Friedreich Ataxia, Gene TAC, Myotonic Dystrophy Type-1, biopharmaceutical, genetic diseases, nucleotide repeat expansions, preclinical-stage, therapy development

Symbol: DSGN

Recent Price: $6.17

Industry: Biotechnology

CEO: Mr. Pratik Shah Ph.D.

Sector: Healthcare

Employees: 57

Address: 6005 Hidden Valley Road, Carlsbad, CA 92011

Phone: 858 293 4900

Last updated: 2024-12-31

Dyadic International, Inc.

Dyadic International, Inc. logo
Market Cap: Lowest
Employees: Lowest

C1 platform

Dyadic International, Inc. is a biotechnology platform company that develops, produces, and sells enzymes and other proteins, focusing on the development and manufacturing of vaccines and drugs using its proprietary C1 platform.

Tags: C1 platform, biotechnology, drug development, enzymes, proteins, vaccines

Symbol: DYAI

Recent Price: $1.78

Industry: Biotechnology

CEO: Mr. Mark A. Emalfarb

Sector: Healthcare

Employees: 7

Address: 140 Intracoastal Pointe Drive, Jupiter, FL 33477-5094

Phone: 561 743 8333

Leadership

  • Patrick Lucy, Chairman of the Board, President and Chief Executive Officer
  • Mark A. Emalfarb, Founder, Chief Executive Officer
  • Seth J. Herbst, MD, Chairman of the Nominating Committee
  • Jack L. Kaye, Chairman of the Audit Committee
  • Arindam Bose, Chairman of the Science and Technology Committee
  • Michael P. Tarnok, Chairman of the Board

Last updated: 2024-12-31

4D Molecular Therapeutics, Inc.

4D Molecular Therapeutics, Inc. logo
Market Cap: Medium
Employees: Low

4D-125, 4D-110, 4D-310, 4D-150, 4D-710

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company developing product candidates using adeno-associated virus vectors, focusing on ophthalmology, cardiology, and pulmonology.

Tags: biotechnology, cardiology, gene therapy, ophthalmology, pulmonology

Symbol: FDMT

Recent Price: $5.30

Industry: Biotechnology

CEO: Dr. David H. Kirn M.D.

Sector: Healthcare

Employees: 201

Address: 5858 Horton Street, EmeryVille, CA 94608

Phone: 510 505 2680

Last updated: 2024-12-31

FibroGen, Inc.

FibroGen, Inc. logo
Market Cap: Lowest
Employees: Medium

Roxadustat, Pamrevlumab

Fibro Gen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing therapeutics to treat serious unmet medical needs, including treatments for anemia, idiopathic pulmonary fibrosis, and Duchenne muscular dystrophy.

Tags: anemia, antibody, biopharmaceutical, cancer, kidney disease, pulmonary fibrosis, therapeutics

Symbol: FGEN

Recent Price: $0.53

Industry: Biotechnology

CEO: Mr. Thane Wettig

Sector: Healthcare

Employees: 486

Address: 409 Illinois Street, San Francisco, CA 94158

Phone: 415 978 1200

Last updated: 2024-12-31

Inozyme Pharma, Inc.

Inozyme Pharma, Inc. logo
Market Cap: Medium
Employees: Lowest

INZ-701

Inozyme Pharma, Inc. is a biopharmaceutical company focused on the development of therapeutics for rare disease conditions involving abnormal mineralization, particularly targeting ENPP1 and ABCC6 deficiencies.

Tags: ABCC6, ENPP1, INZ-701, biopharmaceutical, calciphylaxis, genetic diseases, mineralization, rare diseases, therapeutics

Symbol: INZY

Recent Price: $2.82

Industry: Biotechnology

CEO: Dr. Douglas A. Treco Ph.D.

Sector: Healthcare

Employees: 59

Address: 321 Summer Street, Boston, MA 02210

Phone: 857 330 4340

Leadership

  • Doug Treco, Ph.D., CEO, Chairman of Board of Directors
  • Gayle Gironda, Senior Vice President and Chief People Officer
  • Kurt Gunter, M.D., Senior Vice President and Chief Medical Officer
  • Soojin Kim, Ph.D., Senior Vice President and Chief Technical Operations Officer
  • Yves Sabbagh, Ph.D., Senior Vice President and Chief Scientific Officer, Chair of the Scientific Advisory Board
  • Sanjay S. Subramanian, M.S., MBA, Senior Vice President and Chief Financial Officer
  • Matt Winton, Ph.D., Senior Vice President and Chief Operating Officer
  • Axel Bolte, MBA, Co-founder, Senior Advisor and Member of Board of Directors
  • David Thompson, M.A., M.S., Ph.D., Senior Advisor, Member of Scientific Advisory Board
  • Sarah Bhagat, Ph.D., Director
  • Reinaldo M. Diaz, MBA, Director
  • Martin Edwards M.D., MBA, Director
  • Erik Harris, MBA, Director
  • Rob Hopfner, Rph, Ph.D., MBA, Director
  • Edward Mathers, Director
  • Lynne Sullivan, MST, Director

Last updated: 2024-12-31

Omega Therapeutics, Inc.

Omega Therapeutics, Inc. logo
Market Cap: Lowest
Employees: Low

OMEGA Epigenomic Programming platform

Omega Therapeutics, Inc. is a development-stage biopharmaceutical company leveraging its OMEGA Epigenomic Programming platform to utilize the power of epigenetics for therapeutic interventions across various diseases.

Tags: biopharmaceutical, chronic liver diseases, corneal regeneration, epigenetics, gene control, idiopathic pulmonary fibrosis

Symbol: OMGA

Recent Price: $0.80

Industry: Biotechnology

CEO: Mr. Mahesh Karande

Sector: Healthcare

Employees: 93

Address: 20 Acorn Park Drive, Cambridge, MA 02140

Phone: 617 949 4360

Leadership

  • Mahesh Karande, President & Chief Executive Officer
  • Kaan Certel, Ph.D., Chief Business Officer
  • Jennifer Nelson, Ph.D., Senior Vice President, Research
  • Tony Mullin, Chief People Officer
  • Barbara Chan, Senior Vice President, Finance and Chief Accounting Officer
  • Lisamarie Fahy, Senior Vice President, Clinical Development Operations
  • Joe Newman, Ph.D., Senior Vice President, Early Discovery
  • Jeff Atkinson, Ph.D., Vice President, Technical Operations
  • Eva Stroynowski, Senior Vice President, Investor Relations and Corporate Affairs

Last updated: 2024-12-31

Quince Therapeutics, Inc.

Quince Therapeutics, Inc. logo
Market Cap: Lowest
Employees: Lowest

NOV004

Quince Therapeutics, Inc., a biopharmaceutical company, focuses on advancing precision therapeutics for debilitating and rare diseases, with a bone-targeting drug platform and lead compound NOV004 designed to target bone fracture and disease sites.

Tags: NOV004, biopharmaceutical, biotechnology, bone-targeting, precision therapeutics, rare diseases

Symbol: QNCX

Recent Price: $1.85

Industry: Biotechnology

CEO: Dr. Dirk Thye M.D.

Sector: Healthcare

Employees: 32

Address: 601 Gateway Blvd, South San Francisco, CA 94080

Phone: 415 910 5717

Last updated: 2024-12-31

Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. logo
Market Cap: High
Employees: Low

Gene Therapies

Rocket Pharmaceuticals, Inc. is a biotechnology company focused on developing gene therapies for rare and devastating diseases, with multiple clinical-stage programs targeting disorders such as fanconi anemia, leukocyte adhesion deficiency-I, pyruvate kinase deficiency, and Danon disease.

Tags: Biotechnology, Clinical Stage, Gene Therapy, Healthcare, Rare Diseases

Symbol: RCKT

Recent Price: $12.03

Industry: Biotechnology

CEO: Dr. Gaurav D. Shah M.D.

Sector: Healthcare

Employees: 268

Address: 9 Cedarbrook Drive, Cranbury, NJ 08512

Phone: 609 659 8001

Last updated: 2024-12-31

Entrada Therapeutics, Inc.

Entrada Therapeutics, Inc. logo
Market Cap: Medium
Employees: Low

ENTR-601-44

Entrada Therapeutics, Inc. develops endosomal escape vehicle (EEV) therapeutics for treating neuromuscular diseases, with a lead product candidate ENTR-601-44 in preclinical trials for Duchenne muscular dystrophy and myotonic dystrophy type 1.

Tags: Duchenne muscular dystrophy, EEV therapeutics, biotechnology, myotonic dystrophy type 1, neuromuscular diseases

Symbol: TRDA

Recent Price: $17.37

Industry: Biotechnology

CEO: Mr. Dipal Doshi

Sector: Healthcare

Employees: 168

Address: 6 Tide Street, Boston, MA 02210

Phone: 857 520 9158

Last updated: 2024-12-31

VYNE Therapeutics Inc.

VYNE Therapeutics Inc. logo
Market Cap: Lowest
Employees: Lowest

FCD105, FMX114, VYN201, VYN202

VYNE Therapeutics Inc. is a pharmaceutical company specializing in developing therapeutics for immuno-inflammatory conditions. Key products include FCD105 for acne vulgaris, FMX114 for atopic dermatitis, and BET inhibitors VYN201 and VYN202 for skin and other immuno-inflammatory diseases.

Tags: BET inhibitors, acne vulgaris, atopic dermatitis, immuno-inflammatory, pharmaceutical, therapeutics

Symbol: VYNE

Recent Price: $3.19

Industry: Biotechnology

CEO: Mr. David T. Domzalski

Sector: Healthcare

Employees: 10

Address: 520 U.S. Highway 22, Bridgewater, NJ 08807

Phone: 800 775 7936

Last updated: 2024-12-31

Abeona Therapeutics Inc.

Abeona Therapeutics Inc. logo
Market Cap: Low
Employees: Low

EB-101

Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company, develops gene and cell therapies for life-threatening rare genetic diseases. Its lead program is EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa.

Tags: EB-101, biopharmaceutical, cell therapy, clinical trials, gene therapy, rare genetic diseases

Symbol: ABEO

Recent Price: $5.61

Industry: Biotechnology

CEO: Dr. Vishwas Seshadri M.B.A., Ph.D.

Sector: Healthcare

Employees: 84

Address: 1330 Avenue of the Americas, New York, NY 10019

Phone: 646 813 4701

Last updated: 2024-12-31

Arcturus Therapeutics Holdings Inc.

Arcturus Therapeutics Holdings Inc. logo
Market Cap: Medium
Employees: Low

RNA medicines

Arcturus Therapeutics Holdings Inc. focuses on developing vaccines for infectious and rare diseases, including programs for cystic fibrosis and ornithine transcarbamylase deficiency. The company collaborates with various partners for vaccine and therapeutic development.

Tags: RNA medicines, collaborations, infectious diseases, rare diseases, vaccines

Symbol: ARCT

Recent Price: $16.58

Industry: Biotechnology

CEO: Mr. Joseph E. Payne M.Sc.

Sector: Healthcare

Employees: 180

Address: 10628 Science Center Drive, San Diego, CA 92121

Phone: 858 900 2660

Last updated: 2024-12-31