## NAITBABIES ANNUAL REPORT 2023 News and events from 1 June 2022 – 31 May 2023 **Fetal and neonatal alloimmune thrombocytopenia - FNAIT** Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698. ## **Reference and administrative details of the charity and its Trustees** Charity name: Naitbabies Other names we are known by: NAIT babies Charity number: 1161698 Principle address: 13 Redinnick Terrace Penzance Cornwall, TR18 4HR, England Members of the CIO are also the trustees who administer the charity. Thea Palmer Chair Michelle Minshall Treasurer Stacy Corke Company Secretary Andrea Palmer Trustee **Treasurer** : Michelle Minshall Minshall & Co Bridge House Nantwich Cheshire, CW5 7JX **Independent examiner:** Mr Neill Hallam FCCA Crane and Johnston Chartered Certified Accountants 11 Alverton Terrace Penzance Cornwall, TR18 4JH **Bankers** : HSBC, 1 Green Market Penzance Cornwall, TR18 2SD **Type of governing document** : Constitution **How the charity is constituted** : Charitable Incorporated Organisation (CIO) Foundation **Date of governing document** : 15 May 2015 **Website** : www.naitbabies.org 1 Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698. ## **Naitbabies are members of:** Genetic Alliance UK Rare Disease UK ## **Mission Statement** Our mission is to be a strong voice for parents, children and families who have been diagnosed with or suspect that they may have Fetal and neonatal alloimmune thrombocytopenia - FNAIT. ## **Objectives and Activities** ## **The objects of the charity as set out in the CIO’s constitution are:** The relief of sickness and the preservation and protection of good health of parents, children and their families and carers who have or are affected by the severe bleeding disorder neonatal alloimmune thrombocytopenia – FNAIT; in particular by the provision of support, advocacy and practical assistance as the trustees shall think fit; To carry out, or to provide funds to support research into FNAIT, its causes, treatment and prevention and publish the useful results of such research; and To advance the education of the public, in particular those involved in the diagnosis, treatment and care of those suffering from FNAIT. ## **Summary of the main activities undertaken for the public benefit in relation to these objects.** Naitbabies are a small Charitable Incorporated Organisation run by families whose children have been affected by the severe bleeding disorder FNAIT – Fetal and neonatal alloimmune thrombocytopenia. Registered in the UK we are the only organisation for this disorder. We provide information to the public in general. We run an international FNAIT Parents Support Group and advocate for parents worldwide. To date we have over 1,200 members. We support research into FNAIT and have contact with an expert international medical panel of medical doctors who have a special interest in this devastating disease. Watch our FNAIT video _**here**_ 2 Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698. ## **What is FNAIT?** Fetal and neonatal alloimmune thrombocytopenia FNAIT (NAIT FMAIT) results from incompatibility between mother and baby for platelet-specific antigens that have been inherited from the father and are absent in mother. The maternal immune response may make antibodies to destroy fetal platelets which she sees as foreign. Platelet destruction may cause bleeding into all major organs e.g. the stomach, spinal cord and lungs. The most feared is bleeding into the brain known as intracranial haemorrhage - ICH. Babies are at serious risk of death or permanent neurological impairment such as epilepsy, cortical blindness, cerebral palsy, precocious puberty, motor and cognitive delays and sensory processing disorders. 3 Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698. NAIT is seen as the platelet equivalent of the red blood cell disease Hemolytic Disease of the Fetus and Newborn - HDFN more commonly known as Rhesus disease. Routine antenatal care has included screening of all expectant mothers for HDFN since the early 1970’s. No country carries out antenatal screening for NAIT although it has been known about since the 1950’s and is very well documented. Rarer diseases are also screened for. Treatment is available for mothers who have developed antibodies against their unborn baby’s platelets and the success rate is high. If subsequent pregnancies are not treated they may also be at risk. Platelets forming in bone marrow ## **Highlights of our journey through the last year.** ## **June 2022** NORD. On the 21 June 2022 Naitbabies were invited to register as a patient organisation on the North American Dare Disease Organization Database. https://rarediseases.org/non-member-patient/naitbabies/ ## **July** On July 2 Alan Halliday and his team mates Gareth Rees, Garry Bowman, Andy Donald, James Phillips, Andy Hogg, James Turner, Jeff Biggs and Nathan Turner took part in the UK Yorkshire Three Peaks Challenge a circular trek which encompasses three major peaks in the Yorkshire 4 Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698. Dales, Pen-Y-Ghent, Whernside, and Ingleborough in under 12 hours raising a magnificent £1,473.16. Alan’s 8 year old son Theo was affected by FNAIT after being born with a platelet count of only 11k. He received blood, IVIG and platelet transfusions all within the first 18 hours of his birth. On July 9-13 The International Society on Thrombosis and Haemostasis (ISTH) reconvened for the ISTH 2022 Congress held in London, England. https://www.isth2022.org/ Professor Ulrich Sachs Germany, presented the manuscript PB1110 - _Buffy_ - _coat_ - _derived megakaryocytes_ for _monoclonal antibody immobilization_ of _platelet antigens_ ( _MAIMA_ ) _assay_ . ## **August** **'** _He's never alone'_ One of our FNAIT Parents Support Group members, Carlos Marquez from New Jersey, USA, has a tattoo for each of his 5 children. This one, which depicts a shunt and the words _‘’His fight is my fight’’_ is EXTRA special as his son Luke has already been through five brain surgeries due to intracranial haemorrhages caused by FNAIT and has a shunt fitted into his brain. 5 Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698. A shunt is a hollow tube surgically placed in the brain to help drain cerebrospinal fluid and redirect it to another location in the body where it can be reabsorbed. ## **September** September 28, 2022 Preliminary data from the ongoing Phase 1b study shows RLYB212 rapidly and completely eliminates transfused HPA-1a positive platelets. More news is expected in the first quarter of 2023. Rallybio Announces Positive Preliminary Results for RLYB212, an anti-HPA-1a Monoclonal Antibody for the Prevention of Fetal and Neonatal Alloimmune Thrombocytopenia ## **October** FNAIT SURVEY. Naitbabies have been collaborating with Dr J B Bussel, Weill Cornell Medical University on a questionnaire for women who have fetal and neonatal alloimmune thrombocytopenia. This research project was started in 2020 and delayed for 2 years due to the Covid pandemic. ## **November** AGM – 28 November 2022 ## **December** Grant. In December we were delighted to be able to support Dr T W de Vos, Leiden Medical University by providing a grant of £1000 towards his important FNAIT doctorate thesis publication. ## **January 2023** 31 Jan – FNAIT Survey completed. The results will be published in 2023. ## **February** 28 February Rare Disease Day 6 Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698. ## **March** Latest news on the development of a prophylactic treatment for FNAIT. 6 March 2023, Rallybio Announced Clinical Proof-of-Concept Achieved for its Lead Product Candidate, RLYB212, a Monoclonal Anti-HPA-1a Antibody, in the First Quarter of 2023. https://investors.rallybio.com/news-releases/news-release-details/rallybio-announces-proofconcept-achieved-rlyb212-novel The Company also announced today that RLYB211, a plasma-derived polyclonal anti-HPA-1a antibody, will not be advanced further in clinical development. ## 25 – 31 March – **FNAIT Awareness Week.** Our awareness week competition was won this year by one of our FNAIT families living in Northern Ireland. ## **April** On April 13th and 14th, Leiden University Medical Center together with Sanquin (Sanquin Research and Diagnostic) Services, Holland, organized the 𝐈𝐧𝐭𝐞𝐫𝐧𝐚𝐭𝐢𝐨𝐧𝐚𝐥 𝐂𝐨𝐧𝐬𝐞𝐧𝐬𝐮𝐬 𝐌𝐞𝐞𝐭𝐢𝐧𝐠 𝐨𝐧 𝐅𝐞𝐭𝐚𝐥 𝐚𝐧𝐝 𝐍𝐞𝐨𝐧𝐚𝐭𝐚𝐥 𝐀𝐥𝐥𝐨𝐢𝐦𝐦𝐮𝐧𝐞 𝐓𝐡𝐫𝐨𝐦𝐛𝐨𝐜𝐲𝐭𝐨𝐩𝐞𝐧𝐢𝐚: 𝐅𝐍𝐀𝐈𝐓. After the conference a spokesman for LUMC posted ‘’We were pleased to welcome so many colleagues from various countries with expertise in fetal maternal medicine, in neonatology, in laboratory testing, in basic science and representatives from NAITbabies, who have direct personal experience in how FNAIT itself can impact the life of children and their families’’. ## **May** Support group Membership = 1,611 as of May 29 2023. Naitbabies CIO run a private support group for FNAIT parents with members from all over the world. ## **May 31 2023 - End of Year** ## _**Many thanks to everyone for reading our review and for your continued support!!!!!**_ **Naitbabies.org** 7 Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698. Reylytèiad Charlly no.. 1161698 Naitbable8.org Unaudited Report and Accounts 31 May 2023 Naltbables.oYg Report and account8 contents Pau Gon&Tal 5nformallon Trustees, rapDYl Ind6pèndenl Examlnels Rèpoil Recelpls and paymènts aooount 8181omonl o198sels and Ilablllli98 Nole8 lo Ihe accounls maltbables.org Ganeral Infovmatlo TYu8tèB8 Y D Palmer S Cork6 M Mlnshall A Palmor Iiidopondont Examlnèr Mr Nell Hallam FGCA Cran6 & Johnston hailerod Gorllfiod Acwuii¢onl8 11 Alverlon Terrac8 PenzanGg Corrtwall TRI8 4JH BAiikéYb Hsgc 1 Gregn Markal Penzanco Comwall TR18 28D Contact addve¥ 13 Redlnnlck Terra¢8 Ponzanog Comwall TR18 4HR ,Wgb#lté Chorllthblè Incorporotgd g¥uan18AtJon Reglslrallon numb8r-1161698 Naltbables.o¥ TrugtO85' Repovt Th8 Yru81e8s preuent Ihelr r8porl gnd aooounl8 foT th& parlod ended 84 Mgy 2028, AGtlvllle¥ N8llbables.org Is a non-prolll maklng orggnl88llon Ihal ha8 beeh oieated lo r&lge lh8 knowlgduo and awarene33 of 8 $wei'e bteodlng dlsorder many havo novey ho8Yd of, n60n8lal allglmmuno Ihrombocytopenl8 or NAiT, On 15 May 2016 Naltbabl88 bBoamo 8 reg1st￿ed ohadty and Is now a ¢haFllabl& In¢orporal8d orgAn18allon.116 re91gler8d numbor wllh Charlly Comml&&lon Is'1161tt0a'. Ml$slon Statomtsnt Our mlsslon 18 to be 4 8trong volae for parents, chlldron ahd famlllps who havè been diagn08ed wllh, or sutspgct Ih¢il Ihgy mlght havo ihe ggnallo dlsorder neonatal allolmmune IhToMho¢ytoponla. Wè $upport ro&Baroh Into t4AITIFNAIT, oBu80s, Irèalmehl and prgvonlk>n, The followlng per60n8 #B commltl¢& members durlng Ihg perlDd: Y D Palmer S Corke M Mlnshall A Palmgr Charltablè obJ8ttt Tho objects ol Ihe charlty ore &6 follow6,, 1, Tho rellel ol 8laknes$ and thè praservallon and proleolii)n of good h$allh of parents. chlldren und Ihelr famlllè8 ond c4rer8 who havg or are affec18d by Ihb gonello d180rd8r'Noonatal Allolmmun8 Thrombooylopen18' {a180 know a8 NAJT or FNAIT}. In partl¢ulAr by tha provlulon of svpporl, advooacy and praotlcal a&s181a￿e as the Iru81ees ohall ￿Ink fll,, 2. To o&iry Dtil , or ￿ provlde lunrl810 8uppoII r08earoh Inlo NAITIFNAIT, Its cau888, Iréalment and prevenllon and publlGh the u88111l rèsults of 8tsoh reBearch', and 3. To advance the gducalloh of Ilie publlo, In parlltsular th088 Involved In Ihe dlAgno¥lg, IrÈatMtI)t and car8 01 th08è 8t5ffeTlng from NAITIFNAIT. Thls r8pvrl was approv8d by thè board on ,, and slgn8d on Ils behalf. mgr Tru8tga Naltbablgs.oYg Indepondont oxainlnèes Ygport to thg Tru&t¢68 of Naltbablg$.org I report on the qecounls ol tho ohArl£y for the yéai éhded 31 May 2028, whlch qrg 561 out on pgggs 4 108, R6spectlvo roJponglbllle¥ of tiugtoos anLI oxamlrtfjr The charlly'6 IruBlao6 are re8pon8lble for Ilie wtsparallon of Iha awownt8. The ¢harlly'$ Irust¢e8 aonBldgr that an audll18 nol r6qulred for Ihl& year under seollon 144(2> ol the Cl)ailUes Act 2011 (Ih¥ 2011 Acll and that an Indapendenl eKamlnallon l¥ n8eded, It18 my roaptsn85bS11ty10: exttNlno tho attoounls under $8011on 145 ot the 2011 Aul,, lo follow Ihe procedurgs Idld down In thg General Dlrecllons glven by tho Chailly Comt))Isslon under seoiton 14615)Ib) ol Ihe 201 l Aot; 4nd lo Blgte wh6lh8r partlculqr mallars have come lo my allfjntlon. Basls of Indèpondent report My axamlnallon was oaifled out in accordanoe wllh Iho General Dlrecllon8 glven by Ihe Charlly Coinm18slono1, An tsxamlnallon Inolude8 a 1ov18w ol th9 ao¢ounllng raooids k6pi by Ilie oharlly and ¢omparlson ol tho acoounts pr680nled wllh thosg r800rds, 11 also Inolud98 consld8ralloh of any un8ual116m8 or d180108ur88 In th6 aooounts and Ih8 $8èklng of 6xplanallon8 from you au truulg8$ COn￿rnIng any such mallers, Th8 prooedures undèrtaken do not provlde all thg 6vld6nco IhBI would be Kequlred in an audSt and, oon8oquènlly no oplnlonls glven as lo whelher Iho aocount8 pr85ont a 'Iruo and fall ￿$v/ and the r¢port18 Ilmtted to IhoB¢ mall918 89t out In Ihe Statement below. Indopondotit 6xamlnel$ etatement In conne¢lloh wllh my examlnalon, no n)aller h95 cvma to my aitenllon: - whlGh g5vps mg r¢o4on$ble lo boll8ve Ihal, In any maleilal r￿Peo1, the iaqulrem8nts.' {a) to koep g¢¢ounllrvJ record8 In a￿￿C*TdanQ8 wllh Sectlon 130 of tha 2011 Aol. and (b) to preparè accounts which aocord wllh lh8 aceounllng ioÈoid& and lo comply wllh the ocwunllng requlreMont8 of the 2011 Aol have not bggn m¢1; or lo whlch, In my oplnlon, atlenlk)n should b8 drawn ID order to enable a propor undgrslandlng of the ¢Co￿nIS lo b8 reaah?d, N611 Hallam FCCA Cr&n6 & Johnslo Charl&red Cerllffed A¢ttounlants 11 Alv&rton T8rra¢a PenxBncé Cornwall TR184JH Naltbablès.ovg Recolpt8 & Paymgnts Account for the yeor 6ndècl 34 May 2023 2023 2022 Rocolpls ReGolpl8 lyom oliarltablo aotlvlllo8: Donallon8 Fund Ralslng Inl¢reyl rott8lvabb 5,829 8,647 Totdl 19001pt8 L8s8', Paymebits Cr)sl¥ of oliarlt4blp a¢tlvl(1os: Telephone ahd fax Travèl and subs181once Sl*llon8ry 8nd prfnllng SubsorlPllot)s Bank charges In8uranoa Support Sundry expen808 AcGountanw f808 Advorllslng and PR 184 168 358 31 330 152 123 81 427 233 123 1,160 48 378 11861 360 488 Sub Lotal 1,989 A860t pur¢has9S Comput9r oqulpm8nl & other equlpmenl 8ub total 2,4ts8 1,989 Total paym*n18 Not rfjcelpts Yor the yoar 3,371 6,663 Cash Al bank ahd In hand brought foward 45,611 38,Q48 Croh at bonk 41)d In hatid at 31 May 2028 Naltbablos.oYg 8t8tement of as8et8 and Ilabllltles as at 31 May 2023 Not¢ 2023 2022 Moii8t#¥y a88•ts Qgsh al b8nk ond In hand 48,987 45,611 Tolol 48,987 Unroslrloted fund6 A88al8 retalnod foi thè CIO'S owh 1È8tlmalad wrfllen down valuè> Qomputer ¢qulprnont & olher eqiilpment- unreslrtolad fvhds Computer oqulpmont & olhgr 6quipm8nl puroha8ed durlng the year- unra$trl¢l8d fundy Llablllt1è9 Grant payable - approved al year end Acoounlantty f8e- unreslrkled Iiind 1,000 396 378 378 Th9 acoounl8 were approved by Ihg Tru&lèe8 on by,, and slgned on Iholr b¢hall D Palm8r Trust68 A R Palme¥ Trusleb Naitbables.org Notes to the Accounts for the year ended 31 May 2023 1 Accountlng pollcles AccountiTng Convention The accounts have been prepared on the re￿ip1S and paymenls basis. The accounts are in accordance with applicable accounting standards and comply wilh the Charities IAccounls and Reports) Regulations issued under the Charities Act 2011. Recelpts Receipts are the total amounts received by the CIO for goods and services provided lo the public. Any donations have been included in the accounts when received. Payments Expendllure is included on a payments basis, inolusive of any VAT which cannot be recovered. 2 Funds 2023 Al 1 June 2022 Nel feceipls for the perlod 45,611 3.376 Al 31 May 2023 48,987 3 GuaranteDs The trustees confirm, in accordance wllh the Charitable Incorporated Organisalions IGenerall Regulations 2012, that al the year ending the CIO did not have any outstanding guarantees lo third parties nor any debts secured on assets ol the CIO. 4 Trusteos None of the trustees lor any persons connected with them) received any remuneration during the period. None of the Iruslees We￿ relmbursed for any expenses durlng the perlod,