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Gain Therapeutics, Inc.
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.
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
Mustang Bio, Inc.
Gene and CAR T Cell Therapies
Mustang Bio, Inc. is a biopharmaceutical company specializing in cell and gene therapies for hematologic cancers, solid tumors, and rare genetic diseases. It focuses on developing gene therapy programs and CAR T cell therapies, including treatments for X-linked severe combined immunodeficiency and various types of cancer.
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Symbol: MBIO
Recent Price: $0.19
Industry: Biotechnology
CEO: Dr. Manuel Litchman M.D.
Sector: Healthcare
Employees: 80
Address: 377 Plantation Street, Worcester, MA 01605
Phone: 781 652 4500
Leadership
- Manuel Litchman, M.D., President, Chief Executive Officer and Director
- Greg Furrow, M.S., FRQA, Chief Quality Officer
- James Murphy, B.A., Ad interim Chief Financial Officer
- Michael S. Weiss, Chairman of the Board of Directors
- Lindsay A. Rosenwald, M.D., Director
- Adam J. Chill, Director
- Neil Herskowitz, Director
- Michael J. Zelefsky, M.D., Director
- Mustang Bio, Inc., Company
Last updated: 2024-12-31
Reneo Pharmaceuticals, Inc.
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.
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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.
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.
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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
Taysha Gene Therapies, Inc.
TSHA-120, TSHA-102, TSHA-121, TSHA-118, TSHA-105, TSHA-101
Taysha Gene Therapies, Inc. focuses on developing adeno-associated virus-based gene therapies for treating monogenic diseases of the central nervous system, with a pipeline including treatments for conditions such as giant axonal neuropathy, Rett syndrome, and GM2 gangliosidosis.
Symbol: TSHA
Recent Price: $1.73
Industry: Biotechnology
CEO: Mr. Sean P. Nolan
Sector: Healthcare
Employees: 52
Address: 3000 Pegasus Park Drive, Dallas, TX 75247
Phone: 214 612 0000
Last updated: 2024-12-31
Beam Therapeutics Inc.
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.
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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.
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.
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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
Eloxx Pharmaceuticals, Inc.
ELX-02
Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing ribosome modulation therapies for rare and ultra-rare premature stop codon diseases. The company's lead investigational drug, ELX-02, is in Phase 2 clinical trials for cystic fibrosis and nephropathic cystinosis.
Symbol: ELOX
Recent Price: $0.00
Industry: Biotechnology
CEO: Mr. Sumit Aggarwal M.B.A.
Sector: Healthcare
Employees: 18
Address: 480 Arsenal Way, Watertown, MA 02472
Phone: 781 577 5300
Last updated: 2024-12-31
Fortress Biotech, Inc.
Pharmaceutical and Biotechnology Products
Fortress Biotech, Inc., is a biopharmaceutical company focused on developing and commercializing a range of dermatology products and late-stage product candidates for various medical conditions.
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Symbol: FBIO
Recent Price: $2.18
Industry: Biotechnology
CEO: Dr. Lindsay Allan Rosenwald
Sector: Healthcare
Employees: 186
Address: 1111 Kane Concourse, Bay Harbor Islands, FL 33154
Phone: 781 652 4500
Leadership
- Lindsay A. Rosenwald, M.D., Chairman, President and Chief Executive Officer
- Michael S. Weiss, Executive Vice Chairman, Strategic Development
- David Jin, Chief Financial Officer and Head of Corporate Development
- George Avgerinos, Ph.D., Senior Vice President, Biologics Operations
- Samuel Berry, General Counsel and Corporate Secretary
- Jimmie Harvey, Jr., M.D., Director
- Malcolm Hoenlein, Director
- Dov Klein, Director
- J. Jay Lobell, Director
- Kevin L. Lorenz, J.D., Director
- Lucy Lu, M.D., Director
Last updated: 2024-12-31
4D Molecular Therapeutics, Inc.
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.
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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
Inozyme Pharma, Inc.
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.
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
Neurogene Inc.
NGN-401, NGN-101
Neurogene Inc. develops life-changing genetic medicines for patients and their families affected by neurological diseases, focusing on gene therapy products like NGN-401 for Rett syndrome and NGN-101 for neuronal ceroid lipofuscinosis subtype 5 batten disease.
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Symbol: NGNE
Recent Price: $22.28
Industry: Biotechnology
CEO: Dr. Rachel L. McMinn Ph.D.
Sector: Healthcare
Employees: 91
Address: 535 W 24th Street, New York, NY 10011
Phone: (877) 237-5020
Last updated: 2024-12-31
Nurix Therapeutics, Inc.
NX-2127, NX-5948, NX-1607, NX-0255
Nurix Therapeutics, Inc. is a biopharmaceutical company that focuses on developing small molecule therapies for cancer and immune disorders, with products targeting BTK and CBL-B proteins. The company collaborates with Gilead Sciences and Sanofi.
Symbol: NRIX
Recent Price: $18.64
Industry: Biotechnology
CEO: Dr. Arthur T. Sands M.D., Ph.D.
Sector: Healthcare
Employees: 284
Address: 1700 Owens Street, San Francisco, CA 94158
Phone: 415 660 5320
Last updated: 2024-12-31
PTC Therapeutics, Inc.
Translarna, Emflaza, Tegsedi, Waylivra, Evrysdi
PTC Therapeutics, Inc. is a biopharmaceutical company dedicated to discovering, developing, and commercializing medicines for patients with rare disorders. The company offers a portfolio of products including treatments for Duchenne muscular dystrophy, rare diseases, and spinal muscular atrophy.
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Symbol: PTCT
Recent Price: $45.77
Industry: Biotechnology
CEO: Dr. Matthew B. Klein F.A.C.S., M.D., M.S.
Sector: Healthcare
Employees: 988
Address: 100 Corporate Court, South Plainfield, NJ 07080
Phone: 908 222 7000
Leadership
- Matthew B. Klein, M.D., M.S., F.A.C.S., CEO, PTC Therapeutics, Inc.
- Neil Almstead, Ph.D., Chief Technical Operations Officer
- John Baird, Ph.D., Chief of Staff to the CEO
- Mark E. Boulding, Executive Vice President and Chief Legal Officer
- Lee Golden, M.D., Chief Medical Officer
- Pierre Gravier, Chief Financial Officer
- Mary Frances Harmon, Senior Vice President, Corporate and Patient Relations
- Linda Montella-Carter, Senior Vice President and Chief Information Officer
- Eric Pauwels, Chief Business Officer
- Hege Sollie-Zetlmayer, Chief Human Resources Officer
- Christine Utter, Senior Vice President, Chief Accounting Officer and Head of People Services
- Michael Schmertzler, Chairman
- William F. Bell, Jr., Managing Director and Head of Healthcare Services Practice, L.E.K. Consulting
- Allan Jacobson, Ph.D., University of Massachusetts Chan Medical School
- Stephanie S. Okey, M.S., Former SVP, Head of North America, Rare Disease – Genzyme
- Emma Reeve, Independent Board Director
- Mary Smith, The VENG Group
- David P. Southwell, Former CEO, TScan Therapeutics
- Glenn D. Steele, Jr., M.D., Ph.D., Chairman, GSteele Health Solutions
- Alethia Young, Chief Financial Officer, Bicycle Therapeutics
- Jerome B. Zeldis, M.D., Ph.D., Independent Board Director
Last updated: 2024-12-31
Quince Therapeutics, Inc.
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.
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.
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.
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
Rhythm Pharmaceuticals, Inc.
IMCIVREE
Rhythm Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company dedicated to developing and commercializing treatments for rare genetic diseases of obesity. Their lead product, IMCIVREE, targets specific genetic disorders causing obesity, and they are also advancing other therapeutic candidates through clinical trials.
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Symbol: RYTM
Recent Price: $56.34
Industry: Biotechnology
CEO: Dr. David P. Meeker M.D.
Sector: Healthcare
Employees: 226
Address: 222 Berkeley Street, Boston, MA 02116
Phone: 857 264 4280
Last updated: 2024-12-31
Abeona Therapeutics Inc.
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.
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.
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.
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
Ionis Pharmaceuticals, Inc.
RNA-targeted therapeutics
Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States, offering treatments like SPINRAZA, TEGSEDI, and WAYLIVRA, while developing medicines in phase 3 studies for various indications including TTR amyloidosis, severe hypertriglyceridemia, and amyotrophic lateral sclerosis.
About | About | About | DEI | DEI | DEI | DEI | Contact | Contact | Careers | Careers | Careers | Careers | Careers | Careers | About | About | News | About | About | ESG | DEI | DEI | About | About | About | Management | About | About | About | About | About | About | About | About | About | About | Careers | Careers | Careers | Careers | Careers | ESG | Careers | Careers | Careers | Careers | Careers | Careers | Careers | Careers | Careers | Careers | Careers | Careers | Careers | Careers | Careers | Careers | Careers | About | About | About | About | About | About | About | Jobs | About | About
Symbol: IONS
Recent Price: $34.92
Industry: Biotechnology
CEO: Dr. Brett P. Monia Ph.D.
Sector: Healthcare
Employees: 927
Address: 2855 Gazelle Court, Carlsbad, CA 92010
Phone: 760 931 9200
Last updated: 2024-12-31