IP Library › Granted Patent US 12,338,279
Granted Patent B2
US 12,338,279 · App. 18/988,612 · Granted Jun 24, 2025

Selective and potent inhibitory antibodies of myostatin activation

Inventors: Christopher Chapron (Cambridge, MA); Frederick C. Streich, Jr. (Cambridge, MA); Kaleigh Canonico (Cambridge, MA); Shaun M. Cote (Cambridge, MA); Francis Danehy, Jr. (Cambridge, MA); Melissa Fulham (Cambridge, MA); Yan Huang (Cambridge, MA); Justin William Jackson (Cambridge, MA); Mania Kavosi (Cambridge, MA); Sandeep Kumar (Cambridge, MA)
Assignee: Scholar Rock, Inc.
C07K16/22A61K9/0019A61K31/155A61K38/26A61P3/04A61P3/10A61K2039/505A61K2039/54C07K2317/21C07K2317/565
View Patent ↗
Loading inventors, assignments & file history…
Monitor This Case
Get email alerts when status or documents change.
Order Certified Copies
Most orders are placed with the USPTO same day — all within 24 business hours.
Order via The Patent Place →
Pre-filled with this patent's details
Quick Facts
Patent No.
US 12,338,279
App. No.
18/988,612
Granted
Jun 24, 2025
Kind
B2
Abstract

The present disclosure relates to antibodies and antigen-binding fragments that specifically bind to pro/latent myostatin and uses thereof.

Claims (14)

1. An antibody or antigen-binding fragment thereof that specifically binds to pro/latent myostatin, wherein the antibody or antigen-binding fragment comprises six complementarity determining regions (CDRs), an H-CDR1, an H-CDR2, an H-CDR3, an L-CDR1, an L-CDR2, and an L-CDR3, wherein:

a) the H-CDR1 comprises the amino acid sequence of SEQ ID NO: 201, the H-CDR2 comprises the amino acid sequence of SEQ ID NO: 219, the H-CDR3 comprises the amino acid sequence of SEQ ID NO: 220, the L-CDR1 comprises the amino acid sequence of SEQ ID NO: 216, the L-CDR2 comprises the amino acid sequence of SEQ ID NO: 222, and the L-CDR3 comprises the amino acid sequence of SEQ ID NO: 223, wherein the CDR sequences are numbered according to Kabat numbering system;

b) the H-CDR1 comprises the amino acid sequence of SEQ ID NO: 234, the H-CDR2 comprises the amino acid sequence of SEQ ID NO: 235, the H-CDR3 comprises the amino acid sequence of SEQ ID NO: 236, the L-CDR1 comprises the amino acid sequence of SEQ ID NO: 237, the L-CDR2 comprises the amino acid sequence EVS, and the L-CDR3 comprises the amino acid sequence of SEQ ID NO: 239, wherein the CDR sequences are numbered according to Chothia numbering system; or

c) the H-CDR1 comprises the amino acid sequence of SEQ ID NO: 3, the H-CDR2 comprises the amino acid sequence of SEQ ID NO: 256, the H-CDR3 comprises the amino acid sequence of SEQ ID NO: 257, the L-CDR1 comprises the amino acid sequence of SEQ ID NO: 258, the L-CDR2 comprises the amino acid sequence EVS, and the L-CDR3 comprises the amino acid sequence of SEQ ID NO: 260, wherein the CDR sequences are numbered according to IMGT numbering system.

2. The antibody or antigen-binding fragment thereof of claim 1 , comprising a heavy chain variable domain that is at least 90% identical to the amino acid sequence of SEQ ID NO: 402, a light chain variable domain that is at least 90% identical to the amino acid sequence of SEQ ID NO: 412, or a combination thereof.

3. The antibody or antigen-binding fragment thereof of claim 1 , comprising a heavy chain variable domain comprising the amino acid sequence of SEQ ID NO: 402 and a light chain variable domain comprising the amino acid sequence of SEQ ID NO: 412.

4. The antibody or antigen-binding fragment thereof of claim 1 , wherein the antibody is a human antibody of the IgG1 subtype or of the IgG4 subtype.

5. The antibody or antigen-binding fragment thereof of claim 1 , wherein the antibody is a human antibody of the IgG4 subtype.

6. The antibody or antigen-binding fragment thereof of claim 5 , wherein the antibody comprises an S228P mutation.

7. The antibody or antigen-binding fragment thereof of claim 1 , comprising a heavy chain that is at least 90% identical to the amino acid sequence of SEQ ID NO: 503, a light chain that is at least 90% identical to the amino acid sequence of SEQ ID NO: 504, or a combination thereof.

8. The antibody or antigen-binding fragment thereof of claim 1 , comprising a heavy chain comprising the amino acid sequence of SEQ ID NO: 503 and a light chain comprising the amino acid sequence of SEQ ID NO: 504.

9. A pharmaceutical composition comprising the antibody or antigen-binding fragment thereof of claim 1 and a pharmaceutically acceptable excipient.

10. The pharmaceutical composition of claim 9 , wherein the composition is formulated for subcutaneous administration.

11. The pharmaceutical composition of claim 9 , wherein the composition is formulated for intravenous administration.

Assignments (2)
SECURITY INTEREST Recorded Mar 3, 2026
From: SCHOLAR ROCK, INC.
To: LSI FINANCING LLC, AS ADMINISTRATIVE AGENT
Reel/Frame 075015/0854 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Dec 24, 2024
From: CHAPRON, CHRISTOPHER; STREICH, FREDERICK C., JR.; COTE, SHAUN M.; DANEHY, FRANCIS, JR.; HUANG, YAN; JACKSON, JUSTIN W.; KAVOSI, MANIA; KUMAR, SANDEEP; CANONICO, KALEIGH; FULHAM, MELISSA
To: SCHOLAR ROCK, INC.
Reel/Frame 069676/0515 →
Continuity (6)
Continuation PCTUS2023085574 · Dec 21, 2023
Provisional Application 63588081 · Oct 5, 2023
Provisional Application 63515267 · Jul 24, 2023
Provisional Application 63477552 · Dec 28, 2022
Provisional Application 63476908 · Dec 22, 2022
Related Publication 20250122274A1 · Apr 17, 2025
References Cited (400)
US 3773919A · Boswell et al. · 1973 [cited by applicant]
US 4485045A · Regen · 1984 [cited by applicant]
US 4544545A · Ryan et al. · 1985 [cited by applicant]
US 4816397A · Boss et al. · 1989 [cited by applicant]
US 4816567A · Cabilly et al. · 1989 [cited by applicant]
US 5013556A · Woodle et al. · 1991 [cited by applicant]
US 5223409A · Ladner et al. · 1993 [cited by applicant]
US 5981568A · Kunz et al. · 1999 [cited by applicant]
US 6096506A · Lee et al. · 2000 [cited by applicant]
US 6172197B1 · Mccafferty et al. · 2001 [cited by applicant]
US 6291158B1 · Winter et al. · 2001 [cited by applicant]
US 6582915B1 · Griffiths et al. · 2003 [cited by applicant]
US 6593081B1 · Griffiths et al. · 2003 [cited by applicant]
US 6656475B1 · Lee et al. · 2003 [cited by applicant]
US 6696245B2 · Winter et al. · 2004 [cited by applicant]
US 6858208B2 · Lee et al. · 2005 [cited by applicant]
US 7083784B2 · Dall'acqua et al. · 2006 [cited by applicant]
US 7138501B2 · Ruben et al. · 2006 [cited by applicant]
US 7566768B1 · Lee et al. · 2009 [cited by applicant]
US 10287345B2 · Donovan et al. · 2019 [cited by applicant]
US 10307480B2 · Straub et al. · 2019 [cited by applicant]
US 10751413B2 · Carven et al. · 2020 [cited by applicant]
US 10882904B2 · Donovan et al. · 2021 [cited by applicant]
US 10934349B2 · Pordy et al. · 2021 [cited by applicant]
US 10946036B2 · Long et al. · 2021 [cited by applicant]
US 11135291B2 · Straub et al. · 2021 [cited by applicant]
US 11155611B2 · Donovan et al. · 2021 [cited by applicant]
US 11439704B2 · Carven et al. · 2022 [cited by applicant]
US 11925683B2 · Straub et al. · 2024 [cited by applicant]
US 12006359B2 · Donovan et al. · 2024 [cited by applicant]
US 20020157126A1 · Lee et al. · 2002 [cited by applicant]
US 20030167492A1 · Lee et al. · 2003 [cited by applicant]
US 20050049402A1 · Babcook et al. · 2005 [cited by applicant]
US 20050143306A1 · Junker et al. · 2005 [cited by applicant]
US 20060025340A1 · Knopf et al. · 2006 [cited by applicant]
US 20060216279A1 · Glass et al. · 2006 [cited by applicant]
US 20060263354A1 · Chin et al. · 2006 [cited by applicant]
US 20070087000A1 · Walsh et al. · 2007 [cited by applicant]
US 20070178095A1 · Smith et al. · 2007 [cited by applicant]
US 20070218067A1 · Buttner et al. · 2007 [cited by applicant]
US 20080119426A1 · Dale · 2008 [cited by applicant]
US 20080213251A1 · Sexton et al. · 2008 [cited by applicant]
US 20080299126A1 · Han et al. · 2008 [cited by applicant]
US 20090031435A1 · Stockwell et al. · 2009 [cited by applicant]
US 20090131638A1 · Davies et al. · 2009 [cited by applicant]
US 20090148436A1 · Lavallie et al. · 2009 [cited by applicant]
US 20090324590A1 · Kambadur et al. · 2009 [cited by applicant]
US 20100080811A1 · Davies et al. · 2010 [cited by applicant]
US 20100087631A1 · Han et al. · 2010 [cited by applicant]
US 20100166764A1 · Sayers et al. · 2010 [cited by applicant]
US 20100183616A1 · Green et al. · 2010 [cited by applicant]
US 20100221777A1 · Choe et al. · 2010 [cited by applicant]
US 20100331252A1 · Hamrick et al. · 2010 [cited by applicant]
US 20110165175A1 · Linhard et al. · 2011 [cited by applicant]
US 20110239317A1 · Lee et al. · 2011 [cited by applicant]
US 20110256132A1 · Ashman et al. · 2011 [cited by applicant]
US 20110293630A1 · Stitt et al. · 2011 [cited by applicant]
US 20130065820A1 · Bower et al. · 2013 [cited by applicant]
US 20130209489A1 · Han et al. · 2013 [cited by applicant]
US 20130216548A1 · Neijssen et al. · 2013 [cited by applicant]
US 20130230515A1 · Han et al. · 2013 [cited by applicant]
US 20130336982A1 · Mader et al. · 2013 [cited by applicant]
US 20140017262A1 · Sanicola-Nadel et al. · 2014 [cited by applicant]
US 20140023638A1 · Lavallie et al. · 2014 [cited by applicant]
US 20160074474A1 · Passini et al. · 2016 [cited by applicant]
US 20160199458A1 · Knopf et al. · 2016 [cited by applicant]
US 20170198032A1 · Donovan et al. · 2017 [cited by applicant]
US 20170333558A1 · Straub et al. · 2017 [cited by applicant]
US 20210046180A1 · Carven et al. · 2021 [cited by applicant]
US 20210283166A1 · Long et al. · 2021 [cited by applicant]
US 20210332117A1 · Donovan et al. · 2021 [cited by applicant]
US 20240002490A1 · Nomikos et al. · 2024 [cited by applicant]
US 20240368262A1 · Long et al. · 2024 [cited by applicant]
AU 2011244851A1 · 2011 [cited by applicant]
CN 103097415A · 2013 [cited by applicant]
EP 0171496A2 · 1986 [cited by applicant]
EP 0173494A2 · 1986 [cited by applicant]
EP 2853898A1 · 2015 [cited by applicant]
EP 3922645A1 · 2021 [cited by applicant]
GB 2177096B · 1989 [cited by applicant]
JP 2003520839A · 2003 [cited by applicant]
JP 2009545313A · 2009 [cited by applicant]
JP 2010502633A · 2010 [cited by applicant]
KR 20070105685A · 2007 [cited by applicant]
WO WO9002809A1 · 1990 [cited by applicant]
WO WO9117271A1 · 1991 [cited by applicant]
WO WO9201047A1 · 1992 [cited by applicant]
WO WO9209690A2 · 1992 [cited by applicant]
WO WO9215679A1 · 1992 [cited by applicant]
WO WO9218619A1 · 1992 [cited by applicant]
WO WO9220791A1 · 1992 [cited by applicant]
WO WO9301288A1 · 1993 [cited by applicant]
WO WO96001845A1 · 1996 [cited by applicant]
WO WO0053211A2 · 2000 [cited by applicant]
WO WO2002009641A2 · 2002 [cited by applicant]
WO WO2002085306A2 · 2002 [cited by applicant]
WO WO2003027248A2 · 2003 [cited by applicant]
WO WO2004009776A2 · 2004 [cited by applicant]
WO WO2004024890A2 · 2004 [cited by applicant]
WO WO2004037861A2 · 2004 [cited by applicant]
WO WO2005066204A2 · 2005 [cited by applicant]
WO WO2005084699A1 · 2005 [cited by applicant]
WO WO2005103081A2 · 2005 [cited by applicant]
WO WO2005115439A2 · 2005 [cited by applicant]
WO WO2006116269A2 · 2006 [cited by applicant]
WO WO2007024535A2 · 2007 [cited by applicant]
WO WO2007044411A2 · 2007 [cited by applicant]
WO WO2007047112A2 · 2007 [cited by applicant]
WO WO2007061995A2 · 2007 [cited by applicant]
WO WO2007044411A3 · 2007 [cited by applicant]
WO WO2008030367A2 · 2008 [cited by applicant]
WO WO2008067480A2 · 2008 [cited by applicant]
WO WO2008119426A1 · 2008 [cited by applicant]
WO WO2009038760A2 · 2009 [cited by applicant]
WO WO2010070094A1 · 2010 [cited by applicant]
WO WO2010125003A1 · 2010 [cited by applicant]
WO WO2010144452A1 · 2010 [cited by applicant]
WO WO2011122011A2 · 2011 [cited by applicant]
WO WO2011150008A1 · 2011 [cited by applicant]
WO WO2012024242A1 · 2012 [cited by applicant]
WO WO2013071056A2 · 2013 [cited by applicant]
WO WO2013072902A1 · 2013 [cited by applicant]
WO WO2013074557A1 · 2013 [cited by applicant]
WO WO2013148284A1 · 2013 [cited by applicant]
WO WO2013165972A2 · 2013 [cited by applicant]
WO WO2013186719A1 · 2013 [cited by applicant]
WO WO2014074532A2 · 2014 [cited by applicant]
WO WO2014182676A2 · 2014 [cited by applicant]
WO WO2015070158A1 · 2015 [cited by applicant]
WO WO2015195094A1 · 2015 [cited by applicant]
WO WO2016073853A1 · 2016 [cited by applicant]
WO WO2016073879A2 · 2016 [cited by applicant]
WO WO2016073906A2 · 2016 [cited by applicant]
WO WO2016098357A1 · 2016 [cited by applicant]
WO WO2016168613A1 · 2016 [cited by applicant]
WO WO2017049011A1 · 2017 [cited by applicant]
WO WO2017120523A2 · 2017 [cited by applicant]
WO WO2017218592A1 · 2017 [cited by applicant]
WO WO2018116201A1 · 2018 [cited by applicant]
WO WO2018129395A1 · 2018 [cited by applicant]
WO WO2019193204A1 · 2019 [cited by applicant]
WO WO2020160291A2 · 2020 [cited by applicant]
WO WO2022093724A1 · 2022 [cited by applicant]
WO WO2022164351A1 · 2022 [cited by applicant]
WO WO2022271867A1 · 2022 [cited by applicant]
WO WO2023215384A2 · 2023 [cited by applicant]
WO WO2024064842A1 · 2024 [cited by applicant]
Abdiche et al., “Antibodies Targeting Closely Adjacent or Minimally Overlapping Epitopes Can Displace One Another,” PLoS One. Jan. 6, 2017;12(1):1-22. [cited by applicant]
Abdiche et al., “High-throughput epitope binning assays on label-free array-based biosensors can yield exquisite epitope discrimination that facilitates the selection of monoclonal antibodies with functional activity,” … [cited by applicant]
Ader, D., “Developing the patient-reported outcomes measurement information system (PROMIS),” Medical care 45.5 (2007): S1-S2. [cited by applicant]
Al-Zaidy et al., “Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy,” J Neuromuscul Dis. 2015; 2(3):185-192. [cited by applicant]
Alfano et al., “Validity and reliability of the neuromuscular gross motor outcome,” Pediatric Neurology 122 (2021): 21-26. [cited by applicant]
Altschul et al., “Basic local alignment search tool,” Journal of molecular biology 215.3 (1990): 403-410. [cited by applicant]
Altschul et al., “Gapped BLAST and PSI-BLAST: a new generation of protein database search programs,” Nucleic acids research 25.17 (1997): 3389-3402. [cited by applicant]
Alves et al., “Serum creatinine is a biomarker of progressive denervation in spinal muscular atrophy,” Neurology 94.9 (2020): e921-e931. [cited by applicant]
Amato et al., “Treatment of sporadic inclusion body myositis with bimagrumab,” Neurology 83.24 (2014): 2239-2246. [cited by applicant]
Ambery et al., “MEDI0382, a GLP-1 and glucagon receptor dual agonist, in obese or overweight patients with type 2 diabetes: a randomised, controlled, double-blind, ascending dose and phase 2a study,” The Lancet 391.1014… [cited by applicant]
Amthor et al., “Lack of myostatin results in excessive muscle growth but impaired force generation, ”Proc Natl Acad Sci U S A. Feb. 6, 2007;104(6):1835-1840. [cited by applicant]
Anderson et al., “Identification of a novel pool of extracellular pro myostatin in skeletal muscle,” The Journal of Biological Chemistry, 2008, 283(11):7027-7035. [cited by applicant]
Angal et al., “A single amino acid substitution abolishes the heterogeneity of chimeric mouse/human (IgG4) antibody,” Molecular immunology 30.1 (1993): 105-108. [cited by applicant]
Annoussamy et al., “Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study,” Annals of Clinical and Translational Neurology 8.2 (2021): 359-373. [cited by applicant]
Anterolateral Systems—Deficits, 2017, [online]. Retrieved from: http://www.neuroanatomy.wisc.edu/sc97/text/p2/deficits.htm; on Jul. 11, 2017 (1 page). [cited by applicant]
Applebaum et al., “Modified 30-second Sit to Stand test predicts falls in a cohort of institutionalized older veterans,” Plos one 12.5 (2017): 1-13. [cited by applicant]
Aragon-Gawinska et al., “Nusinersen in patients older than 7 months with spinal muscular atrophy type 1: a cohort study,” Neurology 91.14 (2018): e1312-e1318. [cited by applicant]
Aronne et al., “Continued treatment with tirzepatide for maintenance of weight reduction in adults with obesity: the SURMOUNT-4 randomized clinical trial,” Jama, 331.1, (2024): 38-48. [cited by applicant]
ASIA (American Spinal Injury Association) Impairment Scale, Standard Neurological Classification of Spinal Cord Injury, (2000): 1-2. [cited by applicant]
Australian Application No. 202010134, filed Jul. 27, 2020, for Scholar Rock, Inc.: Examination Report No. 1, issued Oct. 15, 2020, 15 pages. [cited by applicant]
Awano et al., “Spinal muscular atrophy: journeying from bench to bedside,” Neurotherapeutics 11.4 (2014): 786-795. [cited by applicant]
Axente et al., “Clinical features and genetics in non-5q spinal muscular atrophy caused by acid ceramidase deficiency,” Journal of Medicine and Life 14.3 (2021): 1-5. [cited by applicant]
Bahne et al., “Metformin-induced glucagon-like peptide-1 secretion contributes to the actions of metformin in type 2 diabetes,” JCI insight, 3.23, (2018): 1-16. [cited by applicant]
Bailey, C., “GIP analogues and the treatment of obesity-diabetes,” Peptides. Mar. 2020;125:1-7. [cited by applicant]
Baranello et al., “Evaluation of body composition as a potential biomarker in spinal muscular atrophy,” Muscle & Nerve, 2020, 61(4):530-534. [cited by applicant]
Baranello et al., “Risdiplam in type 1 spinal muscular atrophy,” New England Journal of Medicine 384.10 (2021): 915-923. [cited by applicant]
Barrett et al., “A randomized phase 1 safety, pharmacokinetic and pharmacodynamic study of the novel myostatin inhibitor apitegromab (SRK-015): a potential treatment for spinal muscular atrophy,” Advances in Therapy 38.… [cited by applicant]
Bartels et al., “Fatigability in spinal muscular atrophy: validity and reliability of endurance shuttle tests,” Orphanet Journal of Rare Diseases 15 (2020): 1-9. [cited by applicant]
Becker et al., “Myostatin antibody (LY2495655) in older weak fallers: a proof-of-concept, randomised, phase 2 trial,” The Lancet Diabetes & Endocrinology. 2015;3(12):948-957. [cited by applicant]
Belhayara et al., “The metabolic syndrome: emerging novel insights regarding the relationship between the homeostasis model assessment of insulin resistance and other key predictive markers in young adults of Western Al… [cited by applicant]
Benatar, M., “Lost in translation: Treatment trials in the SOD1 mouse and in human ALS,” Neurobiology of Disease, 2007, 26:1-13. [cited by applicant]
Benjamini et al., “Immunology: A Short Course,” 1991, 2nd edition, p. 40 only. [cited by applicant]
Bergen et al., “Myostatin as a mediator of sarcopenia versus homeostatic regulator of muscle mass: insights using a new mass spectrometry-based assay,” Skeletal muscle 5 (2015): 1-16. [cited by applicant]
Bernardo et al., “Postnatal PPARdelta activation and myostatin inhibition exert distinct yet complimentary effects on the metabolic profile of obese insulin-resistant mice,” PLoS One, 2010, 25;5(6):1-11. [cited by applicant]
Bhattacharya et al., “Comparative analysis of silencing expression of myostatin (MSTN) and its two receptors (ACVR2A and ACVR2B) genes affecting growth traits in knock down chicken,” Sci Rep . May 24, 2019;9(1):7789 (13… [cited by applicant]
Biovendor, “Myostatin Propeptide Human, Chicken Polyclonal Antibody,” Product Data Sheet, Apr. 11, 2013, 3 pages. [cited by applicant]
Bird et al. “Single-chain antigen-binding proteins,” Science 242.4877 (1988): 423-426. [cited by applicant]
Bolon et al., “STP position paper: recommended best practices for sampling, processing, and analysis of the peripheral nervous system (nerves and somatic and autonomic ganglia) during nonclinical toxicity studies,” Toxi… [cited by applicant]
Bolon et al., “STP position paper: recommended practices for sampling and processing the nervous system (brain, spinal cord, nerve, and eye) during nonclinical general toxicity studies,” Toxicologic pathology 41.7 (2013… [cited by applicant]
Bowerman et al., “Therapeutic strategies for spinal muscular atrophy: SMN and beyond,” Dis Model Mech. 2017; 10(8):943-954. [cited by applicant]
Brakemeier et al., “Assessment of bulbar function in adult patients with 5q-SMA type 2 and 3 under treatment with nusinersen,” Brain Sciences 11.9 (2021): 1-9. [cited by applicant]
Bräuninger et al., “Epstein-Barr virus (EBV)-positive lymphoproliferations in post-transplant patients show immunoglobulin V gene mutation patterns suggesting interference of EBV with normal B cell differentiation proce… [cited by applicant]
Breitbart et al., “Highly specific detection of myostatin prodomain by an immunoradiometric sandwich assay in serum of healthy individuals and patients,” 2013, PLoS One, 8(11):1-10. [cited by applicant]
Brown et al., “Tolerance to single, but not multiple, amino acid replacements in antibody VH CDR 2: a means of minimizing B cell wastage from somatic hypermutation?” 1996, J Immunol., 156(9):3285-3291. [cited by applicant]
Burch et al., “Reduced serum myostatin concentrations associated with genetic muscle disease progression,” Journal of Neurology, 2017, 264(3):541-553. [cited by applicant]
Butler et al., “Reversing type 1 diabetes with stem cell-derived islets: a step closer to the dream?” The Journal of Clinical Investigation 132.3 (2022): 1-2. [cited by applicant]
Cabri et al., “Therapeutic peptides targeting PPI in clinical development: Overview, mechanism of action and perspectives,” Frontiers in Molecular Biosciences 8 (2021): 1-21. [cited by applicant]
Calder et al., “Small molecules in development for the treatment of spinal muscular atrophy: miniperspective,” Journal of medicinal chemistry 59.22 (2016): 10067-10083. [cited by applicant]
Calucho et al., “Correlation between SMA type and SMN2 copy number revisited: an analysis of 625 unrelated Spanish patients and a compilation of 2834 reported cases,” Neuromuscular Disorders 28.3 (2018): 208-215. [cited by applicant]
Campbell et al., “Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: results of a randomized, placebo-controlled clinical trial,” Muscle & nerve 55.4 (2017): 458-464. [cited by applicant]
Cances et al., “Natural history of type 1 spinal muscular atrophy: a retrospective, global, multicenter study,” Orphanet Journal of Rare Diseases 17.1 (2022): 1-11. [cited by applicant]
Castellana et al., “Resurrection of a clinical antibody: template proteogenomic de novo proteomic sequencing and reverse engineering of an anti-lymphotoxin-α antibody,” Proteomics. Feb. 2011;11(3):395-405. [cited by applicant]
Cava et al., “Preserving healthy muscle during weight loss,” Advances in nutrition 8.3 (2017): 511-519. [cited by applicant]
Cavagnaro, J., “Preclinical safety evaluation of biotechnology-derived pharmaceuticals,” Nature Reviews Drug Discovery 1.6 (2002): 469-475. [cited by applicant]
Cedarbaum et al., “The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. BDNF ALS Study Group (Phase III),” Journal of the neurological sciences 169.1-2 (1999): 13-21. [cited by applicant]
Chan et al., “Bone Geometry Is Altered by Follistatin-Induced Muscle Growth in Young Adult Male Mice,” JBMR Plus, 2021, 5(4):1-12. [cited by applicant]
Chen et al., “Considerations for Developing Combination Therapies in SMA,” Cure SMA Researcher Meeting, Jun. 16, 2016, 57 pages. [cited by applicant]
Chen et al., “The development and validation of a dysphagia-specific quality-of-life questionnaire for patients with head and neck cancer: the MD Anderson dysphagia inventory,” Archives of Otolaryngology—Head & Neck Sur… [cited by applicant]
Chiriboga et al., “Longer-Term treatment with nusinersen: results in Later-onset spinal muscular atrophy from the shine study (1661),” Neurology 94.15 Supplement (2020): 1-3. [cited by applicant]
Chiriboga et al., “Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy,” Neurology 86.10 (2016): 890-897. [cited by applicant]
Chitramuthu et al., “Progranulin modulates zebrafish motoneuron development in vivo and rescues truncation defects associated with knockdown of Survival motor neuron 1,” Molecular neurodegeneration 5 (2010): 1-13. [cited by applicant]
Ciciliot et al., “Muscle type and fiber type specificity in muscle wasting,” Int J Biochem Cell Biol., 2013, 45(10):2191-2199. [cited by applicant]
Clackson et al., “Making antibody fragments using phage display libraries,” Nature 352.6336 (1991): 624-628. [cited by applicant]
Cohen et al., “Muscle wasting in disease: molecular mechanisms and promising therapies,” Nat Rev Drug Discov., 2015, 14(1):58-74. [cited by applicant]
Coratti et al., “Age and baseline values predict 12 and 24-month functional changes in type 2 SMA,” Neuromuscular Disorders 30.9 (2020): 756-764. [cited by applicant]
Coratti et al., “Clinical variability in spinal muscular atrophy type III,” Annals of neurology 88.6 (2020): 1109-1117. [cited by applicant]
Corey, “Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy,” Nature Neuroscience, vol. 20, Feb. 13, 2017, pp. 497-499. [cited by applicant]
Cornell, S., “A review of GLP-1 receptor agonists in type 2 diabetes: a focus on the mechanism of action of once-weekly agents,” Journal of clinical pharmacy and therapeutics 45 (2020): 17-27. [cited by applicant]
Cote et al., “A sensitive and selective immunoassay for the quantitation of serum latent myostatin after in vivo administration of SRK-015, a selective inhibitor of myostatin activation,” SLAS Discovery: Advancing Life … [cited by applicant]
Crawford et al., “Apitegromab in Spinal Muscular Atrophy (SMA): An Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial (P15-5.005),” American Academy of Neurology, May 2022, vol. 98, No. 18 Supp, 8 pages. [cited by applicant]
Crawford et al., “Relationship of pharmacokinetics and pharmacodynamics to apitegromab efficacy in patients with later-onset spinal muscular atrophy (Types 2 and 3 SMA): Results from the TOPAZ study,” Abstracts/Journal … [cited by applicant]
Cully, “Beefing up the right splice variant to treat spinal muscular atrophy,” Nat Rev Drug Discov, 2014, 13, (1 pg.). [cited by applicant]
Cure SMA Presentation filed in opposition of EP Patent No. 3368069 on Apr. 27, 2022 (86 pgs.). [cited by applicant]
D'Ydewalle et al., “Spinal muscular atrophy therapeutics: where do we stand?,” Neurotherapeutics, (2015), 12(2):303-316. [cited by applicant]
Dagbay et al., “Structural basis of specific inhibition of extracellular activation of pro- or latent myostatin by the monoclonal antibody SRK-015,” J. Biol. Chem., 2020, 295(16):5404-5418. [cited by applicant]
Dalbo et al., “Testosterone and trenbolone enanthate increase mature myostatin protein expression despite increasing skeletal muscle hypertrophy and satellite cell number in rodent muscle,” Andrologia, 2017, 49(3):1-11. [cited by applicant]
Dankbar et al. “Myostatin—a new player in inflammatory bone loss,” Annals of the Rheumatic Diseases 70.Suppl 2 (2011): A75-A76. [cited by applicant]
Darras et al., “Nusinersen in later-onset spinal muscular atrophy: long-term results from the phase 1/2 studies,” Neurology 92.21 (2019): e2492-e2506. [cited by applicant]
Darras et al., “Risdiplam-treated infants with type 1 spinal muscular atrophy versus historical controls,” New England Journal of Medicine 385.5 (2021): 427-435. [cited by applicant]
David et al., “Identification of BMP9 and BMP10 as functional activators of the orphan activin receptor-like kinase 1 (ALK1) in endothelial cells,” Blood 109.5 (2007): 1953-1961. [cited by applicant]
Day et al., “Advances and limitations for the treatment of spinal muscular atrophy,” BMC pediatrics 22.1 (2022): 1-15. [cited by applicant]
Day et al., “Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial,” Lancet Neu… [cited by applicant]
De Onis et al., “The WHO Multicentre Growth Reference Study: planning, study design, and methodology,” Food and nutrition bulletin 25 (2004): S15-S26. [cited by applicant]
Deguise et al., “New insights into SMA pathogenesis: immune dysfunction and neuroinflammation,” Ann Clin Transl Neurol., 2017, 4(7):522-530. [cited by applicant]
Dibernardo et al., “Translating preclinical insights into effective human trials in ALS,” Biochimica et Biophysica Acta, 2006, 1762:1139-1149. [cited by applicant]
Ding et al., “BPI-3016, a novel long-acting hGLP-1 analogue for the treatment of Type 2 diabetes mellitus,” Pharmacological Research 122 (2017): 130-139. [cited by applicant]
Dinicolantonio et al., “Postprandial insulin assay as the earliest biomarker for diagnosing pre-diabetes, type 2 diabetes and increased cardiovascular risk,” Open Heart 4.2 (2017): 1-4. [cited by applicant]
Du et al., “Metformin in therapeutic applications in human diseases: Its mechanism of action and clinical study,” Molecular Biomedicine 3.1 (2022): 1-32. [cited by applicant]
Ducata et al., “Solution equilibrium titration for high-throughput affinity estimation of unpurified antibodies and antibody fragments,” Journal of Biomolecular Screening 20.10 (2015): 1256-1267. [cited by applicant]
Dunaway Young et al., “Scoliosis surgery significantly impacts motor abilities in higher-functioning individuals with spinal muscular atrophy,” Journal of Neuromuscular Diseases 7.2 (2020): 183-192. [cited by applicant]
Dunaway Young et al., “Six-minute walk test is reliable and valid in spinal muscular atrophy,” Muscle & nerve 54.5 (2016): 836-842. [cited by applicant]
Duong et al., “Use of the children's hospital of Philadelphia infant test of neuromuscular disorders (CHOP Intend) in X-linked myotubular myopathy: content validity and psychometric performance,” Journal of Neuromuscula… [cited by applicant]
Dwivedi et al., “Validation of the Sydney Swallow Questionnaire (SSQ) in a cohort of head and neck cancer patients,” Oral oncology 46.4 (2010): e10-e14. [cited by applicant]
Egerman et al., “GDF11 Increases with Age and Inhibits Skeletal Muscle Regeneration,” Cell Metabolism, 2015, 22(1):164-174. [cited by applicant]
Ellulu et al., “Obesity and inflammation: the linking mechanism and the complications,” Archives of medical science 13.4 (2017): 851-863. [cited by applicant]
EMA (European Medicines Agency)., Zolgensma, European Public Assessment Report (EPAR). EMA/200482/2020. (2020): 1-150. [cited by applicant]
Eppstein et al., “Biological activity of liposome-encapsulated murine interferon gamma is mediated by a cell membrane receptor,” Proceedings of the National Academy of Sciences 82.11 (1985): 3688-3692. [cited by applicant]
European Patent Application No. 16828657.3, by Scholar Rock, Inc.: Supplementary European Search Report and Opinion, dated Mar. 20, 2019, 14 pages. [cited by applicant]
European Patent Application No. 20179533.3, by Scholar Rock, Inc.: Partial European Search Report, dated Mar. 31, 2021, 12 pgs. [cited by applicant]
European Patent Application No. 20193425.4, by Scholar Rock, Inc.: European Search Report, dated Apr. 1, 2021, 9 pgs. [cited by applicant]
Extended European Search Report in EP Application No. 21170667.6 dated Nov. 11, 2021, 19 pages. [cited by applicant]
Extended European Search Report in EP23158609.0, dated Aug. 8, 2023, 27 pages. [cited by applicant]
Fallah et al., “Comparison of T1-weighted 2D TSE, 3D SPGR, and two-point 3D Dixon MRI for automated segmentation of visceral adipose tissue at 3 Tesla,” Magnetic Resonance Materials in Physics, Biology and Medicine 30 (… [cited by applicant]
Farrar et al., “Emerging therapies and challenges in spinal muscular atrophy” Ann Neural, 2017, 81(3): 355-368. [cited by applicant]
Feng et al., “Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset,” Human Molecular Genetics, 2016, 25(5):964-975. [cited by applicant]
Ferrara et al., “Recombinant renewable polyclonal antibodies,” MAbs, 2015, 7(1):32-41. [cited by applicant]
Fidler, “Scholar Rock Rolls Up $36M To Move Muscle Drug To Clinical Trials,” 2016, https://xconomy.com/boston/2016/01/04/scholar-rock-rolls-up-36m-to-move-muscle-drug-to-clinical-trials/, 3 pages. [cited by applicant]
Finkel et al., “Nusinersen versus sham control in infantile-onset spinal muscular atrophy,” New England Journal of Medicine 377.18 (2017): 1723-1732. [cited by applicant]
Finkel et al., “Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study,” The Lancet 388.10063 (2016): 3017-3026. [cited by applicant]
Fock et al., “Diet and exercise in management of obesity and overweight,” J Gastroenterol Hepatol. Dec. 2013; 28 Suppl 4:59-63. [cited by applicant]
Foster, “Malonyl-CoA: the regulator of fatty acid synthesis and oxidation”, J Clin Invest. 2012;122(6):1958-1959. [cited by applicant]
Frohlich et al., “GDF11 inhibits adipogenesis and improves mature adipocytes metabolic function via WNT/β-catenin and ALK5/SMAD2/3 pathways,” Cell Proliferation 55.10 (2022): 1-15. [cited by applicant]
Garito et al., “Bimagrumab improves body composition and insulin sensitivity in insulin-resistant individuals,” Diabetes, obesity and metabolism 20.1 (2018): 94-102. [cited by applicant]
Gascon et al., “Non-viral delivery systems in gene therapy,” Chapter 1 in: Gene therapy-tools and potential applications. IntechOpen, (2013) 1-43. [cited by applicant]
GDF-11/BMP-11 Mouse anti-Human, Clone: 743833, R&D Systems(TM), [online]. Retrieved from: http://www.fishersci.co.uk/shop/products/gdf-11bpm-11-mouse-anti-human-clone-743833-r-d-systems/15724724; on Feb. 28, 2019 (4 pgs… [cited by applicant]
Ge et al., “GDF11 Forms a Bone Morphogenetic Protein 1-Activated Latent Complex That Can Modulate Nerve Growth Factor-Induced Differentiation of PC12 Cells,” Molecular and Cellular Biology, 2005, 25(14):5846-5858. [cited by applicant]
Giangregorio et al., “Bone Loss and Muscle Atrophy in Spinal Cord Injury: Epidemiology, Fracture Prediction, and Rehabilitation Strategies,” J Spinal Cord Med., 2006, 29(5):489-500. [cited by applicant]
Glanzman et al., “The Children's Hospital of Philadelphia infant test of neuromuscular disorders (CHOP Intend): test development and reliability,” Neuromuscular Disorders 20.3 (2010): 155-161. [cited by applicant]
Glanzman et al., “Validation of the Expanded Hammersmith Functional Motor Scale in spinal muscular atrophy type II and III,” Journal of child neurology 26.12 (2011): 1499-1507. [cited by applicant]
Godoy-Matos et al., “NAFLD as a continuum: from obesity to metabolic syndrome and diabetes,” Diabetology & metabolic syndrome 12 (2020): 1-20. [cited by applicant]
Gogliotti et al., “Characterization of a commonly used mouse model of SMA reveals increased seizures susceptibility and heightened fear response in FVB/N mice,” Neurobiol Dis., 2011, 43(1):142-151. [cited by applicant]
Golan et al., “LY2495655, an antimyostatin antibody, in pancreatic cancer: a randomized, phase 2 trial,” Journal of Cachexia, Sarcopenia and Muscle. 2018;9(5):871-879. [cited by applicant]
Golay, A., “Metformin and body weight,” International Journal of Obesity 32.1 (2008): 61-72. [cited by applicant]
Gonzalez et al., “BMP-1/Tolloid-like Metalloproteases Process Endorepellin, the Angiostatic C-terminal Fragment of Perlecan,” The Journal of Biological Chemistry, 2005, 280(8):7080-7087. [cited by applicant]
Gonzalez Trotter et al., “34-OR: The Effect of Combined Activin A and Myostatin Blockade on Body Composition—A Phase 1 Trial. Diabetes,” 2024;73(Supplement_1), 4 pages. [cited by applicant]
Gorgey et al., “Effects of spinal cord injury on body composition and metabolic profile—Part I” J Spinal Cord Med. 2014; 37(6):693-702. [cited by applicant]
Goyal et al., “Evaluation of TNF-α and IL-6 levels in obese and non-obese diabetics: pre-and postinsulin effects,” North American journal of medical sciences 4.4 (2012): 180-184. [cited by applicant]
Graham et al., “A Soluble Myostatin Inhibitor Does Not Prevent Sublesional Muscle Atrophy 56 Days After Spinal Cord Injury in Mice,” Medicine & Science in Sports & Exercise, 2015, Abstract No. 2219:587. [cited by applicant]
Guidance for Industry: S6 Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals. FDA, Jul. 1997, 1-14. [cited by applicant]
Guidance for Industry: S9 Nonclinical Evaluation for Anticancer Pharmaceuticals. FDA, Mar. 2010, 1-12. [cited by applicant]
“Guideline on development, production, characterization and specification for monoclonal antibodies and related products,” European Medicines Agency, (2016): 1-13. [cited by applicant]
Guo et al., “Myostatin Inhibition in Muscle, but Not Adippose Tissue, Decreases Fat Mass and Improves Insulin Sensitivity,” PLoS One, 2009, 4(3):1-11. [cited by applicant]
Gupta et al., “Myopathy associated with statins and SGLT2—a review of literature,” Current problems in cardiology 46.4 (2021): 1-13. [cited by applicant]
Hagan, “When are mice considered old?” 2017, https://www.iax.org/news-and insights/jax-blog/2017/november/when-are-mice-considered-old, 2 pages. [cited by applicant]
Haley et al., “Assessing mobility in children using a computer adaptive testing version of the pediatric evaluation of disability inventory,” Archives of Physical Medicine and Rehabilitation 86.5 (2005): 932-939. [cited by applicant]
Hamrick, M., “The skeletal muscle secretome: an emerging player in muscle-bone crosstalk,” Bonekey Rep. Apr. 11, 2012;1:(2012): 1-5. [cited by applicant]
Hanna et al., “Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial,” The Lancet Neurology 18.9 (2019): 834-844. [cited by applicant]
Hansen et al., “Incretin mimetics: a novel therapeutic option for patients with type 2 diabetes—a review,” Diabetes Metab Syndr Obes. May 17, 2010;3:155-63. [cited by applicant]
Harms et al., “Brown and beige fat: development, function and therapeutic potential,” Nature medicine 19.10 (2013): 1252-1263. [cited by applicant]
Herweijer et al., “Progress and prospects: naked DNA gene transfer and therapy,” Gene therapy 10.6 (2003): 453-458. [cited by applicant]
Heymsfield et al., “Effect of Bimagrumab vs Placebo on Body Fat Mass Among Adults With Type 2 Diabetes and Obesity: A Phase 2 Randomized Clinical Trial,” JAMA Network. 4 (2021):1-13. [cited by applicant]
History of Change for Study NCT05115110: A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Ambulat Spinal Muscular Atrophy, Nov. 2021, URL:https://clinicaltrials.go… [cited by applicant]
History of Changes for Study NCT03921528: An Active Treatment Study of SRK-015 in Patients with Type 2 or Type 3 Spinal Muscular Atrophy, Jan. 2020. URL:https://clinicaltrials.gov/ct2/history/NCT03921528?V_13=View#Study… [cited by applicant]
Holliger et al., “Diabodies”: small bivalent and bispecific antibody fragments, Proceedings of the National Academy of Sciences 90.14 (1993): 6444-6448. [cited by applicant]
Holzbaur et al., “Myostatin inhibition slows muscle atrophy in rodent models of amyotrophic lateral sclerosis,” Neurobiol Dis., 2006, 23(3):697-707. [cited by applicant]
Hori et al., “Elimination of plasma soluble antigen in cynomolgus monkeys by combining pH-dependent antigen binding and novel Fc engineering” MAbs., 2022, 14(1):1-12. [cited by applicant]
Hrebicek et al., “Detection of insulin resistance by simple quantitative insulin sensitivity check index QUICKI for epidemiological assessment and prevention,” The Journal of Clinical Endocrinology & Metabolism 87.1 (20… [cited by applicant]
Hua et al., “Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model,” Genes & development 24.15 (2010): 1634-1644. [cited by applicant]
Hua et al., “Enhancement of SMN2 Exon 7 inclusion by Antisense Oligonucleotides Targeting the Exon,” PLOS Biology 5.4 (2007): 0729-0742. [cited by applicant]
Hua et al., “Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model,” Nature 478.7367 (2011): 123-126. [cited by applicant]
Human Myostatin ELISA—Prodomain specific, BioVendor Laboratory Medicine, Inc., 2005, XP055100354, Retrieved from the Internet: URL:http://deltaclon.es/pdf/RD193058100.pdf, 10 pages. [cited by applicant]
Huston et al., “Protein engineering of antibody binding sites: recovery of specific activity in an anti-digoxin single-chain Fv analogue produced in [cited by applicant]
Hwang et al., “Hepatic uptake and degradation of unilamellar sphingomyelin/cholesterol liposomes: a kinetic study,” Proceedings of the National Academy of Sciences 77.7 (1980): 4030-4034. [cited by applicant]
Iannaccone et al., “The PedsQL™ in pediatric patients with spinal muscular atrophy: feasibility, reliability, and validity of the pediatric quality of life inventory™ generic core scales and neuromuscular module,” Neuro… [cited by applicant]
Igawa et al., “Engineered Monoclonal Antibody with Novel Antigen-Sweeping Activity In Vivo”. PLoS One, 2013, 8(5): 1-10. [cited by applicant]
Igawa et al., “pH-dependent antigen-binding antibodies as a novel therapeutic modality,” Biochimica et Biophysica Acta 1844 (2014) 1943-1950. [cited by applicant]
Incretin mimetic drugs for type 2 diabetes—FDA, downloaded on Sep. 9, 2024, 1 page. [cited by applicant]
International Preliminary Report on Patentability for Application No. PCT/US2015/059468, dated May 9, 2017 (12 pgs.). [cited by applicant]
International Preliminary Report on Patentability for Application No. PCT/US2015/059515, dated May 9, 2017 (12 pgs.). [cited by applicant]
International Preliminary Report on Patentability for Application No. PCT/US2015/059557, dated May 9, 2017 (8 pgs.). [cited by applicant]
International Preliminary Report on Patentability for Application No. PCT/US2016/052014, dated Mar. 20, 2018 (11 pgs.). [cited by applicant]
International Search Report and Written Opinion for Application No. PCT/US2016/052014, dated Jan. 9, 2017 (17 pgs.). [cited by applicant]
International Search Report and Written Opinion in International Application No. PCT/US2016/043712, by Scholar Rock, Inc., mailed Jan. 13, 2017, 22 pages. [cited by applicant]
International Search Report and Written Opinion in International Application No. PCT/US2017/012606, by Scholar Rock, Inc., mailed Jul. 24, 2017, 35 pages. [cited by applicant]
International Search Report and Written Opinion in International Application No. PCT/US2017/037332, by Scholar Rock, Inc., mailed Nov. 14, 2017, 19 pages. [cited by applicant]
International Search Report and Written Opinion in International Application No. PCT/US2018/012686, by Scholar Rock, Inc., mailed Apr. 3, 2018, 13 pages. [cited by applicant]
International Search Report and Written Opinion of the International Searching Authority received in PCT/US2021/056517, mailed Mar. 2, 2022 (23 pgs.). [cited by applicant]
International Search Report and Written Opinion of the International Searching Authority received in PCT/US2022/034588, mailed on Oct. 6, 2022 (10 pages). [cited by applicant]
International Search Report and Written Opinion of the International Searching Authority received in PCT/US2023/020843, mailed Oct. 30, 2023 (19 pgs.). [cited by applicant]
International Search Report and Written Opinion of the International Searching Authority received in PCT/US2023/085574, mailed May 7, 2024 (16 pgs.). [cited by applicant]
International Search Report for Application No. PCT/US2015/059468, dated Apr. 4, 2016 (6 pgs.). [cited by applicant]
International Search Report for Application No. PCT/US2015/059515, dated Mar. 25, 2016 (8 pgs.). [cited by applicant]
International Search Report for Application No. PCT/US2015/059557, dated May 19, 2016 (5 pgs.). [cited by applicant]
Ito et al., “Skeletal muscle atrophy and short-term mortality in patients with acute exacerbation of idiopathic pulmonary fibrosis: an observational cohort study,” Respiratory Investigation 61.4 (2023): 371-378. [cited by applicant]
Jablonka et al., “Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders,” Neurol Res Pract., 2022, 4(7):522-530. [cited by applicant]
Japanese Patent Application No. 2019-517209, filed Jun. 13, 2017, by Scholar Rock, Inc., Decision to Grant a Patent, mailed Dec. 8, 2020 (7 pgs.). [cited by applicant]
Jarolim et al., “Determination of Cardiac Troponin with a Single-Molecule High-Sensitivity Assay and Outcomes in Patients with Stable Coronary Artery Disease: Analysis from Prove IT-TIMI 22,” 2013 AACC Annual Meeting Ab… [cited by applicant]
Jedrzejowska, M., “Advances in newborn screening and presymptomatic diagnosis of spinal muscular atrophy,” Degenerative Neurological and Neuromuscular Disease (2020): 39-47. [cited by applicant]
Jiang et al., “Genomic analysis of a spinal muscular atrophy (SMA) discordant family identifies a novel mutation in TLL2, an activator of growth differentiation factor 8 (myostatin): a case report,” BMC Medical Genetics… [cited by applicant]
Jobling et al., “Isoform-specific activation of latent transforming growth factor beta (LTGF-beta) by reactive oxygen species,” Radiat Res., 2006, 166(6):839-848. [cited by applicant]
Jones et al., “A 30-s chair-stand test as a measure of lower body strength in community-residing older adults,” Research quarterly for exercise and sport 70.2 (1999): 113-119. [cited by applicant]
Jones et al., “Validation of quantitative magnetic resonance for the determination of body composition of mice,” International journal of body composition research 7.2 (2009): 67-72. [cited by applicant]
Kariya et al., “Requirement of enhanced Survival Motoneuron protein imposed during neuromuscular junction maturation,” The Journal of Clinical Investigation, 2014, 124(2):785-800. [cited by applicant]
Karlin et al., “Applications and statistics for multiple high-scoring segments in molecular sequences,” Proceedings of the National Academy of Sciences 90.12 (1993): 5873-5877. [cited by applicant]
Karlin et al., “Methods for assessing the statistical significance of molecular sequence features by using general scoring schemes,” Proceedings of the National Academy of Sciences 87.6 (1990): 2264-2268. [cited by applicant]
Kaufmann et al., “Observational study of spinal muscular atrophy type 2 and 3: functional outcomes over 1 year,” Arch Neurol. Jun. 2011;68(6):779-86. [cited by applicant]
Kaufmann et al., “Prospective cohort study of spinal muscular atrophy types 2 and 3,” Neurology 79.18 (2012): 1889-1897. [cited by applicant]
Knappik et al., “Recombinant Antibody Expression and Purification,” Chapter 203 of The Protein Protocols Handbook, 2009, (pp. 1929-1943). [cited by applicant]
Kohler et al., “Continuous cultures of fused cells secreting antibody of predefined specificity,” Nature 256.5517 (1975): 495-497. [cited by applicant]
Kong et al., “Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA,” Science translational medicine 13.578 (2021): 1-30. [cited by applicant]
Kubo et al., “A new method for SMN1 and hybrid SMN gene analysis in spinal muscular atrophy using long-range PCR followed by Sequencing,” Journal of Human Genetics, Published Feb. 26, 2015, pp. 233-238. [cited by applicant]
Lakshman et al., “Measurement of myostatin concentrations in human serum: circulating concentrations in young and older men and effects of testosterone administration,” Molecular and cellular endocrinology 302.1 (2009):… [cited by applicant]
Lander et al., “Appion: an integrated, database-driven pipeline to facilitate EM image processing,” Journal of structural biology 166.1 (2009): 95-102. [cited by applicant]
Landfeldt et al., “Quality of life of patients with spinal muscular atrophy: a systematic review,” European journal of paediatric neurology 23.3 (2019): 347-356. [cited by applicant]
Latres et al., “Activin A more prominently regulates muscle mass in primates than does GDF8,” Nature Communications, 2017, 8:1-13. [cited by applicant]
Latres et al., “Myostatin blockade with a fully human monoclonal antibody induces muscle hypertrophy and reverses muscle atrophy in young and aged mice,” Skeletal Muscle, 2015, 5:1-13. [cited by applicant]
Le Berre et al., “The psychometric properties of a modified sit-to-stand test with use of the upper extremities in institutionalized older adults,” Perceptual and motor skills 123.1 (2016): 138-152. [cited by applicant]
Lee et al., “Regulation of muscle growth by multiple ligands signaling through activin type II receptors,” Proceedings of the National Academy of Sciences 102.50 (2005): 18117-18122. [cited by applicant]
Lee et al., “Regulation of myostatin activity and muscle growth,” Proceedings of the National Academy of Sciences 98.16 (2001): 9306-9311. [cited by applicant]
Lee, “Myostatin: A Skeletal Muscle Chalone,” Annu Rev Physiol., 2023, 10;85:269-291. [cited by applicant]
Le Verche et al., “Skeletal Muscle in Spinal Muscular Atrophy As an Opportunity for Therapeutic Intervention,” Chapter 21 in Spinal Muscular Atrophy: Disease Mechanisms and Therapy, (2017): 341-356. [cited by applicant]
Lefebvre et al., “Identification and characterization of a spinal muscular atrophy-determining gene,” Cell 80.1 (1995): 155-165. [cited by applicant]
Li et al., “Glutazumab, a novel long-lasting GLP-1/anti-GLP-1R antibody fusion protein, exerts anti-diabetic effects through targeting dual receptor binding sites,” Biochemical Pharmacology 150 (2018): 46-53. [cited by applicant]
Ling et al., “Severe neuromuscular denervation of clinically relevant muscles in a mouse model of spinal muscular atrophy,” Human Molecular Genetics, 2012, vol. 21, No. 1, pp. 185-195. [cited by applicant]
Liu et al., “Activin Receptor Type 118 Inhibition Improves Muscle Phenotype and Function in a Mouse Model of Spinal Muscular Atrophy,” PLoS One, 2016, 11 (11): 1-17. [cited by applicant]
Liu et al., “New practice in semaglutide on type-2 diabetes and obesity: clinical evidence and expectation,” Frontiers of Medicine, vol. 16, No. 1, Feb. 1, 2022, 17-24. [cited by applicant]
Liu et al., “The Smn-Independent Beneficial Effect of Trichostatin A on an Intermediate Mouse Model of Spinal Muscular Atrophy,” PLOS ONE, 2014, 9(7):1-9. [cited by applicant]
Loffredo et al., “Growth differentiation factor 11 is a circulating factor that reverses age-related cardiac hypertrophy,” Cell, 2013, 153(4):828-839. [cited by applicant]
Long et al., “Specific inhibition of myostatin activation is beneficial in mouse models of SMA therapy,” Human Molecular Genetics, 2019, 28(7):1076-1089. [cited by applicant]
Lu et al., “Gdf11 gene transfer prevents high fat diet-induced obesity and improves metabolic homeostasis in obese and STZ-induced diabetic mice,” Journal of Translational Medicine 17 (2019): 1-16. [cited by applicant]
Madeira et al., “The EMBL-EBI search and sequence analysis tools APIs in 2019,” Nucleic acids research 47.W1 (2019): W636-W641. [cited by applicant]
Main et al., “The Hammersmith functional motor scale for children with spinal muscular atrophy: a scale to test ability and monitor progress in children with limited ambulation,” European Journal of Paediatric Neurology… [cited by applicant]
Malik et al., “Pediatric dose selection for therapeutic proteins,” The Journal of Clinical Pharmacology 61 (2021): S193-S206. [cited by applicant]
Mariot et al., “Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches,” Nature Communications, 2017, 8(1):1-8. [cited by applicant]
Markovits et al., “The diversity of the immune response to the A2 domain of human factor VIII,” Blood. Apr. 4, 2013;121(14):2785-95. [cited by applicant]
Marks et al., “By-passing immunization: human antibodies from V-gene libraries displayed on phage,” Journal of molecular biology 222.3 (1991): 581-597. [cited by applicant]
Mashhood et al., “Reproducibility of hepatic fat fraction measurement by magnetic resonance imaging,” Journal of Magnetic Resonance Imaging 37.6 (2013): 1359-1370. [cited by applicant]
Matsuda et al., “Insulin sensitivity indices obtained from oral glucose tolerance testing: comparison with the euglycemic insulin clamp,” Diabetes care 22.9 (1999): 1462-1470. [cited by applicant]
Mazzone et al., “Revised upper limb module for spinal muscular atrophy: development of a new module,” Muscle & nerve 55.6 (2017): 869-874. [cited by applicant]
Mcallister et al., “Modified 30-second sit-to-stand test: reliability and validity in older adults unable to complete traditional sit-to-stand testing,” Journal of geriatric physical therapy 43.3 (2020): 153-158. [cited by applicant]
Mchorney et al., “The SWAL-QOL and SWAL-CARE outcomes tool for oropharyngeal dysphagia in adults: III. Documentation of reliability and validity,” Dysphagia 17 (2002): 97-114. [cited by applicant]
Mcpherron et al., “Metabolic Functions of Myostatin and GDF11,” Immunol Endocr Metab Agents Med Chem., 2010, 10(4):217-231. [cited by applicant]
Mcpherron et al., “Regulation of skeletal muscle mass in mice by a new TGF-p superfamily member,” Nature 387.6628 (1997): 83-90. [cited by applicant]
Meece, J., “The role of the pharmacist in managing type 2 diabetes with glucagon-like peptide-1 receptor agonists as add-on therapy,” Advances in therapy 34 (2017): 638-657. [cited by applicant]
Mendell et al., “A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy,” Molecular Therapy 23.1 (2015): 192-201. [cited by applicant]
Mendell et al., “Five-year extension results of the phase 1 START trial of onasemnogene abeparvovec in spinal muscular atrophy,” JAMA neurology 78.7 (2021): 834-841. [cited by applicant]
Mercuri et al., “Long-term progression in type II spinal muscular atrophy: a retrospective observational study,” Neurology 93.13 (2019): e1241-e1247. [cited by applicant]
Mercuri et al., “Nusinersen versus sham control in later-onset spinal muscular atrophy,” New England Journal of Medicine 378.7 (2018): 625-635. [cited by applicant]
Mercuri et al., “Patterns of disease progression in type 2 and 3 SMA: implications for clinical trials,” Neuromuscular Disorders 26.2 (2016): 126-131. [cited by applicant]
Mercuri et al., “SUNFISH Part 2: Efficacy and Safety of Risdiplam (RG7916) in Patients with Type 2 or Non-Ambulant Type 3 Spinal Muscular Atrophy (SMA) (1260),” Neurology 94.15 Supplement (2020): 1-3. [cited by applicant]
Mercuri et al., Presented at World Muscle Society Congress 2020, p. 257. [cited by applicant]
Mercuri et al., “Spinal muscular atrophy—insights and challenges in the treatment era,” Nature Reviews Neurology 16.12 (2020): 706-715. [cited by applicant]
Miller et al., “The danger of weight loss in the elderly,” J Nutr Health Aging. Aug.-Sep. 2008;12(7):487-91. [cited by applicant]
Mix et al., “Quality of life in SMA patients under treatment with nusinersen,” Frontiers in neurology 12 (2021): 1-8. [cited by applicant]
Mokdad et al., “Prevalence of obesity, diabetes, and obesity-related health risk factors, 2001,” Jama 289.1 (2003): 76-79. [cited by applicant]
Monani et al., “A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2,” Human molecular genetics 8.7 (1999): 1177-1183. [cited by applicant]
Monani, U., “Spinal muscular atrophy: a deficiency in a ubiquitous protein; a motor neuron-specific disease,” Neuron 48.6 (2005): 885-896. [cited by applicant]
Montes et al., “Nusinersen improves walking distance and reduces fatigue in later-onset spinal muscular atrophy,” Muscle & nerve 60.4 (2019): 409-414. [cited by applicant]
Morrison et al., “A soluble activin type IIB receptor improves function in a mouse model of amyotrophic lateral sclerosis,” Exp Neurol, 2009, 217(2):258-268. [cited by applicant]
Morrison et al., “Chimeric human antibody molecules: mouse antigen-binding domains with human constant region domains,” Proceedings of the National Academy of Sciences 81.21 (1984): 6851-6855. [cited by applicant]
Mosler et al., “The anabolic steroid methandienone targets the hypothalamic-pituitary-testicular axis and myostatin signaling in a rat training model,” Archives of Toxicology, 2012, 86(1):109-119. [cited by applicant]
Muramatsu et al., “Novel myostatin-specific antibody enhances muscle strength in muscle disease models,” Scientifc Reports, vol. 11, No. 1, Jan. 25, 2021, 16 pages. [cited by applicant]
Nadkarni et al., “Regulation of glucose homeostasis by GLP-1,” Progress in molecular biology and translational science 121 (2014): 23-65. [cited by applicant]
Nakano et al., “Remogliflozin etabonate improves fatty liver disease in diet-induced obese male mice,” Journal of Clinical and Experimental Hepatology 5.3 (2015): 190-198. [cited by applicant]
Nardone et al., “Inflammatory bowel diseases and sarcopenia: the role of inflammation and gut microbiota in the development of muscle failure,” Frontiers in Immunology 12 (2021): 1-11. [cited by applicant]
Naryshkin et al., “SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy,” Science, 2014, vol. 345, Issue 6197, pp. 688-693. [cited by applicant]
National Cancer Institute, “Common Terminology Criteria for Adverse Events,” Version 5.0, Published Nov. 2017, 1-147. [cited by applicant]
Nayerossadat et al., “Viral and nonviral delivery systems for gene delivery,” Advanced biomedical research 1.2 (2012): 1-11. [cited by applicant]
Nguyen et al., “Oral-033: Bimagrumab + Semaglutide Causes> 30% Fat Loss and 5% Lean Mass Increase in Obese Mice After 2 Weeks,” Obesity, vol. 30, No. SI, Nov. 1, 2022, 4-54. [cited by applicant]
O'Hagen et al., “An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients,” Neuromuscular Disorders 17.9-10 (2007): 693-697. [cited by applicant]
Oestreich et al., “Myostatin deficiency partially rescues the bone phenotype of osteogenesis imperfecta model mice,” Osteoporos Int. Jan. 2016;27(1):161-70. [cited by applicant]
Ojala et al., “In Search of a Cure: The Development of Therapeutics to Alter the Progression of Spinal Muscular Atrophy,” Brain Sci., Feb. 5, 2021; 11(2):194, 39 pages. [cited by applicant]
Opposition filed in EP Patent No. 3368069 on Apr. 28, 2021 (37 pgs.). [cited by applicant]