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Global Biopharmaceutical CMO & CRO Market is Expected to Reach $27.95 Billion by 2028: Players Include Toyobo, Samsung Biologics, Patheon, AbbVie and Binex - ResearchAndMarkets.com

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Thursday, May 4, 2023

The abbreviations for contract research organisations and contract manufacturing companies in the pharmaceutical industry are biopharmaceutical CMO and CRO, respectively.

Key Points:

The abbreviations for contract research organisations and contract manufacturing companies in the pharmaceutical industry are biopharmaceutical CMO and CRO, respectively.
Consumer demand for biopharmaceutical treatments has been a major driver of the biopharmaceutical industry's phenomenal growth.
Global Biopharmaceutical CMO and CRO Market is segmented based on the source, service, product, and region.
The biopharmaceutical CMO and CRO market is anticipated to grow quickly in the coming years as a result of these factors.

Alnylam Announces U.S. Food and Drug Administration Acceptance of Supplemental New Drug Application for OXLUMO® for the Treatment of Advanced Primary Hyperoxaluria Type 1

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Tuesday, March 1, 2022

FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Risk, Therapy, Type, ESC, Therapeutic index, European Medicines Agency, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pain, Kidney, European Commission, Injection site reaction, Medical Affairs Bureau, Medication, Royal commission, Safety, Itch, Child, Alnylam Pharmaceuticals, Review, Erythema, Swelling, New Drug Application, Food, Adult, PH1, PDUFA, Shanda, European Directive on Traditional Herbal Medicinal Products, FDA, RNA interference, Nasdaq, EMA, Committee for Medicinal Products for Human Use, EVP, Patient, Enhance, GO, Prescription Drug User Fee Act, Milk, Committee, CHMP, Plasma, EGFR, Pregnancy, Profile, Pharmaceutical industry, Medical device, Medicine, Birth control, HAO1, Lumasiran, ILLUMINATE-C Phase 3 Study, Primary Hyperoxaluria Type 1 (PH1), RNAi, Alnylam Pharmaceuticals, LUMASIRAN, ILLUMINATE-C PHASE 3 STUDY, PRIMARY HYPEROXALURIA TYPE 1 (PH1), RNAI, ALNYLAM PHARMACEUTICALS

This filing acceptance is a positive step for patients with advanced PH1, who are at risk for the devastating complications of systemic oxalosis.

Key Points:

This filing acceptance is a positive step for patients with advanced PH1, who are at risk for the devastating complications of systemic oxalosis.
OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
The most common adverse reaction that occurred in patients treated with OXLUMO was injection site reaction (38%).
Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of advanced primary hyperoxaluria type 1 (PH1).

Alnylam to Present Full 18-Month Results from the HELIOS-A Phase 3 Study of Vutrisiran at the Société Francophone du Nerf Périphérique Annual Meeting

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Friday, January 14, 2022

The meeting will be held January 21-22 in Paris, France, and convenes healthcare professionals focused on the peripheral nervous system.

Key Points:

The meeting will be held January 21-22 in Paris, France, and convenes healthcare professionals focused on the peripheral nervous system.
Vutrisiran has been granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis.
In the U.S., vutrisiran has received an action date under the Prescription Drug User Fee Act (PDUFA) of April 14, 2022.
Alnylam Management will discuss the HELIOS-A 18-month results via a conference call on Friday, January 21, 2022, at 8:30 am ET.

Alnylam Submits CTA Application for ALN-APP, an Investigational RNAi Therapeutic for the Treatment of Alzheimer’s Disease and Cerebral Amyloid Angiopathy

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Wednesday, December 22, 2021

Mental Health, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, ALN-APP, Early-Onset Alzheimer’s Disease, RNAi, Alnylam Pharmaceuticals, ALN-APP, EARLY-ONSET ALZHEIMER’S DISEASE, RNAI, ALNYLAM PHARMACEUTICALS

As such, ALN-APP represents the start of Alnylams expansion of RNAi therapeutic opportunities in extra-hepatic tissues.

Key Points:

As such, ALN-APP represents the start of Alnylams expansion of RNAi therapeutic opportunities in extra-hepatic tissues.
ALN-APP is an investigational, intrathecally administered RNAi therapeutic targeting amyloid precursor protein (APP) in development in collaboration with Regeneron Pharmaceuticals for the treatment of Alzheimers disease (AD) and cerebral amyloid angiopathy (CAA).
Alzheimers disease (AD) is the most common neurodegenerative disease and the most common form of dementia, affecting over 30 million people worldwide.
Available treatment options include symptomatic treatment and treatment to reduce amyloid deposits in the brain.

Alnylam Initiates Phase 2 Study of Lumasiran in Patients with Recurrent Kidney Stone Disease

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Monday, December 20, 2021

Health, Genetics, Clinical Trials, General Health, Pharmaceutical, Biotechnology, the Phase 2 Study in Patients with Recurrent Kidney Stone Disease, Lumasiran, Recurrent Kidney Stone Disease, RNAi, Alnylam Pharmaceuticals, THE PHASE 2 STUDY IN PATIENTS WITH RECURRENT KIDNEY STONE DISEASE, LUMASIRAN, RECURRENT KIDNEY STONE DISEASE, RNAI, ALNYLAM PHARMACEUTICALS

Recurrent kidney stone disease is a prevalent disease, associated with significant clinical burden, including pain, infection, hospitalizations, and an increased risk of developing chronic kidney disease and kidney failure.

Key Points:

Recurrent kidney stone disease is a prevalent disease, associated with significant clinical burden, including pain, infection, hospitalizations, and an increased risk of developing chronic kidney disease and kidney failure.
The Phase 2 trial is a randomized, double-blind, placebo-controlled study to evaluate the safety, efficacy, pharmacodynamics, and pharmacokinetics of lumasiran administered subcutaneously in patients with recurrent calcium oxalate kidney stone disease and elevated urinary oxalate levels.
Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of advanced primary hyperoxaluria type 1 (PH1) and recurrent kidney stone disease.
Thus, by silencing HAO1 and depleting the GO enzyme, lumasiran inhibits production of oxalate the metabolite that directly contributes to the pathophysiology of PH1 and recurrent kidney stone disease.

Alnylam Submits CTA Application for ALN-XDH, an Investigational RNAi Therapeutic for the Treatment of Gout

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Monday, December 20, 2021

Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Intellectual property, Biology, Gout, Private Securities Litigation Reform Act, Risk, RNA interference, Medication, Disease, Therapy, Physiology, Instagram, LinkedIn, RNA, RNA transfection, FDA, Pain, CTA, Therapeutic index, Inflammatory arthritis, Uric acid, Growth, Nobel Prize in Physiology or Medicine, Medicines and Healthcare products Regulatory Agency, EMA, U.S. Securities and Exchange Commission, United Kingdom, Xanthine dehydrogenase, MHRA, Edema, Safety, Security (finance), Drug development, Flare, Chronic kidney disease, Sanofi, Alnylam Pharmaceuticals, Kidney, Tophus, Cell, Program, Nasdaq, Science, COVID-19, Enhance, SEC, Marketing, Twitter, Nobel Prize, Government, Company, XDH, CNS, Regeneron Pharmaceuticals, Gene silencing, Novartis, Patient, Sirna, Central nervous system, Joint, Pharmaceutical industry, Vaccine, Medicine, ALN-XDH, Gout, RNAi, Alnylam Pharmaceuticals, ALN-XDH, Gout, RNAI, ALNYLAM PHARMACEUTICALS

We believe the existing treatment landscape for gout has substantial limitations attributed to an incomplete response to standard of care, tolerability issues, and poor patient convenience.

Key Points:

We believe the existing treatment landscape for gout has substantial limitations attributed to an incomplete response to standard of care, tolerability issues, and poor patient convenience.
ALN-XDH is an investigational, subcutaneously administered RNAi therapeutic targeting xanthine dehydrogenase (XDH) in development for the treatment of gout.
Reducing XDH with an RNAi therapeutic is expected to result in potent urate lowering, essential in preventing gout-associated flares and managing the disease.
Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform.

Alnylam Presents New Data for Zilebesiran, an Investigational RNAi Therapeutic for the Treatment of Hypertension, at the American Heart Association Scientific Sessions 2021

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Saturday, November 13, 2021

Biotechnology, Health, Genetics, Pharmaceutical, Cardiology, Zilebesiran Phase 1 Study, Zilebesiran, Hypertension, RNAi, Alnylam Pharmaceuticals, ZILEBESIRAN PHASE 1 STUDY, ZILEBESIRAN, HYPERTENSION, RNAI, ALNYLAM PHARMACEUTICALS

Findings on the pharmacodynamic and antihypertensive effects of zilebesiran six months after a single dose were presented at the American Heart Association (AHA) Scientific Sessions 2021, taking place virtually from November 13-15, 2021.

Key Points:

Findings on the pharmacodynamic and antihypertensive effects of zilebesiran six months after a single dose were presented at the American Heart Association (AHA) Scientific Sessions 2021, taking place virtually from November 13-15, 2021.
After a single dose of 800 mg, mean reductions in 24-hour SBP of >20 mm Hg were observed at Month 6.
To view the data presented by Alnylam at the AHA Scientific Sessions, please visit www.alnylam.com/capella .
This release discusses investigational RNAi therapeutics and is not intended to convey conclusions about efficacy or safety as to any investigational RNAi therapeutics.

Dicerna Announces Two Targets Meet Preclinical Proof of Principle Criteria in Neurodegeneration and Pain Under Global Research Collaboration and Licensing Agreement With Lilly

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Friday, November 12, 2021

This milestone triggers two single-digit multimillion-dollar milestone payments to Dicerna, which the Company expects to receive in the fourth quarter of 2021.

Key Points:

This milestone triggers two single-digit multimillion-dollar milestone payments to Dicerna, which the Company expects to receive in the fourth quarter of 2021.
In 2018, Dicerna and Lilly announced a global licensing and research collaboration focused on the discovery, development and commercialization of potential new therapies for cardiometabolic disease, neurodegenerative diseases and pain.
Including these two targets, there are currently seven candidates in preclinical or clinical development under the agreement that are targeted to address cardiometabolic, neurodegenerative or pain indications.
Ribonucleic acid interference, or RNAi, provides a unique advantage to other disease inhibitor technologies, like small-molecule pharmaceuticals or monoclonal antibodies.

Alnylam Presents Positive Results from ILLUMINATE-C Phase 3 Study of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1

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Friday, November 5, 2021

Research, Clinical Trials, Biotechnology, Other Health, Health, Pharmaceutical, General Health, Other Science, Science, GO, Society, Milk, Safety, Alnylam Pharmaceuticals, AES, American Society of Nephrology, Pregnancy, CKD, Cohort study, Plasma, Kidney, Therapeutic index, Baylor College of Medicine, AUC, General manager, Disease, RNA interference, Swelling, Department, ESC, EGFR, EMA, Food, Profile, European Medicines Agency, Associate professor, Chronic kidney disease, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FDA, Medication, Dialysis, 1991 World Sportscar Championship, Pain, Itch, Hyperoxaluria, Therapy, Diagnosis, Severe cognitive impairment, Enhance, ASN, Hydroxyacid oxidase (glycolate oxidase) 1, PH1, Cardiomyopathy, Nasdaq, Patient, HAO1, Risk, AE, Bone, Child, Injection site reaction, Erythema, Adult, Pharmaceutical industry, Birth control, Lumasiran, ILLUMINATE-C Phase 3 Study, Primary Hyperoxaluria Type 1 (PH1), RNAi, Alnylam Pharmaceuticals, LUMASIRAN, ILLUMINATE-C PHASE 3 STUDY, PRIMARY HYPEROXALURIA TYPE 1 (PH1), RNAI, ALNYLAM PHARMACEUTICALS

PH1 patients progressing to or being diagnosed with end-stage kidney disease often have substantial elevations in plasma oxalate despite intensive hemodialysis.

Key Points:

PH1 patients progressing to or being diagnosed with end-stage kidney disease often have substantial elevations in plasma oxalate despite intensive hemodialysis.
ILLUMINATE-C enrolled 21 patients: six patients in Cohort A who did not require dialysis and 15 patients on hemodialysis in Cohort B.
OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of primary hyperoxaluria type 1 (PH1).