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CPhI India 2024CPhI India 2024
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26-28 November, 2024
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11-14 November, 2024
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11-13 November, 2024
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CPhI India 2024CPhI India 2024
Industry Trade Show
Not Confirmed
26-28 November, 2024
Industry Trade Show
Not Confirmed
11-14 November, 2024
The Pharmacy Technolog...The Pharmacy Technology
Industry Trade Show
Not Confirmed
11-13 November, 2024
Digital content
INTERVIEW #SpeakPharma
[Sponsored by another company]https://www.pharmacompass.com/speak-pharma/lgm-pharma-has-made-significant-strides-in-meeting-the-evolving-needs-of-its-clients
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09 Nov 2024
// GLOBENEWSWIRE
https://www.globenewswire.com/news-release/2024/11/09/2977959/20739/en/Ultragenyx-Presents-Positive-Update-on-GTX-102-Angelman-Syndrome-Program-at-FAST-s-17th-Annual-Global-Science-Summit.html
05 Nov 2024
// GLOBENEWSWIRE
https://www.globenewswire.com/news-release/2024/11/05/2975192/20739/en/Ultragenyx-Reports-Third-Quarter-2024-Financial-Results-and-Corporate-Update.html
29 Oct 2024
// GLOBENEWSWIRE
https://www.globenewswire.com/news-release/2024/10/29/2971208/20739/en/Ultragenyx-to-Host-Conference-Call-for-Third-Quarter-2024-Financial-Results-and-Corporate-Update.html
18 Oct 2024
// GLOBENEWSWIRE
https://www.globenewswire.com/news-release/2024/10/18/2965735/20739/en/Ultragenyx-Reports-Inducement-Grant-Under-Nasdaq-Listing-Rule-5635-c-4.html
07 Oct 2024
// GLOBENEWSWIRE
https://www.globenewswire.com/news-release/2024/10/07/2958870/20739/en/Ultragenyx-Receives-Breakthrough-Therapy-Designation-for-Setrusumab-UX143-in-Osteogenesis-Imperfecta.html
04 Oct 2024
// FIERCE BIOTECH
https://www.fiercebiotech.com/biotech/ultragenyx-tweaks-gene-therapy-dosing-dial-efficacy-after-seeing-signs-activity
Details:
UX143 (setrusumab), is a sclerostin inhibitor antibody drug candidate, which is currently being evaluated for the treatment of osteogenesis imperfecta.
Lead Product(s): Setrusumab
Therapeutic Area: Genetic Disease Brand Name: UX143
Study Phase: Phase II/ Phase IIIProduct Type: Large molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 07, 2024
Lead Product(s) : Setrusumab
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Ultragenyx Receives Breakthrough Designation For Setrusumab in Osteogenesis Imperfecta
Details : UX143 (setrusumab), is a sclerostin inhibitor antibody drug candidate, which is currently being evaluated for the treatment of osteogenesis imperfecta.
Brand Name : UX143
Molecule Type : Large molecule
Upfront Cash : Not Applicable
October 07, 2024
Details:
UX701 is an investigational AAV9 gene therapy designed to deliver stable expression of the ATP7B copper transporter. It is being evaluated in clinical trials for the treatment of Wilson Disease.
Lead Product(s): UX701
Therapeutic Area: Genetic Disease Brand Name: UX701
Study Phase: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 03, 2024
Lead Product(s) : UX701
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Ultragenyx Provides Update On Stage 1 Cohorts in Cyprus2+ Study Evaluating UX701
Details : UX701 is an investigational AAV9 gene therapy designed to deliver stable expression of the ATP7B copper transporter. It is being evaluated in clinical trials for the treatment of Wilson Disease.
Brand Name : UX701
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
October 03, 2024
Details:
GTX-102 is an investigational antisense oligonucleotide therapy designed to inhibit the expression of UBE3A, currently being evaluated for the treatment of angelman syndrome.
Lead Product(s): GTX-102
Therapeutic Area: Genetic Disease Brand Name: GTX-102
Study Phase: Phase I/ Phase IIProduct Type: Large molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 17, 2024
Lead Product(s) : GTX-102
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Ultragenyx Reports Successful End-of-Phase 2 Meeting for GTX-102 in Angelman Syndrome
Details : GTX-102 is an investigational antisense oligonucleotide therapy designed to inhibit the expression of UBE3A, currently being evaluated for the treatment of angelman syndrome.
Brand Name : GTX-102
Molecule Type : Large molecule
Upfront Cash : Not Applicable
July 17, 2024
Details:
The net proceeds of the offering to continue to advance the late-stage clinical program UX143 (setrusumab), which is being evaluated for the treatment of osteogenesis imperfecta.
Lead Product(s): Setrusumab
Therapeutic Area: Genetic Disease Brand Name: UX143
Study Phase: Phase IIIProduct Type: Large molecule
Sponsor: J.P. Morgan
Deal Size: $350.0 million Upfront Cash: Undisclosed
Deal Type: Public Offering June 13, 2024
Lead Product(s) : Setrusumab
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Partner/Sponsor/Collaborator : J.P. Morgan
Deal Size : $350.0 million
Deal Type : Public Offering
Ultragenyx Prices Public Offering Of Common Stock, Pre-Funded Warrants
Details : The net proceeds of the offering to continue to advance the late-stage clinical program UX143 (setrusumab), which is being evaluated for the treatment of osteogenesis imperfecta.
Brand Name : UX143
Molecule Type : Large molecule
Upfront Cash : Undisclosed
June 13, 2024
Details:
UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated in phase 3 clinical trials for the treatment of sanfilippo syndrome type A (MPS IIIA).
Lead Product(s): Rebisufligene Etisparvovec
Therapeutic Area: Genetic Disease Brand Name: UX111
Study Phase: Phase IIIProduct Type: Cell and Gene therapy
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 12, 2024
Lead Product(s) : Rebisufligene Etisparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Ultragenyx Plans Accelerated Approval for UX111 in Sanfilippo Syndrome A
Details : UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated in phase 3 clinical trials for the treatment of sanfilippo syndrome type A (MPS IIIA).
Brand Name : UX111
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
June 12, 2024
Details:
UX143 (setrusumab), is a sclerostin inhibitor antibody drug candidate, which is currently being evaluated for the treatment of osteogenesis imperfecta.
Lead Product(s): Setrusumab
Therapeutic Area: Genetic Disease Brand Name: UX143
Study Phase: Phase II/ Phase IIIProduct Type: Large molecule
Sponsor: Mereo BioPharma Group
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 11, 2024
Lead Product(s) : Setrusumab
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Partner/Sponsor/Collaborator : Mereo BioPharma Group
Deal Size : Not Applicable
Deal Type : Not Applicable
Ultragenyx & Mereo BioPharma: New Phase 2 Data on Setrusumab in Osteogenesis Imperfecta
Details : UX143 (setrusumab), is a sclerostin inhibitor antibody drug candidate, which is currently being evaluated for the treatment of osteogenesis imperfecta.
Brand Name : UX143
Molecule Type : Large molecule
Upfront Cash : Not Applicable
June 11, 2024
Details:
DTX401 is an investigational AAV8 gene therapy designed to deliver G6Pase-α in patients with Glycogen Storage Disease Type Ia.
Lead Product(s): DTX401,Prednisolone
Therapeutic Area: Genetic Disease Brand Name: DTX401
Study Phase: Phase IIIProduct Type: Cell and Gene therapy
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 30, 2024
Lead Product(s) : DTX401,Prednisolone
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Ultragenyx Reports Positive Results from Phase 3 DTX401 Gene Therapy Study
Details : DTX401 is an investigational AAV8 gene therapy designed to deliver G6Pase-α in patients with Glycogen Storage Disease Type Ia.
Brand Name : DTX401
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
May 30, 2024
Details:
UX143 (setrusumab) is a fully human monoclonal antibody that inhibits sclerostin, a critical protein in bone-signaling pathways affecting bone-forming cell maturation in osteogenesis imperfecta.
Lead Product(s): Setrusumab
Therapeutic Area: Genetic Disease Brand Name: UX143
Study Phase: Phase IIIProduct Type: Large molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 30, 2024
Lead Product(s) : Setrusumab
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Ultragenyx Completes Enrollment in Setrusumab Studies for Osteogenesis Imperfecta
Details : UX143 (setrusumab) is a fully human monoclonal antibody that inhibits sclerostin, a critical protein in bone-signaling pathways affecting bone-forming cell maturation in osteogenesis imperfecta.
Brand Name : UX143
Molecule Type : Large molecule
Upfront Cash : Not Applicable
April 30, 2024
Details:
GTX-102 is an investigational antisense oligonucleotide therapy designed to inhibit the expression of UBE3A, currently being evaluated in the treating of Patients with Angelman Syndrome.
Lead Product(s): GTX-102
Therapeutic Area: Genetic Disease Brand Name: GTX-102
Study Phase: Phase I/ Phase IIProduct Type: Large molecule
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 15, 2024
Lead Product(s) : GTX-102
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Ultragenyx Announces Positive Interim Phase 1/2 Data for GTX-102 in Angelman Syndrome
Details : GTX-102 is an investigational antisense oligonucleotide therapy designed to inhibit the expression of UBE3A, currently being evaluated in the treating of Patients with Angelman Syndrome.
Brand Name : GTX-102
Molecule Type : Large molecule
Upfront Cash : Not Applicable
April 15, 2024
Details:
UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated in phase 3 clinical trials for the treatment of sanfilippo syndrome type A (MPS IIIA).
Lead Product(s): UX111
Therapeutic Area: Genetic Disease Brand Name: UX111
Study Phase: Phase IIIProduct Type: Cell and Gene therapy
Sponsor: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 06, 2024
Lead Product(s) : UX111
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Partner/Sponsor/Collaborator : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Ultragenyx Reports UX111 Treatment Reduces Heparan Sulfate in Sanfilippo Syndrome
Details : UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated in phase 3 clinical trials for the treatment of sanfilippo syndrome type A (MPS IIIA).
Brand Name : UX111
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
February 06, 2024
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