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08-10 October, 2024
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26-27 September, 2024
Oligo Chemistry & Ther...Oligo Chemistry & Therapeutics 2024
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27 September, 2024
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CPhI Worldwide, MilanCPhI Worldwide, Milan
Industry Trade Show
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08-10 October, 2024
Industry Trade Show
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26-27 September, 2024
Oligo Chemistry & Ther...Oligo Chemistry & Therapeutics 2024
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27 September, 2024
Digital content
INTERVIEW #SpeakPharma
[Sponsored by another company]https://www.pharmacompass.com/speak-pharma/ice-is-pioneering-sustainability-innovation-in-bile-acid-research-for-neurodegenerative-diseases
VLOG #PharmaReel
[Sponsored by another company]DATA COMPILATION #PharmaFlow
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19 Sep 2024
// FDA
https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=761108
19 Sep 2024
// FDA
https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=125166
10 Jul 2024
// FDA
https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=125561
10 Jul 2024
// FDA
https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=125513
28 Jun 2024
// FDA
https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=761108
28 Jun 2024
// FDA
https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=125166
Details:
Ultomiris (ravulizumab) has been approved in the US as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder.
Lead Product(s): Ravulizumab
Therapeutic Area: Rare Diseases and Disorders Brand Name: Ultomiris
Study Phase: ApprovedProduct Type: Large molecule
Sponsor: AstraZeneca
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 25, 2024
Lead Product(s) : Ravulizumab
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved
Partner/Sponsor/Collaborator : AstraZeneca
Deal Size : Not Applicable
Deal Type : Not Applicable
ULTOMIRIS® (ravulizumab-cwvz) Approved in the US for the Treatment of Adults with Neuromyelitis O...
Details : Ultomiris (ravulizumab) has been approved in the US as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder.
Brand Name : Ultomiris
Molecule Type : Large molecule
Upfront Cash : Not Applicable
March 25, 2024
Details:
The collaboration aims to develop an undisclosed initial therapeutic molecule that applies JCR’s proprietary J-Brain Cargo®, blood-brain barrier penetration technology, for the treatment of a neurodegenerative disease.
Lead Product(s): Undisclosed
Therapeutic Area: Neurology Brand Name: Undisclosed
Study Phase: DiscoveryProduct Type: Undisclosed
Recipient: JCR Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration March 19, 2024
Lead Product(s) : Undisclosed
Therapeutic Area : Neurology
Highest Development Status : Discovery
Recipient : JCR Pharmaceuticals
Deal Size : Undisclosed
Deal Type : Collaboration
JCR Pharmaceuticals Announces Achievement of Milestone Using J-Brain Cargo® Technology for Neurod...
Details : The collaboration aims to develop an undisclosed initial therapeutic molecule that applies JCR’s proprietary J-Brain Cargo®, blood-brain barrier penetration technology, for the treatment of a neurodegenerative disease.
Brand Name : Undisclosed
Molecule Type : Undisclosed
Upfront Cash : Undisclosed
March 19, 2024
Details:
AG-10 (acoramidis) is an investigational, next-generation, orally-administered, highly potent, small molecule stabiliser of transthyretin (TTR). It is being evaluated in phase 3 clinical trials for the treatment of Transthyretin Amyloid Cardiomyopathy.
Lead Product(s): Acoramidis Hydrochloride
Therapeutic Area: Genetic Disease Brand Name: AG10
Study Phase: Phase IIIProduct Type: Small molecule
Recipient: BridgeBio Pharma
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 02, 2024
Lead Product(s) : Acoramidis Hydrochloride
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Recipient : BridgeBio Pharma
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : AG-10 (acoramidis) is an investigational, next-generation, orally-administered, highly potent, small molecule stabiliser of transthyretin (TTR). It is being evaluated in phase 3 clinical trials for the treatment of Transthyretin Amyloid Cardiomyopathy.
Brand Name : AG10
Molecule Type : Small molecule
Upfront Cash : Not Applicable
February 02, 2024
Details:
Under the agreement, Alexion expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.
Lead Product(s): Gene Therapy
Therapeutic Area: Rare Diseases and Disorders Brand Name: Undisclosed
Study Phase: PreclinicalProduct Type: Cell and Gene therapy
Recipient: Pfizer Inc
Deal Size: $1,000.0 million Upfront Cash: Undisclosed
Deal Type: Licensing Agreement September 20, 2023
Lead Product(s) : Gene Therapy
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Preclinical
Recipient : Pfizer Inc
Deal Size : $1,000.0 million
Deal Type : Licensing Agreement
Details : Under the agreement, Alexion expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.
Brand Name : Undisclosed
Molecule Type : Cell and Gene therapy
Upfront Cash : Undisclosed
September 20, 2023
Details:
The collaboration aims to identify novel drug targets for rare neurodegenerative and neuromuscular diseases by leveraging CONVERGE, a full-stack platform that combines highly predictive human tissue datasets with machine learning to find new targets.
Lead Product(s): Undisclosed
Therapeutic Area: Neurology Brand Name: Undisclosed
Study Phase: DiscoveryProduct Type: Undisclosed
Recipient: Verge Genomics
Deal Size: $882.0 million Upfront Cash: $42.0 million
Deal Type: Collaboration September 08, 2023
Lead Product(s) : Undisclosed
Therapeutic Area : Neurology
Highest Development Status : Discovery
Recipient : Verge Genomics
Deal Size : $882.0 million
Deal Type : Collaboration
Details : The collaboration aims to identify novel drug targets for rare neurodegenerative and neuromuscular diseases by leveraging CONVERGE, a full-stack platform that combines highly predictive human tissue datasets with machine learning to find new targets.
Brand Name : Undisclosed
Molecule Type : Undisclosed
Upfront Cash : $42.0 million
September 08, 2023
Details:
Under the agreement, Alexion will expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.
Lead Product(s): Gene Therapy
Therapeutic Area: Rare Diseases and Disorders Brand Name: Undisclosed
Study Phase: PreclinicalProduct Type: Cell and Gene therapy
Recipient: Pfizer Inc
Deal Size: $1,000.0 million Upfront Cash: Undisclosed
Deal Type: Licensing Agreement July 28, 2023
Lead Product(s) : Gene Therapy
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Preclinical
Recipient : Pfizer Inc
Deal Size : $1,000.0 million
Deal Type : Licensing Agreement
Details : Under the agreement, Alexion will expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.
Brand Name : Undisclosed
Molecule Type : Cell and Gene therapy
Upfront Cash : Undisclosed
July 28, 2023
Details:
AG10 (acoramidis) is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin (TTR), being investigated for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).
Lead Product(s): Acoramidis Hydrochloride
Therapeutic Area: Rare Diseases and Disorders Brand Name: AG10
Study Phase: Phase IIIProduct Type: Small molecule
Recipient: BridgeBio Pharma
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 17, 2023
Lead Product(s) : Acoramidis Hydrochloride
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase III
Recipient : BridgeBio Pharma
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : AG10 (acoramidis) is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin (TTR), being investigated for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).
Brand Name : AG10
Molecule Type : Small molecule
Upfront Cash : Not Applicable
July 17, 2023
Details:
ALXN2040 (danicopan) is an investigational oral medicine in development as an add-on to standard of care C5 inhibitor therapy Ultomiris (ravulizumab) or Soliris (eculizumab) for patients with PNH who experience clinically significant EVH.
Lead Product(s): Danicopan,Eculizumab,Ravulizumab
Therapeutic Area: Rare Diseases and Disorders Brand Name: ALXN2040
Study Phase: Phase IIIProduct Type: Small molecule
Recipient: AstraZeneca
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 09, 2023
Lead Product(s) : Danicopan,Eculizumab,Ravulizumab
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase III
Recipient : AstraZeneca
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : ALXN2040 (danicopan) is an investigational oral medicine in development as an add-on to standard of care C5 inhibitor therapy Ultomiris (ravulizumab) or Soliris (eculizumab) for patients with PNH who experience clinically significant EVH.
Brand Name : ALXN2040
Molecule Type : Small molecule
Upfront Cash : Not Applicable
June 09, 2023
Details:
Under the agreement, the two companies will begin research and development of new treatments using the J-Brain Cargo®, blood-brain barrier (“BBB”) penetration technology, in combination with an undisclosed effector molecule for the treatment of a neurodegenerative disease.
Lead Product(s): Undisclosed
Therapeutic Area: Neurology Brand Name: Undisclosed
Study Phase: DiscoveryProduct Type: Undisclosed
Recipient: JCR Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration April 03, 2023
Lead Product(s) : Undisclosed
Therapeutic Area : Neurology
Highest Development Status : Discovery
Recipient : JCR Pharmaceuticals
Deal Size : Undisclosed
Deal Type : Collaboration
Details : Under the agreement, the two companies will begin research and development of new treatments using the J-Brain Cargo®, blood-brain barrier (“BBB”) penetration technology, in combination with an undisclosed effector molecule for the treatment of a ne...
Brand Name : Undisclosed
Molecule Type : Undisclosed
Upfront Cash : Undisclosed
April 03, 2023
Details:
The acquisition creates an opportunity to accelerate Alexion’s growth in genomic medicines through unique technology, an experienced rare disease R&D team, and expertise in pre-clinical development.
Lead Product(s): AAV-based Gene Therapy
Therapeutic Area: Rare Diseases and Disorders Brand Name: Undisclosed
Study Phase: DiscoveryProduct Type: Cell and Gene therapy
Recipient: LogicBio Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Acquisition November 16, 2022
Lead Product(s) : AAV-based Gene Therapy
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery
Recipient : LogicBio Therapeutics
Deal Size : Undisclosed
Deal Type : Acquisition
Alexion, AstraZeneca Rare Disease, Completes Acquisition of LogicBio® Therapeutics
Details : The acquisition creates an opportunity to accelerate Alexion’s growth in genomic medicines through unique technology, an experienced rare disease R&D team, and expertise in pre-clinical development.
Brand Name : Undisclosed
Molecule Type : Cell and Gene therapy
Upfront Cash : Undisclosed
November 16, 2022
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