Sanofi Genzyme is a global business unit of Sanofi that specializes in rare diseases, multiple sclerosis, immunology, and oncology. Their mission is to help individuals with challenging and complex conditions that are often difficult to diagnose and treat. The company is committed to discovering and advancing new therapies to provide hope to patients and their families worldwide. Genzyme became a part of Sanofi in 2011 and has since expanded its focus to include multiple sclerosis, oncology, and immunology. Their approach is shaped by a long history of developing highly specialized treatments and building strong relationships with physician and patient communities. Founded in 1981, Genzyme quickly became one of the world's leading biotechnology companies, known for its pioneering work in treating rare genetic disorders and innovative contributions to medical science and biomanufacturing.

Tags

Endocrinology and Metabolic Disease

Nervous System Diseases

Congenital Disorders

Small molecule drug

Enzyme

AAV based gene therapy

Disease domain score

A glimpse into the focused therapeutic areas

Technology Platform

Most used technologies in drug development

Targets

Most frequently developed targets

Disease Domain	Count

Endocrinology and Metabolic Disease	36
Nervous System Diseases	27
Neoplasms	15
Immune System Diseases	14
Hemic and Lymphatic Diseases	12
Infectious Diseases	9

Top 5 Drug Type	Count

Small molecule drug	37
Enzyme	8
AAV based gene therapy	6
Hormone	6
Monoclonal antibody	5

Top 5 Target	Count

AT III(Antithrombin-III)	3
INSR(Insulin receptor)	3
Tubulin(Tubulin)	2
UGCG(Ceramide glucosyltransferase)	2
α-glucosidase(Acidic alpha-glucosidase)	2

286

Drugs associated with Genzyme Corp.

Mavorixafor

Target

CXCR4

Mechanism

CXCR4 antagonists

Active Org.

X4 Pharmaceuticals, Inc. [+2]

Originator Org.

Genzyme Corp.

Active Indication

WHIM Syndrome [+7]

Inactive Indication

HIV Infections

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

First Approval Date26 Apr 2024

Fidanacogene elaparvovec

Target

factor IX

Mechanism

Factor IX gene transference

Active Org.

Pfizer Inc. [+1]

Originator Org.

The Children's Hospital of Philadelphia

Active Indication

Hemophilia B

Inactive Indication-

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

First Approval Date27 Dec 2023

Nirsevimab

Target

RSV F protein

Mechanism

Respiratory syncytial virus F protein inhibitors

Active Org.

AstraZeneca PLC [+5]

Originator Org.

AstraZeneca AB

Active Indication

Respiratory Syncytial Virus Infections [+2]

Inactive Indication-

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

EU [+3]

First Approval Date31 Oct 2022

749

Clinical Trials associated with Genzyme Corp.

NCT06105554 / RecruitingPhase 1/2IIT

Phase I/II Open Label Study of Belumosudil Mesylate Alone, and in Combination With Dexamethasone, in Patients With Relapsed/Refractory Multiple Myeloma

Phase 1 is to find the recommended dose of belumosudil mesylate that can be given to patients with relapsed/refractory MM.
Phase 2 is to learn if the dose of belumosudil mesylate found in Phase 1 can help to control the disease.

Start Date30 Jan 2024

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

[+1]

NCT05911321 / RecruitingPhase 2IIT

Study of Isatuximab Plus Pomalidomide and Dexamethasone in Highly Toxicity-vulnerable Subjects With Relapsed or Refractory Multiple Myeloma

This research study aims to evaluate the safety and effectiveness of the combination of isatuximab, pomalidomide, and dexamethasone (Isa-Pd) for the treatment of relapsed or refractory multiple myeloma (RRMM), which refers to multiple myeloma that has returned or has not responded to prior treatment. The study will specifically investigate the impact of administering lower-than-standard doses of pomalidomide and dexamethasone. Using lower doses of pomalidomide and dexamethasone in this setting has not been approved by the Food and Drug Administration (FDA).

Start Date05 Dec 2023

Sponsor / Collaborator

UNC Lineberger Comprehensive Cancer Center

[+1]

NCT06121180 / RecruitingPhase 2IIT

A Phase II Study of Cemiplimab Plus Ziv-Aflibercept for Subjects With Metastatic Uveal Melanoma

The goal of this clinical research study is to find out if Cemiplimab plus Ziv-Aflibercept is safe and effective in treating your condition of metastatic (spread to other parts of your body) uveal melanoma. This research study will test the study drugs to see if the combination of Cemiplimab plus Ziv-Aflibercept can make tumors shrink or stop growing.

Start Date31 Oct 2023

Sponsor / Collaborator

H. Lee Moffitt Cancer Center & Research Institute, Inc.

[+2]

100 Clinical Results associated with Genzyme Corp.

0 Patents (Medical) associated with Genzyme Corp.

3,221

Literatures (Medical) associated with Genzyme Corp.

2022·Journal of Infectious Diseases

Dengue endemicity, force of infection, and variation in transmission intensity in 13 endemic countries

Author: Nealon, Joshua ; Bouckenooghe, Alain ; Cortes, Margarita ; Coudeville, Laurent ; Frago, Carina ; Macina, Denis ; Tam, Clarence C.

Dengue endemicity varies but comparative, multicountry data are extremely limited. An improved understanding is needed to prioritize prevention, including vaccination, which is currently recommended only under specific epidemiol. conditions. We used serol. study data from 46 geog. sites in 13 countries to estimate dengue force of infection (FOI, the proportion of children seroconverting per yr) under assumptions of either age-constant or age-varying FOI, and the age at which 50% and 80% of children had been infected. After exclusions, 13 661 subjects were included. Estimated constant FOI varied widely, from 1.7% (Singapore) to 24.1% (the Philippines). In the site-level anal. 44 sites (96%) reached 50% seroconversion and 35 sites (75%) reached 80% seroconversion by age 18 years, with significant heterogeneity. These findings confirm that children living in dengue-endemic countries receive intense early dengue exposure, increasing risk of secondary infection, and imply serosurveys at fine spatial resolutions are needed to inform vaccination campaigns.

2022

Recent progress in late stage deuterations of complex molecules

Author: Volker, Derdau

Deuterated drugs have seen a continuous interest in pharmaceutical industry. While the gold-rush mood from the early 21^th century has faded a bit, there is a proven evidence that the substitution of hydrogen by its isotope deuterium can have a significant pos. effect for patients. With the first deuterated drugs on the market and further ones in clin. trials the need for the development of convenient deuteration methods became evident. The possible applications reach from small milligram reactions for medicinal chem. optimization to large industrial batches with tons of material in process chem. All these efforts combine the need for cost-efficient, easy to handle and selective deuteration reactions. One approach to fulfill all these criteria can be the late stage deuteration reaction of complex mols. Late stage deuterations follow the concept of the Hydrogen Isotope Exchange (HIE) reactions, which are considered as the most fundamental of all C-H functionalization's. In the presentation an overview and perspective of the most convenient late stage deuteration methods is given and discussed.

2022

Development of a robust control strategy for continuous diafiltration

Author: Malladi, Shashi ; Thomas, Crystal Y. ; Coolbaugh, Michael J.

Batch processing has traditionally been employed in the pharmaceutical industry for the purification of monoclonal antibodies (mAbs). However, in recent years, integrated continuous biomanufg. (ICB) technologies have been increasingly developed and adopted within these purification processes due to their advantages such as process intensification, smaller footprint, and more homogenous product quality. Nevertheless, implementation of end-to-end ICB still requires (1) addnl. development for post-chromatog. unit operations, including diafiltration and (2) a high-fidelity continuous control strategy systematically integrating all the unit operations. This presentation addresses both considerations, as described below: Most prior ICB efforts have focused primarily on the chromatog. operations in the protein purification process. Diafiltration, which occurs towards the end of the process train for buffer exchange, also has the potential for continuous operation. Operating DF in a continuous format requires precise control over the process parameters such that the exiting protein concentration remains well-controlled and the target buffer exchange is achieved, regardless of the fluctuations in the feed stream. In this work, a control framework for continuous diafiltration (cDF) was designed to address the stated requirements. Experiments were conducted to develop semi-empirical process models relating the key parameters governing the DF operation. The models were subsequently coupled with simulations to determine the optimal design and operating space, which were then adopted to develop the control. The control strategy was evaluated for its robustness in conjunction with other unit operations. Our automated cDF will not only be useful in developing an integrated workflow but will also provide guidelines for improved design of cDF filters. In summary, the development of an integrated continuous control strategy is critical for end-to-end success, but challenging, as it would have to ideally identify all real-time process disturbances and minimize their subsequent effects on the critical quality attributes of the drug substance. Our unified exptl. and modeling approach, as demonstrated for diafiltration operation, can be leveraged towards the development and implementation of a successful continuous control strategy essential for end-to-end ICB.

444

News (Medical) associated with Genzyme Corp.

04 Apr 2024

·www.globenewswire.com

Pharming Group announces the 2024 Annual General Meeting of Shareholders

Leiden, The Netherlands, April 4, 2024: Pharming Group N.V. (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM / Nasdaq: PHAR) announces that the Company’s 2024 Annual General Meeting of Shareholders (the “AGM”) will be held on Tuesday, May 21, 2024 at 14:30 CEST. The Notice to Convene, Explanatory Notes, Proxy and other meeting documents for the AGM can be found on the Company’s website under Investors/Shareholder Meetings. The agenda of the AGM includes the reappointment of Ms. Barbara Yanni and Mr. Mark Pykett as Non-Executive Directors for periods of four years, (ii) the adoption of the updated Remuneration Policy for members of the Board of Directors (iii) the authorization for the Board of Directors to issue shares and/or options, and to restrict or exclude pre-emptive rights in relation thereto, up to 10% of the issued share capital for general corporate purposes, including issuances for M&A transactions and issuances pursuant to equity incentive plans and (iv) the authorization for the Board of Directors to repurchase shares. The AGM will be held at the Corpus Congress Centre, Willem Einthovenstraat 1, 2342 BH in Oegstgeest, the Netherlands, and will be webcast live. About Pharming Group N.V. Pharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules, biologics, and gene therapies that are in early to late-stage development. Pharming is headquartered in Leiden, Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific. For more information, visit www.pharming.com and find us on LinkedIn. Inside Information This press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation. For further public information, contact: Pharming Group, Leiden, The NetherlandsMichael Levitan, VP Investor Relations & Corporate CommunicationsT: +1 (908) 705 1696E: investor@pharming.com FTI Consulting, London, UKVictoria Foster Mitchell/Alex Shaw/Amy ByrneT: +44 203 727 1000 LifeSpring Life Sciences Communication, Amsterdam, The NetherlandsLeon MelensT: +31 6 53 81 64 27E: pharming@lifespring.nl

Executive Change

04 Apr 2024

·www.globenewswire.com

Pharming Group announces the filing of its 2023 Annual Report and Form 20-F

Leiden, The Netherlands, April 4, 2024: Pharming Group N.V. (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM / Nasdaq: PHAR) announces the filing of its Annual Report for the year ended December 31, 2023 (the “Period”). The Annual Report is available under Investors/Financial documents on the Pharming.com website. The Company also announces the filing of its Annual Report on Form 20-F with the U.S. Securities and Exchange Commission (SEC) for the Period. The 2023 Annual Report on Form 20-F can be found under Investors/SEC filings on Pharming.com and through the SEC website once it has been filed. About Pharming Group N.V. Pharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules, biologics, and gene therapies that are in early to late-stage development. Pharming is headquartered in Leiden, Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific. For more information, visit www.pharming.com and find us on LinkedIn. Inside Information This press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation. For further public information, contact: Pharming Group, Leiden, The NetherlandsMichael Levitan, VP Investor Relations & Corporate CommunicationsT: +1 (908) 705 1696E: investor@pharming.com FTI Consulting, London, UKVictoria Foster Mitchell/Alex Shaw/Amy ByrneT: +44 203 727 1000 LifeSpring Life Sciences Communication, Amsterdam, The NetherlandsLeon MelensT: +31 6 53 81 64 27E: pharming@lifespring.nl

04 Apr 2024

·www.prnewswire.com

Cadenza Bio, Inc. Appoints Michael A. Panzara, MD, MPH, to its Board of Directors

OKLAHOMA CITY, April 4, 2024 /PRNewswire/ -- Cadenza Bio, Inc., a pioneering preclinical biotechnology company dedicated to developing innovative small-molecule drugs for the treatment of multiple sclerosis (MS) and other demyelinating and inflammatory diseases, proudly announces the appointment of Michael A. Panzara, MD, MPH, to its esteemed board of directors. Dr. Panzara brings over 22 years of invaluable biopharmaceutical experience to Cadenza Bio. He currently serves as chief medical officer at Neurvati Neurosciences, a neuroscience-focused biotechnology company working to advance the development of clinical-stage product candidates for neurological and neuropsychiatric disorders. Prior to his role at Neurvati, Dr. Panzara held key leadership positions at Wave Life Sciences, Sanofi, Genzyme, and Biogen, showcasing his deep understanding of neurological disorders and drug development. He also serves on the board of directors of Athira Pharma, Inc., a neurology-focused clinical-stage biopharmaceutical company. Over his career, Dr. Panzara oversaw development activities and global regulatory approvals for several MS drugs. He served as the head of the Multiple Sclerosis, Neurology, and Ophthalmology Therapeutic Area for Global Development at Sanofi Genzyme, where he oversaw global regulatory approvals of LEMTRADA® (alemtuzumab) and AUBAGIO® (teriflunomide) and was responsible for development strategy and oversight within these therapeutic areas. During his time at Biogen, he served as global clinical lead for the development of TYSABRI® (natalizumab) for MS and managed clinical development activities for all late-stage MS products including AVONEX® (interferon beta-1a), PLEGRIDY® (PEG-interferon beta-1a), and TECFIDERA® (dimethylfumarate). "We are delighted to welcome Dr. Panzara to our board of directors," said Dr. Carol Curtis, chief executive officer of Cadenza Bio. "His extensive experience in developing therapies for MS and other neurological disorders aligns perfectly with our mission at Cadenza Bio. Dr. Panzara's strategic insights and deep understanding of the industry will be invaluable as we continue to advance our innovative drug pipeline and work towards bringing transformative treatments to patients in need." Dr. Panzara received his undergraduate degree in biology from the University of Pennsylvania and his medical degree from Stanford University School of Medicine. He completed his neurology training at Massachusetts General Hospital and received postdoctoral training in immunology and rheumatology at Brigham and Women's Hospital. Additionally, Dr. Panzara earned his master of public health degree from the Harvard School of Public Health. With Dr. Panzara joining the board of directors, Cadenza Bio is in a stronger position to achieve its goal of developing groundbreaking therapies for MS and other demyelinating and inflammatory diseases. "I look forward to joining the board of directors at Cadenza Bio at such an exciting time in the growth of the company," said Dr. Panzara. "Their approach in developing innovative dual-action therapeutic agents with neuroprotective and immunomodulatory effects has great potential to significantly expand our ability to address the unmet needs that remain for many MS patients." For more information about Cadenza Bio and its groundbreaking research, please visit . About Cadenza Bio, Inc. Cadenza Bio, Inc. is a preclinical biotechnology company focused on developing innovative small-molecule drugs for the treatment of demyelinating and inflammatory diseases. The company's mission is to break the cycle of disease and transform lives by developing therapies that not only alleviate symptoms but also halt progression, promote repair, and restore quality of life for patients. Cadenza Bio's MS drug pipeline, emerging from a collaboration between leading researchers, demonstrates promising preclinical results and offers hope for patients suffering from these debilitating conditions. SOURCE Cadenza Bio, Inc.

Executive Change

100 Deals associated with Genzyme Corp.

100 Translational Medicine associated with Genzyme Corp.

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Pipeline

Pipeline Snapshot as of 03 Jul 2024

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

Preclinical

Phase 1 Clinical

Phase 2 Clinical

Phase 3 Clinical

Approved

218

Other

Current Projects

Drug(Targets)	Indications	Global Highest Phase

Glyburide ( Kir6.2 x SUR1 )	Diabetes Mellitus, Type 2 More	Approved
Rifampin ( RNAP )	Meningococcal Infections More	Approved
Eliglustat ( UGCG )	Gaucher Disease More	Approved
Insulin Glargine/Lixisenatide ( GLP-1R x INSR )	Diabetes Mellitus, Type 2 More	Approved
Insulin glargine (Sanofi) ( INSR )	Diabetes Mellitus, Type 1 More	Approved

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